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Applied Clinical Trials March 2021

Bioclinica has launched Source Document Manager. The solution enables clinical trial sponsors and contract research organizations to collect, de-identify, translate, organize, and manage source documents using a cloud-based application.

Bioclinica Source Document Manager supports online protected health information redaction at the site level with verification by a team of quality control experts and boasts direct integration with an online translation service to translate source documents. Additional features include:

Bioclinica Source Document Manager can be used as a standalone data collection and storage module to collect and review source documents online, or it can be integrated with an existing EDC system or other data tracking system to send redacted and quality-controlled source documents to their destination.

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Bioclinica Launches Source Document Manager

Rigorously designed and implemented clinical trials are at the core of delivering innovative medicines to patients, and they have been especially important when developing COVID-19 vaccines and treatments. While our understanding of medicines has progressed enormously in the past 100 years, based on the lessons we have learned from clinical trials that have been conducted, the way we design and implement clinical trials remains quite primitive.

Our analysis of 330,000 clinical trials, the majority planned and implemented in the past 15 years, make it clear that there is no such thing as a perfectly designed protocol. This clinical trial database has been built through a combination of artificial intelligence and human intelligence with data dynamically extracted and tabulated from over 80,000 sources. One third of all trials can benefit from minor edits, while another third require significant improvement, as evidenced by numerous implemented amendments. For the remaining third, they often struggle from the get-go, are prone to enrollment challenges, require many amendments, and can often be on the brink of failure. As a matter of fact, most of the final third of trials do fail.

Unfortunately, various flaws in a clinical trial design are not immediately obvious at the planning stage and when writing the protocol. Even at the stage when we need to amend a protocol, the problem in a trial design is still heavily disguised as an operational challenge, such as enrollment challenge caused by poor investigator site selection.

Many corrections to the original protocol design are not complicated. Common protocol amendments are associated with inclusion and exclusion criteria, such as age, gender, disease stages and severity. Other amendments can be more complicated and can negatively impact the operational deliverability of the study, such as selection of primary end points.

As an example, Phesi has analyzed a particular multiple sclerosis trial (NCT00355134)

 that was originally designed to include only female patients. This may appear logical as multiple sclerosis is a disease more prominent in women. However, when analyzing 29,070 patients from 91 multiple sclerosis clinical trial arms from our synthetic patient profile database, there were 66.65% women and 33.35% men, a 2:1 ratio. When the clinical trial faced operational issues, with classic linear thinking, the trial design team added 25% more investigator sites, hoping to compensate for the smaller patient population, but still achieve a similar enrollment cycle time as a multiple sclerosis trial including both genders. Even though the protocol was further amended to include both male and female patients, the enrollment cycle time was significantly longer, and the resultant patient population deviated from the general patient population of the 3:1 ratio (844 women among 1083 randomized patients (78%)), which potentially jeopardizes any medical conclusion from the trial.

When analyzing recent Phase III multiple sclerosis trials from a statistical point of view, the most dominant value in gender inclusion for this type of trial is to include both men and women. This is the modal design value—a statistical concept, which simply means ‘the most frequently used’ value in other similar clinical trials.

 In this example, just three trials out of 168 were designed to only include female patients, and of those, two ended up including both genders through protocol amendments.

Preemptive and extensive protocol design analysis conclusively shows that a clinical trial design should start from industry modal values. Let us use another multiple sclerosis clinical trial to demonstrate the power of applying modal value-based design analysis in clinical protocol design. NCT00245622 was a Phase II trial designed to evaluate Autologous T Cell Vaccine (TCV) for multiple sclerosis—undoubtedly an innovative approach in this condition’s therapy. It is not uncommon to test various innovative ideas in designing a protocol with an exciting and innovative clinical development candidate. In its original design, amongst others NCT00245622

 included the following key inclusion criteria:

Diagnosis of MS within the past three years according to the McDonald criteria (2001).

Baseline Expanded Disability Status Scale (EDSS) score between 0 and 3.5 inclusively.

In analyzing 233 Phase II MS clinical trials, Phesi detects that these trial design components significantly differ from the industry modal values.

For patient age, the modal value is 18 to 55 years old. NCT00245622 was the only analyzed trial to include patients aged 18 to 45 years.

For Expanded Disability Status Scale (EDSS), the modal value is between 0 and 5.5. An EDSS score of 0 to 3.5 in trial NCT00245622 is a notable deviation from the modal value of 0 to 5.5.

Instead of using modal values to guide protocol design, and to identify and mitigate the risk at the planning and design stage, this clinical trial team learned the lesson the hard way. After a series of protocol amendments over a 12-month period, the trial design finally came back to modal values. However, this was only after losing time and enrollment momentum, incurring significant financial costs to the study team and their stakeholders.

A planned clinical trial doesn’t have to be restricted to use industry modal values. But this integrated clinical data science approach gives the study team the opportunity to challenge why a particular value outside of modal value has been adopted. If the team is proactively aware that they are designing outside of the modal values, they can objectively and often quantitatively assess the operational risks and other associated risks with the approach. This predictive analytical approach has tremendous benefits but clearly requires a significant data pool to increase confidence. It also requires an applied combination of artificial intelligence, machine learning, natural language processing, and integrated algorithms, to extract meaning from data sets.

While we use patient eligibility as an example here, a meaningful modal value guided protocol design requires a comprehensive analysis of all protocol design elements in a structured way. This includes primary and secondary outcome measure(s), treatment duration, comparators, and so on.

Modal value guided trial design cannot be conducted in a vacuum. An optimized trial design would have the best chance of coming from an integrated approach by examining the target patient population characteristics and other variables. It will certainly benefit from understanding how different design modifications can impact the operational implementation feasibility through a detailed Gross Site Enrolment Rate (GSER) analysis.

Peter A Calabresi, et al., Safety and efficacy of fingolimod in patients with relapsing-remitting multiple sclerosis (FREEDOMS II): a double-blind, randomized, placebo-controlled, Phase III trial Lancet Neurol. 2014 Jun;13(6):545-56.

Gen Li, METHOD AND SYSTEM FOR DEVELOPING CLINICAL TRIAL PROTOCOLS, International Patent Publication No. WO2020033754, published February 13, 2020.

Edward Fox, et al. A randomized clinical trial of autologous T-cell therapy in multiple sclerosis: subset analysis and implications for trial design. Mult Scler. 2012 Jun;18(6):843-52.

The analysis of 330,000 clinical trials says the way the industry designs and implements trials remains to be quite primitive.

