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Applied Clinical Trials September 2021

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Events

Biotech Week Boston — 9/20-9/23

 presented data at the MDS Congress 2021 that predicted the placebo response in Parkinson's disease (PD) in a multi-center, multi-national clinical study. This approach–called 

–to mitigate the negative impact of the placebo response is an effective tool to increase study power of randomized clinical trials (RCTs), resulting in increased success rates and decreased clinical trial timelines and cost.

The predictive, multivariate Placebell model is based on patients' psychological traits and other factors associated with the placebo response (e.g., baseline PD intensity). For this study, 94 subjects with mild to moderate PD received placebo treatment for 3 months in a blinded administration. The placebo response was measured by: MDS-UPDRS part III (primary endpoint); MDS-UPDRS part I, II and IV; IGAC, PGAC; PDQ-39 (PD questionnaire); ESS (Epworth Sleep Scale); FSS (Fatigue Severity Scale). Tools4Patient's proprietary Multidimensional Psychological Questionnaire (MPsQ) was administered once (at baseline) and was used to assess various psychological traits related to the placebo response.

Read more about the presentation and model 

Articles

Tools4Patient Develops Predictive Model of the Placebo Response in Parkinson's Disease

In a move that revealed just how difficult it is to craft policies designed to curb industry pricing practices, Democrats failed to gain sufficient support this week to advance long-debated legislation to permit Medicare drug price negotiations. Three centrist Democrats opposed the measure in debate on Sept. 15, sufficient to block the bill from moving out of the key House Energy and Commerce Committee.

The vote put a temporary halt on efforts to approve the Democrats’ lead proposal to permit government negotiations of reimbursement for certain medicines covered by Medicare, a key provision to help pay for the $3.5 trillion budget package on a tight schedule for moving through Congress. Opposition from Reps. Kurt Schrader (D-Or), Scott Peters (D-Ca) and Kathleen Rice (D-NY) has sent party leaders back to the drawing boards in search of drug pricing reform provisions more acceptable to moderates in both the House and Senate. The spending bill may move forward without this provision from the House Ways & Means Committee, which continues to debate related health care spending provisions.

The three centrists offered a more modest drug pricing measure, which they claim would lower consumer out-of-pocket costs, while also maintaining incentives for private investment and innovation. A major change here would limit price negotiations to fewer medicines covered by Medicare Part B—only those drugs that lack competition or patent exclusivity. Such a revision would raise much less revenue and is adamantly opposed by progressive Democrats. They see it as caving in to Big Pharma and severely limiting savings for patients.

These developments on drug pricing legislation reflects the success of industry in raising fears that such measures will block patient access to needed medicines and dry up investment in important R&D on new therapies. PhRMA (Pharmaceutical Research and Manufacturers of America) is spending more than $1 million on TV ads as part of a massive lobbying and communications campaign emphasizing thepotential harm to patients seeking cures for deadly diseases. A PhRMA full-page ad in the Washington Post ran on Sept. 15 to highlight how Medicare price negotiations threatens “patient’s ability to get the medicines they need” and that one legislative proposal “would cut $1.5 trillion from innovative research companies” over 10 years.

 In countries with such price controls, claims the letter signed by all of PhRMA’s members, new cancer therapies and breakthroughs are “either delayed or not available at all.”

Smaller biotech firms and financial investors similarly emphasize that drug price negotiations won’t just harm Big Pharma, but will dry up investment in vital R&D operations. A letter to the White House and to House and Senate leaders on Sept. 8, which was signed by 400 small biotech company heads and leading investment companies in the field, emphasized that “draconian” price negotiation legislation “would immediately halt private funding of drug discovery and development.”

Meanwhile, legislators backing the Medicare price negotiation plan counter-charge that industry is exploiting consumer fears and using its vast resources to block any fair and reasonable cost-cutting efforts. But opposition to the Democrats’ main price cutting proposal already is stronger in the Senate, where many key members have voiced concerns about government interference in private sector investment. The situation sets the stage for intense negotiations and Congressional infighting in the coming weeks.

https://catalyst.phrma.org/americas-biopharmaceutical-companies-to-congress-government-negotiation-is-the-wrong-approach

https://nopatientleftbehind.docsend.com/view/a6bxibzxysaeggm3?mkt_tok=NDkwLUVIWi05OTkAAAF_bv9xwWlCfMU6-GDrBC04DroGiq0E6LkCXRRXVM5gpeUxbbDlenS_eRSMkmpN7GABcT_aORMtrBCmuadp_SMHsP8iMIufGdRevi5t-hcrKZc4

Democrats failed to gain sufficient support this week to advance long-debated legislation to permit Medicare drug price negotiations.