Modal Value Guided Clinical Trial Design Effectively Reduces Protocol Amendments

Medidata has launched Medidata Risk Management, the latest addition to its Risk-Based Quality Management (RBQM) suite, a holistic group of solutions aiming to simplify the adoption of proactive clinical operations oversight activities. These include centralized monitoring, reduced source data verification and source data review, remote source document review, and implementation of decentralized trial activities.

Built on the Medidata Clinical Cloud and fully integrated with Medidata’s centralized monitoring tool, Medidata Detect, Medidata Risk Management supports sponsors and contract research organizations (CROs) as they identify critical data and processes, associated risks, and implement mitigation strategies as defined within ICH E6 (R2). With recent updates to Medidata Detect, study teams can also compare site differences across all participants to uncover trends or anomalies and determine whether there are discrepancies in data collection or integrity to be investigated.

 Medidata Launches Holistic RBQM Functionality

For anyone who was wondering in recent weeks what was holding up FDA emergency use authorization for Johnson & Johnson to distribute vaccine produced by the Emergent BioSolutions Bayview facility in Baltimore, the reason is clear from the multiple red flags and violations at the facility cited in an April 2020 field inspection report. Even before Emergent disclosed that it had to discard 15 million doses of the J&J vaccine drug substance, FDA inspectors had uncovered multiple deficiencies and quality control issues. Journalists and investigators have disclosed additional government reports and complaints about Emergent’s operations and practices, raising multiple questions about how and why federal health officials continued to ramp up contracts with the company.

A picture of wide manufacturing violations appears in a recently disclosed FDA form 483 report on a site visit to Emergent’s Baltimore vaccine plant conducted from 4/9/2020 to 4/20/2020. The report describes inadequate controls for computer and records systems, disregard of established test procedures and laboratory control mechanisms, failure to investigate deviations from test methods, unclear quality control unit responsibilities and procedures, and lack of employee training on cGMP practices.

Most prescient is the last observation citing deficient operations to prevent contamination or mix-ups of products by having separate or defined areas for holding rejected components before disposition. The FDA investigator noted the absence of a report on a non-conforming material held in the “reject cage,” which was needed to explain the rejection and how the material was disposed. The year-old inspection report indicates that Trump administration officials and subsequently Biden staffers were aware of the serious quality control issues at the Emergent facility, even before the discovery that millions of doses of J&J vaccine doses were, in fact, discarded due to a mix-up with vaccine ingredient being produced for AstraZeneca. Despite the serious concerns raised by FDA and other analysts, pandemic preparedness officials continued to award ever- larger contracts to Emergent for vaccine ingredient production.

In an unprecedented move, J&J announced last week that it would assume full responsibility for manufacturing its vaccine drug substance at the Emergent facility.

 The company is shifting operations and quality experts to the site and “significantly increasing the number of manufacturing, quality and technical operations personnel” to work with Emergent staffers. At the same time, Emergent was instructed by the Biden administration to halt production of the AstraZeneca vaccine, offering to assist AZ in identifying other facilities to handle its production.

Even so, Emergent executives downplayed the situation, emphasizing in a press release that it was receiving an additional $23 million from the Biomedical Advanced Research and Development Authority (BARDA) to improve its manufacturing operations to expand J&J vaccine production. CEO Robert Kramer highlighted the company’s long-standing collaboration with the federal government in expanding vaccine production, and that the Bayview facility remains an Emergent property.

This followed an Emergent statement that its quality control checks had identified only one single noncompliant batch, and that there was nothing unusual in having to discard some material in the complex vaccine production process.

The continued award of ever-larger manufacturing contracts to Emergent reflects its political connections in Washington as well as still limited U.S. capacity for producing large quantities of critical vaccines. J&J has imported its vaccine substance from its plant in the Netherlands and also signed an agreement for Merck to produce its vaccine and has expanded its collaboration with Catalent to ramp up production of its vaccine at its Bloomington, Ind. facility.

One result of “uneven” production from the Emergent Baltimore plant is that federal health authorities now indicate that J&J will provide less than a million vaccine doses in the coming weeks, 85% less than the expected five million doses a week.

https://www.fda.gov/media/147437/download

https://www.jnj.com/johnson-johnson-statement-on-u-s-covid-19-vaccine-manufacturing-updated

https://investors.emergentbiosolutions.com/news-releases/news-release-details/company-statement-emergent-biosolutions-statement-status-current

A picture of wide manufacturing violations appears in a recently disclosed FDA form 483 report on a site visit to Emergent’s Baltimore vaccine plant.

FDA Inspectors Cited Critical Problems at Emergent Facility

Following several months of negotiations involving regulators, industry and patient and consumer advocates, updated programs supporting the development and review of prescription medicines are being readied for approval by federal agencies and ultimately by Congress. The fee programs so critical to FDA operations for regulating drugs and other medical products must be renewed every five years, this time in 2022, and a range of entities need to sign off on various changes and updates to the programs. The negotiators are finalizing “commitment letters” that spell out what industry will pay and what policies and programs FDA will implement with those funds, to be referenced in implementing legislation.

Although it may seem that there’s plenty of time to finalize new user fee packages and to gain legislative approval before the current authority expires in Sept. 2022, there is pressure to move the proposal forward in the next few months to meet this deadline. The discussions involve separate user fee packages for drugs and biologics (PDUFA VII), generic drugs (GDUFA III), and biosimilars (BSUFA III). The legislators usually look to authorize the new fee packages as part of a broader bill enacting a host of projects and requirements for drug regulation, development and marketing, possibly including an updated version of the 21st Century Cures Act with provisions governing clinical research and data disclosure.

FDA kicked off the fee negotiating process for renewing the Prescription Drug User Fee Act (PDUFA VII) at a public meeting in July 2020. Negotiations have involved subgroups for manufacturing and inspections, digital health and informatics, finance, regulatory decision tools, and pre- and post-market review.

 A main topic has been the need to increase fee revenues allotted to the Center for Biologics Evaluation and Research (CBER) to provide additional support and resources to oversee a fast-expanding volume of applications for cell and gene therapies, including expedited development of regenerative medicine therapies. And industry continues to press FDA for progress in hiring and retention of a “world-class technical and scientific staff.”

PDUFA agreements direct the Center for Drug Evaluation and Research (CDER) to utilize fee revenues to streamline and manage multiple meetings with sponsors and to advance rare disease therapies and use of real-world evidence, model-informed drug development, and complex clinical trial designs. A new program may test broader use of “splitreal-time” review of certain innovative drugs.

The program also will continue support for innovative manufacturing technologies and efforts to address more clearly CMC (chemistry, manufacturing and controls) issues during development. There is agreement on enhancing oversight of postmarket drug safety and Risk Evaluation and Mitigation Strategies, and to utilize more broadly analytics from FDA’s Sentinel Initiative. An important goal is to further implement digital health technologies and bioinformation to streamline drug development and review.

All these initiatives will continue to boost industry fees. Drug application fees are slated to reach $4 million over the five years of the program ending in 2027, up from nearly $3 million today. This increase reflects the steady rise in the portion of FDA operations funded by industry user fees, which now account for about half of the agency’s budget for regulating drugs and biologics. While some observers complain that this arrangement gives industry too much influence over FDA decisions, Congress seems fairly content with the financial arrangement. And FDA officials maintain that increased reliance on fee revenues does not sway approval decisions.

Negotiations also are winding up for reauthorization of the Generic Drug User Fee Amendments (GDUFA III), which also was launched at a July 2020 public meeting. After making notable changes in fee categories and rates in 2017 revisions, current discussions have focused on plant inspection processes, drug master file use, application review goal dates, and enhancements to the inactive ingredient database.

 Main innovations aim to facilitate the review of complex generics, of the continued rise in supplements seeking post-approval changes, and for controlled correspondence. These and other changes aim to achieve more application approvals in the first review cycle, possibly by assessing facility status earlier in the review process. 

Similarly, FDA is considering revisions to the Biosimilar User Fee Act (BsUFA III) to further streamline the approval process and facilitate patient access to less costly biotech therapies. That discussion began with a public meeting in November 2020, where FDA officials presented their goals for updating the program, acknowledging the need for additional guidance on interchangeable biosimilars.

 FDA seeks to better manage meetings with sponsors, who want advisory sessions to begin earlier in product development. Manufacturers emphasized the importance of building a capable staff to manage this program, along with upgrading information technology supporting BsUFA. Consumer advocates would like FDA to address how reimbursement and misinformation stymies biosimilar marketing, but that may lie outside the user fee agreement.

And, separately, FDA is launching its new user fee program for over-the-counter drugs, after agreeing to drop levies on manufacturers that jumped in to produce hand sanitizer to combat the spread of COVID-19. FDA included fees for such firms in its December 2020 publication of policies governing the new OTC monograph drug user fee program (OMFUA), which was authorized by Congress in March 2020 as part of broader pandemic legislation. But an outcry from firms such as liquor distillers forced FDA to drop the fees for companies that only produce hand sanitizer in response to the public health emergency. A new fee schedule was published March 26, 2021 with revised fees for facilities and for OTC products for the current year.

https://www.fda.gov/industry/prescription-drug-user-fee-amendments/pdufa-vii-fiscal-years-2023-2027

https://www.fda.gov/drugs/development-approval-process-drugs/gdufa-iii-reauthorization-negotiation-sessions?utm_medium=email&utm_source=govdelivery

https://www.fda.gov/industry/biosimilar-user-fee-amendments/public-meeting-reauthorization-biosimilar-user-fee-act-bsufa-11192020-11192020

https://www.fda.gov/news-events/fda-brief/fda-brief-fda-republishing-fee-rates-under-over-counter-monograph-drug-user-fee-program

Updated programs supporting the development and review of prescription medicines are being readied for approval by federal agencies and ultimately by Congress

FDA, Industry Move to Finalize New Drug User Fee Programs

Clinical trials are an essential part of getting new treatments from lab to patient but during the COVID-19 pandemic, trials for treatments outside of COVID-19 had been heavily disrupted. However, in light of all this immediate disruption there have been claims that the swift action taken to produce a vaccine and the adoption of technology could set a positive precedent for the future of clinical trial research.

Below, we focus on the implementation of clinical trials in Europe. We address the issues affecting ongoing trials; including data management, compliance with protocols, measures to be taken by sponsors, and the recommendations and regulations imposed by the authorities. We also look at the challenges surrounding the opening of new trials, including trials for COVID-19 treatments and vaccines.

Impact of COVID-19 on ongoing clinical trials

Some of the problems encountered in ongoing clinical trials due to COVID-19 include:

increased difficulties in patient recruitment,

tensions between stakeholders concerning payment (including CRO/sponsor),

management of participating patients who test positive for COVID-19,

compliance with local government regulations related to COVID-19 (e.g., protective measures), and

interruptions or terminations of clinical trials.

The response from authorities concerning ongoing clinical trials has been to issue specific recommendations at both the European and national level, with the goal of providing clarity and harmonization. Regulatory flexibility has been increased although this is not meant to be continued permanently. The driving principle when adjusting the conduct of clinical trials is the implementation of a proportionate response, based on a risk-benefit assessment, where patient safety is always the first priority.

The pandemic has also led to the increased use of digital technologies, such as virtual inspections, which can be used to meet reporting requirements. However, there is no certainty as to whether these elements will stay. In fact, many regulators have said they will be returning to usual protocol.

Guidance issued by the European Medicines Agency (EMA), the Heads of Medicines Agency (HMA) and the European Commission

has allowed sponsors to take relaxed measures to adapt the conduct of ongoing clinical trials to overcome Coronavirus-related challenges without always having to notify or request authorization from competent authorities.

As an overarching principle, sponsors should always strive to ensure compliance with the protocol to the highest level possible when setting up exceptional measures.

What about new clinical trials in the context of the current pandemic?

Under European guidance, it is still possible to open new clinical trials. However, these new trials should only be initiated if the sponsor has deemed it feasible, and if there is a need for the trial to be completed as soon as possible. Additional risks related to COVID-19 must of course be taken into account for participating patients (benefit/risk section of the protocol)

Most competent authorities, in line with European guidance, have announced they would give priority to trials on treatment or prevention of COVID-19, or on serious diseases without satisfactory treatment options (unmet medical needs). It is true for example in France where the Ministry of Health has put pressure on Ethics Committees to deal quickly with COVID-19 trials.

In order to facilitate the launch of clinical trials relating to COVID-19, authorities have allowed enhanced communication with them, as well as expedited authorization procedures for initial assessment of authorization requests. Regulatory flexibility with regards the collection of patients' consent has also been allowed (e.g., oral consent with a witness, electronic signature, etc.).

The future of clinical trials across Europe

Progress is being made to restart clinical trials that were paused or delayed due to the pandemic. Most of the issues that caused delays to trials were due to patient enrolment issues and availability of test sites, due to having to prioritize the pandemic. However, for those trials that were able to continue, we saw how technology was revolutionary in allowing them to progress. The use of digital technology, such as wearables, virtual inspections and apps meant that players involved from sponsors and clinicians to regulators were forced to embrace technology.

 But will this become the norm? Regulators across Europe have announced that they hope to return to 

 from mid to late 2021 and also the Clinical Trial Regulation issued in 2014 is about to be enforced, despite the fact that it does not take into account the possibility of the brand-new IT tools in the context of clinical trials, in particular during pandemics

Thus, it shows that there will still be a journey ahead for pharmaceutical companies and other businesses involved in the process to get technology firmly embedded into the clinical trials process.

is an Associate of Life Sciences; both of Fieldfisher

EMA/HMA/EC, Guidance on the management of clinical trials during the Covid-19 (coronavirus) pandemic, last updated on 4 February 2021; EMA, Points to consider on implications of coronavirus disease (COVID-19) on methodological aspects of ongoing clinical trials, 26 June 2020.

https://www.iconplc.com/insights/blog/2020/08/05/post-pandemic-clinical-tr/

A look at the issues in Europe affecting ongoing trials including data management, measures being taken by sponsors, and more.

COVID-19: A New Era for Clinical Trial Research in Europe?

For many pharmaceutical organizations, decentralized clinical trials (DCTs) have quickly become a strategic priority, one that is expected to endure long after the COVID-19 pandemic ends. Once viewed as nice-to-have pilot projects, DCTs—trials conducted remotely or through local healthcare providers to improve the patient experience—arrived when Pfizer launched a virtual trial

 in 2011. While drug-based interventional DCTs only experienced a 7% CAGR between 2014 and 2019, they jumped to 77% between the second halves of 2019 and 2020 after the onset of COVID-19, according to ZS’s DCT database, referencing data from more than 1,000 trials.

Decentralized trials offer a patient-centric approach, addressing various patient needs that often go unmet in traditional designs. DCTs benefit sponsors by accelerating clinical development, enabling more representative patient access and developing a stronger evidence package than traditional trials. Yet, DCT implementation doesn’t have to be all or nothing; trial decentralization can be viewed as a spectrum, with most DCTs combining in-person and remote activities to make clinical trial participation easier for patients. But one thing is certain: COVID-19 has shifted the balance of tradeoffs for the pharma industry, and despite operational challenges that may arise during decentralization, the benefits outweigh the investment. In the era of disruption, now is the time to take the risk.

Through our work with pharma clients, we have observed four primary drivers that suggest DCTs are here to stay, even in a post-pandemic society:

 As a result of COVID-19, regulatory agencies including the FDA and European Medicines Agency have clarified their guidance on key decentralized modalities, such as telemedicine across state lines, direct-to-patient investigational product shipments and more. The FDA’s new Digital Health Center of Excellence

 to advance the use of DCTs indicates a smoother regulatory landscape through 2021 and beyond.

 Evolving technology—fueled by record-level venture capital investments

 in digital health—is enabling trial decentralization. Furthermore, horizontal and vertical integration among technology solution providers is creating more end-to-end technology solutions to deliver DCTs.

Pharma investment and capability development:

 Pharma is creating focused DCT capability groups with dedicated leadership and investing in processes and technology to operate DCTs at scale.

 Patients’ increasing use of digital healthcare, such as telemedicine and digital pharmacies, have enhanced expectations of their clinical trial experience, which we don’t expect to revert moving forward.

DCTs offer patients a more streamlined experience, reduce the burden of time-consuming in-person visits and enable wider patient access, especially in rural and underrepresented communities.

Like patients, sponsors also benefit from DCTs. As of 2016, 85% of trials failed to finish on time, financially impacting sponsors by up to $8 million per day, according to Pharmafile.

Decentralization can reduce these hurdles for sponsors by speeding up recruitment, increasing compliance and reducing drop-out rates. Additionally, collecting high-quality clinical and real-world data through DCTs can build stronger evidence packages and better reflect the value created for patients. Sponsors can use novel digital endpoints to generate differentiated label claims through DCTs as well.

Still, decentralization—like most new innovations—adds complexity to the clinical trial process. It requires stakeholders to devote time and resources before the benefits can be realized. Sponsors must learn how to effectively and flexibly implement DCTs, as well as support patients and sites on their respective learning journeys. From a regulatory perspective, DCTs are still nascent and without consistent standards, meaning sponsors may have to initially focus on select priority markets. Lastly, while initial proof points to DCTs providing value are emerging—especially for increasing recruitment speed

—more work is needed to understand their impact. Sponsors should consider ways to benchmark DCT performance versus traditional trials to understand and capture the value of DCT.

Sponsors considering DCTs must root their approach in a deep understanding of patient needs and experiences. Designing approaches and solutions that reduce the patient burden and provide the right behavioral incentives for patients will lead to better patient engagement and improved trial performance. It’s also important to recognize that sites will remain key to patient experiences: As sponsors develop DCT solutions, providing options that are adaptive to site workflow and capability will be critical.

It’s easy to assume solely decentralizing trials and introducing new technology will improve the patient experience, but this isn’t necessarily true. In fact, a multitude of disjointed study apps may even increase the burden, while home nurse visits may be more stressful than visiting a community center. Sponsors must first listen to patients and understand their needs, and then create a more personalized trial experience that meets those needs. It’s important for sponsors with diverse clinical development pipelines to tailor this exercise to specific trial patient populations, endpoints and intervention modalities. Furthermore, within each trial, sponsors should strive to enable optionality for individual patients based on their engagement preferences.

Beyond the approach itself, sponsors also must ensure that decentralization options are considered early in clinical development planning, allowing sufficient time for patient research, endpoint validation and vendor selection activities. If decentralization is an operational afterthought, the process will be more difficult to incorporate and, moreover, the extent to which the trial can be decentralized will be constrained by traditional, site-centered protocols. Shifting this thinking upstream will enable study teams to assess how decentralization can improve the patient experience.

Developing the digital capabilities needed for DCTs is also critical. There are new types of data collected in DCTs and oftentimes new insight generation opportunities from continuous data collection. Digital solutions must enable a unified user experience and one source of truth for data integration. Additionally, while the infrastructure can be gradually built—with numerous digital solutions and providers in the market—sponsors must have an enterprise architecture blueprint to guide the development to enable connectivity and optionality. Developing the DCT infrastructure without a plan would lead to future challenges with technological integration and agility.

Finally, since DCTs are cross-functional in nature, they require collaboration across sponsors’ trial design, trial operations, regulatory, digital health and innovation functions. These teams must overcome functional silos and establish new ways of working to successfully drive DCTs. Sponsors must also establish consistent processes to facilitate scaling DCTs from a proof-of-concept to an organizational capability. One key step here is organizational knowledge sharing: applying learnings rapidly and efficiently across study teams.

For sponsors wondering where to begin or focus DCT efforts across a portfolio, a combination of their business needs and feasibility of decentralization between therapy areas can act as a guide. Some therapy areas are a better fit for decentralized trials, based on factors such as the investigational medicinal product’s administration route (e.g., oral vs. infusion), type of assessment procedure (e.g., non-invasive vs. invasive biopsy) and whether the condition being studied is chronic or acute. Although ZS’s DCT database found cardiovascular, neurology and diabetes as the most common therapy areas for DCTs, they can also be applied in other therapeutic areas. In fact, oncology and infectious diseases make up 5% and 4%, respectively, of all DCTs historically. That said, for many therapy areas, full decentralization may not be feasible or right for patients; instead, identifying specific procedures that can be decentralized or combined into a single visit can drive valuable improvements to the patient experience.

As DCTs become more common, they will raise the bar for patient experience and patient data collection. They offer an opportunity to fundamentally change how we perform clinical research. As pharma companies embark on this journey, they can’t do it alone. The good news is that patients, investigators, sites and regulators are prepared to make the change and unlock the value of moving clinical trials closer to patients.

https://www.pfizer.com/news/press-release/press-release-detail/pfizer_conducts_first_virtual_clinical_trial_allowing_patients_to_participate_regardless_of_geography

https://www.fda.gov/news-events/press-announcements/fda-launches-digital-health-center-excellence

https://www.appropriations.senate.gov/imo/media/doc/AGRept.pdf

https://rockhealth.com/reports/2020-market-insights-report-chasing-a-new-equilibrium/

http://www.pharmafile.com/news/511225/clinical-trials-and-their-patients-rising-costs-and-how-stem-loss

https://www.sciencedirect.com/science/article/pii/S2451865420300740

https://www.sciencedirect.com/science/article/pii/S2451865418300358

Assessing the opportunities, considerations and implications of decentralized trials—and why they’re here to stay

Why Decentralized Clinical Trials Are the Way of the Future

 this week, presenting a host of strategies to strengthen the nation’s transportation, housing, jobs, education, and home healthcare, while also advancing research and science in multiple industries. The aim is to appeal broadly to Americans in all regions and across the political spectrum with the promise of better lives post-pandemic. With a cost of $2.3 trillion over eight years, and possibly up to $4 trillion over the next decade, the administration seeks to fund much of the tab with the hefty boost in the corporate tax rate over 15 years and to increase taxes on companies’ foreign earnings, changes that notably would affect biopharma companies.

The largest investments provide funds to modernize transportation infrastructure, expand affordable housing, bolster utilities, and expand home and community-based care for the elderly and disabled. Within these initiatives are multiple programs related to R&D and technology, such as funds for electric vehicle development, to expand high-speed broadband and to improve the power grid. An emphasis on boosting support for research and development aims to make American industry more competitive with China and other advanced nations, while also creating more high-wage jobs and maintaining American global leadership in manufacturing and economic development.

A number of innovation and R&D programs fall under the $280 billion allotted for jobs creation and expanded research infrastructure to support the “technologies of the future” important to American competitiveness “on the global stage.” There’s $50 billion for the National Science Foundation to collaborate and build on high-tech programs in fields like biotechnology, semiconductors, advanced computing, and advanced energy technologies. There’s another $35 billion to address the climate crisis and advance clean energy technology. Efforts to retool and revitalize American manufacturing include a new office to monitor domestic industrial capacity and support production of critical goods such as semiconductors. And there’s more than $50 billion available for investment in US manufacturers and a plan for “regional innovation hubs” to spread new opportunities across the country.

Advancing American manufacturing also supports activities to “protect Americans from future pandemics.” That initiative provides $30 billion to invest in medical countermeasures manufacturing, shore up the strategic national stockpile, accelerate the timeline for R&D and testing of new therapeutics, and train personnel for epidemic and pandemic response. One notable proposal is to accelerate the nation’s ability to respond quickly to a medical emergency by developing prototype vaccines through Phase I and II trials. Biopharma manufacturing also would gain from investment in new technologies for rapid scale up of vaccine production, in expanded emergency production capacity and enhanced US infrastructure for biopreparedness, and in greater production onshore of active pharmaceutical ingredients. 

The main source of funding for the massive program is to boost the corporate tax rate to 28%, up from the current 21% that was part of a major Trump administration tax cut. That’s still lower than the 35% corporate tax rate before the change in 2017, but still projected to provide up to $3 trillion to pay for the jobs and infrastructure package. President Biden explained that the plan aims to end “the race to the bottom on corporate tax rates” around the world. And reformers maintain that corporations actually pay only about a 16% tax rate, largely due to incentives for shifting production and jobs overseas.

Biden aims to fix that by revising current policies that exempt from US taxes most foreign assets and higher taxes paid overseas. A range of policy changes would discourage foreign based companies from shifting profits from US operations to avoid taxes, while also discouraging foreign takeovers of US firms. The global operations of most biopharma companies means that any changes in the international tax arena will have a significant impact on industry operations and business decisions.

Republicans already are loudly opposing the tax changes, and the package is slated to undergo significant changes to gain sufficient support for enactment.

New American Jobs Plan set to be funded by corporate taxes will affect biopharma industry.

Biden Infrastructure Package Would Boost R&D–and Corporate Taxes

Komodo Health has announced a new client engagement with Janssen. Janssen Research & Development, LLC (Janssen) will be using Komodo's real-world data and AI software. Janssen plans to use Komodo's services aiming to improve feasibility, site selection and patient recruitment for clinical trials.

The partnership will focus on offering real-time visibility into patient volumes, the ability to identify trial sites, increasing target recruitment, understanding patient cohorts before launch and reaching diverse patient populations. Janssen will utilize Komodo’s Healthcare Map of 325M U.S. de-identified patient journeys, the largest of any health IT company, and Komodo's industry solutions Prism, Sentinel, and Pulse.

Komodo Health Announces Clinical Development Agreement with Janssen

Reify Health and SubjectWell have announced an innovative partnership aimed at restructuring the workflow of research sites to manage patient referrals from sponsor-driven recruitment campaigns. With this pairing, referrals from SubjectWell’s marketplace are delivered directly into Reify Health’s StudyTeam, offering a common platform for sites and sponsors to manage pre-screening, enrollment, and reporting for clinical trials. 

With this partnership, SubjectWell’s qualified referrals are delivered via Reify Health’s Recruitment Partner Interface (RPI) directly into StudyTeam. Sites are then able to update, manage, and report on the entire enrollment funnel from a single, secure platform.

Reify Health and SubjectWell Partner to Boost Patient Recruitment

Reltio, a cloud-native master data management software provider, has introduced Reltio Enterprise 360 Site Intelligence. The software centralizes detailed data about study sites and principal investigators. Features include the ability to search all current, complete, accurate information available on healthcare organizations/ study sites, and HCP/PIs; evaluate and rate study sites and principal investigators using artificial intelligence (AI), quantitative metrics, and qualitative criteria, and manage consent, contracting, and collaboration workflows.

Reltio Launches Enterprise 360 Site Intelligence 

The pandemic has dramatically impacted many aspects of clinical trials, ranging from operations to recruitment/retention to clinical trial public perception and technology. In this interview, Lan Fong, Founder and Principal Consultant at Avery Biopharma Consulting and Venture Partner at Congress Avenue Ventures, discusses her perspective on how the pandemic is shifting the clinical trials industry.

MA: How has the pandemic impacted COVID versus non-COVID trials?

 The impact on non-COVID trials was prominent because they have been deprioritized within the clinical trials industry, and the priority has shifted towards COVID-related trials. For non-COVID-related trials at sites, it has been challenging to execute protocols with traditional methodologies in an environment experiencing many disruptions. For example, sites had to have COVID testing measures and protocols in place, limiting patient visit capacity and volume at sites. Sites have had to re-adjust their staff schedules and face the need to ensure enough social distancing procedures if a patient comes in for less than 30 minutes for a quick blood draw or a short assessment. There also have been impacts on patient retention; for instance, if a trial is being executed in a large city, such as Boston or New York, most patients who commonly use public transit did not want to continue using that mode of transportation, henceforth, missing study visits or dropping out of the study altogether. As a result, there have been many COVID-related protocol deviations. Regulators have also issued new guidance around COVID-related disruptions to clinical trials, and as a result, CROs have defined a new category of COVID-related protocol deviations to capture them as a separate category in the clinical study report.

COVID-trials have been just as challenging to execute during the pandemic. COVID studies are pressed against aggressive timelines and face delays in site activation, manufacturing delays, and supply chain issues. There are also investigational product stability and logistical issues, such as monitoring expirations and ensuring stability at specific time points while operating in a tumultuous and rapidly changing environment, as there were shutdowns and delays in transportation. Even though clinical research centers prioritized COVID-related protocols, there were also site activation and IRB approval delays as much of the work had to shift to being conducted virtually when previously the sites, IRBs, and vendors were not necessarily set up to do so.

MA: How have the disruptions impacted minority group participation in clinical research?

 Recruiting minorities has always been incredibly challenging, even before COVID; reaching out to diverse groups has inherently been difficult because, despite FDA mandates for minority inclusion in clinical trials, there has always been less willingness for minorities to participate. Throwing in pandemic challenges has not only hurt overall study recruitment but, recruitment especially for minorities. For example, in larger cities, patients could quickly attend clinical trial visits via public transit. However, since few patients want to take public transit, they don’t have the means to afford different and less risky transportation options (i.e., Uber, Lyft). There has been a massive increase in recruitment and retention budgets from sponsors to support patient transportation and recruitment. It’s a tough decision for a sponsor because, sometimes, if sponsors have poor recruitment/retention strategies or are trying to learn how to recruit a particular patient population in their trial, they will not yield a good return on their recruitment/retention budget investment (ROI). Then you throw the challenges of being in a pandemic, and that decreases the ROI. So, sponsors have had to take a hard look at their enrollment projections and their statistical analysis plans to see if they can reduce the number of subjects needed to evaluate the study’s endpoints. Sponsors have also conducted early data cuts and interim analyses to see if they can uncover early signals.

MA: How has the pandemic impacted public perception of clinical trials?

 With the pandemic and the development of the COVID vaccines, I believe people have come to appreciate the need for clinical trials, and it has put this clinical research front and center. Before the pandemic, clinical trials were not something many people thought about, so I’m glad that it has raised awareness and emphasized the importance of clinical research and the need for clinical trials. Additionally, people now understand the importance of drug development and how badly we need to increase the speed at which new treatments are developed. Vaccine development has been accelerated, timelines have been shortened to address the critical need, and some traditional drug development barriers have been broken due to emergency protocols used to accelerate COVID vaccine development. It has also been a delicate balance between government and industry to establish trust and rigor in science; to successfully accelerate drug development without compromising subjects’ safety is challenging. Despite that, I believe people now understand what’s so essential about clinical trials and hopefully support those efforts.

MA: How many ways exist to execute decentralized clinical trials?

 The adoption of decentralized strategies has been discussed a lot, and I’ve seen two different ways of approaching it. First, many clinical trials center around major medical institutions that have been doing clinical research for a long time. I’ve seen integrated research organizations (IROs) making naive research sites into clinical trial sites by supplementing them with clinical research staffing, such as traveling CRAs and CRCs. The IROs implement their own SOPs at the sites and provide data entry, quality management, recruitment, and staff support to sites to operate studies and conduct visits at patients’ homes. Second, some companies are working on digital solutions, such as electronic consent, electronic clinical outcomes assessments (eCOAs), and electronic patient-reported outcomes (ePROs). Other solutions are moving away from paper and onto electronic modes of data collection, or eSource. Additionally, there is a sudden shift in patient support solutions for recruitment and retention. For example, some companies are providing transportation services or conducting mobile nursing/ phlebotomy. While these platforms have existed for some time, never has there been more need for more digital solutions that can integrate seamlessly with existing systems and databases. 

Another interesting trend I’ve seen includes integration strategies and capabilities into CROs, which have provided exposure and growth opportunities for companies specializing in decentralization. Some of these companies have acquired smaller companies that were building these capabilities and are in the process of integrating with their existing platforms. The goal is to make it a seamless and more familiar platform for sponsors to bring those capabilities onto their trials since the CROs have already vetted and conducted due diligence on these companies. However, the execution and actual integration can be challenging.

MA: What other areas from sponsors have arisen as a result of decentralization?

: Remote monitoring and risk-based monitoring have been hot topics for a long time. In the past few years, there’s been much debate around whether you need to do 100% source data verification or if the monitoring plan can be centered around critical variables. Sponsors and CROs also have to decide between on-site monitoring, remote monitoring, or partial remote monitoring, depending on the ease in which the CROs, vendors, and sites can use a fully secure, Part 11 compliant source data platform. Despite this topic being discussed for many years, many sponsors are hesitant to move away from 100% source data verification, and many vendors are not equipped to perform remote monitoring. While every piece of clinical trial data is essential, the necessity of source data verification on every data entry also depends on whether the case report forms were created thoughtfully and intentionally, without unnecessary data fields. While the industry has been slow to move away from traditional methods, it is now more critical than ever to adopt innovative solutions to execute clinical trials in modern times, especially amid a pandemic.

, MBA, MSF, is a thought leader and expert in the application of business analytics toward clinical trials, and Editorial Advisory Board member for and regular contributor to Applied Clinical Trials.

Lan Fong, Founder and Principal Consultant at Avery Biopharma Consulting and Venture Partner at Congress Avenue Ventures, discusses her perspective on how the pandemic is shifting the clinical trials industry.

Pandemic Impact on Trials, Public Perception, and Decentralization

Peter O'Donnell is a freelance journalist who specializes in European health affairs and is based in Brussels, Belgium.

Accusations that Europe is slow to take advantage of advances in healthcare are a commonplace–and the melancholy evidence often supports the contention. Even the officials and politicians at the head of the European Union, which exists on the basis of determined optimism about the potential of its 27 member countries working together, are obliged to recognize there are failings that should be remedied. The EU's "new pharmaceutical strategy for Europe," now being gradually implemented, acknowledges that "though Europe’s response has demonstrated strengths, existing vulnerabilities have been thrown into sharp focus" by recent health challenges. "A new EU approach is needed to ensure we have a strong, fair competitive and green industry that delivers for patients, and which draws on the potential of the digital transformation of health and care, driven by technological advances in fields such as artificial intelligence and computational modelling," it has announced grandiosely.

"There is a growing consensus that policies need to be rethought so as to stimulate innovation, in particular in areas of unmet needs, and for pharmaceutical innovation to be more patient-centered, health system oriented and take account of multi-disciplinary requirements, such as in long-term care settings," accepts the strategy. But on one of the promising areas of development, the role of advanced diagnostics, it has little to say: no more, in fact, than a passing reference in the context of the campaign to tackle antimicrobial resistance, where it identifies the need to "draft evidence-based guidance on existing and new diagnostics."

A new review from the industry grouping representing many of Europe's smaller research-intensive companies sets out the case more eloquently for advanced diagnostics. "Despite the many scientific breakthroughs across European academic centers and hospitals, the adoption of advanced diagnostics and technologies in the clinic and healthcare systems is not a foregone conclusion, and the rate of adoption in Europe is falling behind that of other regions globally," laments Eucope.

"Developing an advanced diagnostics ecosystem in Europe: a proposal for change"

, the central argument of the review is that the value of advanced diagnostics is not sufficiently recognized, and as a consequence clinical practices are not keeping up with the accelerating cycle of innovation in novel diagnostics. In addition, in Europe it takes too long to bring advanced diagnostics to market, in processes that are "complex and fraught with uncertainty." To make matters worse, "essential clinical supporting infrastructure is lacking in Europe"–in terms of an appropriately trained workforce, data sharing and data storage infrastructure capabilities, or digital health recording within healthcare systems.

Among the downsides of missed opportunities in Europe, the review notes that advanced diagnostics have enabled the development of novel trial designs, which permit the testing of patients with multiple tumor histologies and/or tumor molecular aberrations–through master protocols comprising both umbrella trials and basket trials fall. Health technology assessment bodies in Europe are also slow to accept the benefits of advanced diagnostics or to fully appreciate their role, says the review. "HTA bodies should acknowledge the challenges of implementing traditional study designs for advanced diagnostics–as they are not fit for these technologies–and accept novel study designs (e.g. basket trials, umbrella trials, and retrospective studies) that demonstrate the clinical utility of advanced diagnostics and prioritize timely patient access."

"With a rapidly growing number of advanced diagnostic tests available to patients, serious consideration needs to be given to ensure a robust and healthy diagnostics ecosystem in Europe that allows innovators to thrive," the review argues, calling for infrastructure to support sequencing on a large scale, wider use of basic supporting digital infrastructure such as electronic health records across European healthcare systems, insufficient bioinformatics infrastructure and computational tools needed to interpret sequencing results, including data storage and periodic reevaluation of incidental findings that may attain clinical relevance over time.

One of the factors identified as hampering realization of the potential in Europe is that genomic tests have been performed only in public laboratories within hospitals, limited to the research setting, and largely focused on oncology. It is only recently that decision makers have begun considering commercial tests.

The review suggests that private–public partnerships can offer a route to faster adoption of advanced diagnostics in the clinical setting. It cites the case of Genomics England, with its interface and in-depth collaboration between NHS commissioning and technology companies. It also salutes the partnership agreed between the precision oncology company Guardant Health and the Vall d’Hebron Institute of Oncology in Barcelona, Spain, which is establishing the first Guardant-based liquid biopsy testing service in Europe. The opening of this service is expected to significantly increase the number of cancer patients identified as eligible for clinical trials based on available precision medicines and help accelerate research and development of the next wave of cancer therapeutics, claims the review.

Eucope, the industry association behind the review, with a heavy investment among its member companies in products that are designed to serve the growing possibilities of personalized treatment, concludes with a call for greater integration: "A coherent strategy for personalized medicines should articulate the approach to disease profiling and consider the integration of advanced diagnostics into clinical practice," it says.

Rate of adoption in Europe for advanced diagnostics is falling behind that of others' globally.

Another European Gap that Needs to be Filled: Advanced Diagnostics

Patients as Partners — 4/7-4/9 *VIRTUAL*

A 3-Day Festival of Ideas from Patients, Pharma and FDA on How Patient Engagement and Involvement Gets Done

https://theconferenceforum.org/conferences/patients-as-partners/overview/

Events

Patients as Partners — 4/7-4/9

Cytel announces that its clinical trial design and statistical software solution Solara is now available for general release to the pharma industry. Solara is available in two formats: standalone SaaS product, and as part of a consultancy solution. The consultancy solution is directed to smaller biopharma companies, which may lack an in-house biostatistician to lead the clinical trial design process.

Cytel Opens Solara to General Release & Launches New Trial Optimization Solution for Biotechs

Many sponsors have discussed the challenges of operating clinical trials during the pandemic, but there hasn’t been much discussion around the patient experience, and how patient feedback can help sponsors better design their studies to meet patients’ evolving needs. In this interview, Valerie Powell, Vice President of Research Services at HealthiVibe, discusses her experience with obtaining patient insights and helping her clients apply them to trial design and conduct in a pandemic.

Moe Alsumidaie: What challenges have you seen Sponsors and sites face as they continue to recruit and retain patients in global clinical trials during this pandemic?

 The challenges fall into at least three categories. First of all, many study subjects are fearful and unwilling to attend in-person trial visits, so they either aren’t enrolling or are dropping out. Secondly, patients and caregivers are focused on daily living issues during the pandemic, not on medical care, including trial participation. Lastly, site staff burden has increased sharply. Hospital-based sites are dealing with COVID surges, with some PIs being called into service to help and study staff adapting to changes in their work environments, working from home or juggling study requirements with cumbersome on-site pandemic precautions. The studies themselves are taxing staff, as they deal with delays, cancellations, and protocol changes to accommodate the shift to virtual visits, and the significant burden related to remote monitoring visits.

MA: How has patient willingness to enroll in clinical trials changed? What are you hearing from patients now?

 Clinical trial participation can be scary—and now, we have the added fears of contracting the virus at site visits and receiving an experimental treatment during a pandemic. While this fear can decrease willingness to participate, changes to trial designs—telemedicine and home health visits—can make participation more attractive if done in ways that resonate with patients and caregivers. A shift that I expect could have an even more significant impact is the increased familiarity with clinical trials (due to the COVID-19 vaccine trials being in the news). This familiarity can breed acceptance.

First, patients are hearing and learning more about trials. Now laypeople who have never considered a trial know more about the trial process, which builds comfort and helps break down misconceptions. Second, because of the worldwide focus on the need for a vaccine, the public sees the value of clinical trials in a new and real way. Finally, the emphasis on involving diverse populations in the vaccine trials could help break down some barriers in enrolling diverse populations in our trials. I’m hopeful that we will see prolonged support of clinical trials and reduced stigma around participation – it could be a small silver lining to this pandemic experience.

MA: What are patients requesting to help make them feel safe participating in clinical trials during these challenging times?

 To feel safe and be willing to participate, what patients want is not always what the sponsors expect. It varies by therapeutic area, the severity of the disease, demographics, and other factors. For some conditions, patients are very enthusiastic about home visits and telemedicine; in other conditions, such as severe chronic illnesses and cancers, we see patients asking for reassurance that they’ll still see the study doctor regularly – that’s what will make them feel safe, despite the pandemic. Some patient populations are also very enthusiastic about using devices – vital sign monitors, sleep monitors, and adherence apps, to name a few – while others are resistant due to discomfort with the technology or privacy concerns. Ultimately, each trial and each patient population are different. Designing a trial and testing technologies with the targeted patients remains the best way to ensure you’ll have robust enrollment and retain trial subjects.

MA: How have sponsors and sites pivoted to meet the needs of patients during the COVID pandemic?

 As mentioned, many sponsors have shifted to home health nurse visits and the use of study-support technologies. The pandemic has also caused us to finally take a close look at the burden of study participation—frequent and extended study visits, multiple procedures—and find ways to scale back study requirements to focus on need-to-know data, not nice-to-know data. Many sites have taken a hands-on approach to explain study requirements, reassuring patients, and genuinely coaching study subjects throughout the process. The most successful sponsors have engaged both sites and patients to learn what will work for them before implementing any changes.

MA: What still needs to be done to ensure successful, patient-centric clinical trials during COVID times and beyond?

 Engaging with patients to co-design clinical trials is still seen as an optional activity by most sponsors. Even sponsors who engage patients regularly often do so when it’s too late and a protocol cannot be changed. I’m hopeful the challenges of operating studies during the pandemic have begun to cause a shift—that sponsors see the benefit of engaging patients early and often. Some of our clients give patients and caregivers a seat at the table from very early in the development process, before portfolio entry, to identify meaningful endpoints and effectively shape clinical development programs. For those of us who’ve dedicated our careers to this work, that’s a very good sign. It is true patient engagement.

Valerie Powell, Vice President of Research Services at HealthiVibe, discusses her experience with obtaining patient insights and helping her clients apply them to trial design and conduct in a pandemic. 

Let’s Not Forget the Patient Perspective in Clinical Trials

The drug development enterprise is undergoing a transformation of great magnitude driven by the cross-industry digitization of product development and the rise of Big Data. In 2020, we witnessed the acceleration of this evolution due to challenges created by the COVID-19 global health crisis. Out of necessity, the drug development sector is now embracing technologies and digital methods that were previously not as widely used. The sector is being forced to adapt to a new normal characterized by the maturation of data management solutions, an expanded number of data sources, and the unprecedented increase in data volume.

The sources of information and data management solutions that support drug development have evolved considerably over the past 40 years. During the 1980s and 1990s, clinical development focused on the gathering of small scientific data sets that yielded limited insights. In comparison, during the first two decades of this century, the enterprise gathered not only scientific data, but also collected operational data to improve the product development process. The sector’s use of data continues to evolve into what is now a nascent patient-centered data driven system that requires flexible models supported by augmented analytics and machine learning. These methods are expected to support an increasingly decentralized, predictive, and personalized drug development process.

The continued contribution of the drug development community toward improving the quality of lives of patients, researchers, and the public at large, is and will continue to be highly dependent upon the careful execution of strategies to make vast amounts of data meaningful and usable. This is achievable by pairing data with powerful analytics and then using those insights to develop safe and effective processes and products.

Although the drug development enterprise is undergoing major transformation, literature about what the sector should do to support and prepare its workforce for these changes is scant.

What follows is a discussion of original research conducted by the Tufts Center for the Study of Drug Development (Tufts CSDD) to address workforce development in the era of digitization. The research is primarily based on an in-depth discussion with thought leaders and senior executives. Tufts CSDD identified recurring themes for discussion in articles in academic journals and the trade press between 2015 and 2019. Discussion topics included: (1) challenges and opportunities caused by the sector’s digital transformation, (2) skills and competencies of future drug development professionals, (3) new roles that are expected to emerge within drug development, (4) changes in talent recruitment and retention practices, and (5) the reshaping of corporate mindsets and cultures to become digitally proficient organizations.

In December of 2019, Tufts CSDD moderated an in-person discussion in New York City among 60 executives from pharmaceutical and biotechnology companies, technology solutions companies, professional trade organizations, and academia. Their functional expertise included clinical research, clinical operations, data management, regulatory affairs, human resources, digital technology, and R&D strategy.

Participants first discussed how digital transformation and patient engagement have changed the nature of clinical and operational data. Industry now prioritizes the collection of patient-centered data in addition to scientific and operational data. This new emphasis on patient-engaged science is facilitating an open, customized, and predictive framework for drug discovery and development. But patient-engaged science also leads to even larger data volume; this requires wide-spread knowledge of advanced analytics, cross-platform accessibility, continuous and flexible learning, and effective leadership within and between organizations and regulatory bodies.

Trends shaping the digital transformation of the drug development enterprise

The drug development sector is embracing technologies and digital methods that were previously not as widely used due to the COVID-19 global health crisis. 

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Applied Clinical Trials March 2021