Pharma Blocks Democrats’ Drug Pricing Plan

Medications don’t work for patients who don’t take them – C. Everest Koop

The above statement may sound as a no-brainer. Unfortunately, medication adherence continues to remain one of the biggest healthcare challenges not only for commercially available treatments but also for Investigational Medicinal Products (IMPs) in clinical trials. Research has shown that, across all therapeutic areas, adherence rates drop by 40% within a year of clinical trial commencement.

Incorrect medication intake can pose multiple risks such as sub-optimal therapeutic effects, higher chance of side effects, increased use of medical resources, or even incorrect assessments and conclusions for the Investigational Medicinal Products with an early termination of potential life-saving compounds.

Physicians too have limited to no real-time insights into the activities at home, and hence incorrect intake is picked up only during the next interaction or a few even go unnoticed. This can have a significant impact on the safety and health of patients.

While patient medication adherence has its set of challenges, life sciences organizations worldwide too are grappling with a highly complex and convoluted medication process that involves multiple stakeholders, isolated systems, manual, and often paper-based processes resulting in a labor-intensive, costly, and error-prone process. To give an example, 20-25% of on-site monitoring activities are linked with IMP handlings including verifying documents related to kit receipt, dispensing, return, destruction, manual pill counts, patient medication diaries, and so on. Inadequate drug accountability is attributed as one of the top five major inspection findings

 behind inadequate patient medication adherence.

In addition, IMPs are traditionally prepared at the study level requiring overage calculations to ensure sufficient stock at a global level. This, coupled with the regular needs for relabeling, (i.e., linked with new IMP stability data) results in significant supply waste. Studies have shown that typically at least 20-40% of prepared IMPs are never used by a patient.

And lastly, due to the COVID-19 pandemic, the already complex medication flows have been further disrupted. Alternative delivery methods to ship IMPs directly to the patient’s home or local pharmacies were put in place to ensure continued access to study drug. Virtual mechanisms to explain dosing regimen, digital enablement for medication intake and verification, involvement of home nurses, etc. are just a few examples of methodologies that were embraced in a record time to ensure continuity. Many of these technologies are not new but were unable to pass the prototype stage pre-COVID, as there were always the big “what if”, “can we do this”, “show me your business model/ROI” questions that halted broader roll out.

In this article, we will get a flavor of what the next generation medication management in clinical trials should entail and share some insights on smart technologies and synergistic partnerships, backed by real-life implementation use cases. We shall look at three key tenets to enable smart medication management:

Complicated treatment regimen, complex and difficult-to-understand medication instructions, limited to no personalized support, and lack of real-time insights into the happenings at home are just a few factors that can increase patient non-adherence.

 To give an example, currently multilingual medication booklet labels are used as standard in clinical trials. These medication booklet labels usually run up to 60 pages with each official language in any country requiring a separate label, the print font is extremely small, and information shared is incomplete (e.g., for dosing instructions, the patient needs to consult another document). It is therefore no surprise that patients have a hard time understanding and complying to medication instructions. Many booklet labels are hence returned unopened by patients.

But the good news is that many smart technology investments and cross-industry partnerships have happened in this area enabling a more patient-tailored approach.

Personalized notifications specifying details of when and how to consume the medication, multimedia tools such as hands-on and easy-to-understand instructions and educational videos in patient’s native language, accessible anytime, anywhere, using their own smartphone can guide patients in a personalized way throughout their medication intake journey.

Digitalized, all-in-one medication labels combining data from different sources, and real-time label updates is another example of a smart solution that has been highly commended by patients.

 Real-time insight for the sites of patient’s understanding in label updates can also be highly beneficial in case of critical communications such as early study termination

In addition, medication packages too can be of great support for patients to adhere to their dosing regimen. For example, smart packages—which are the traditional medication packages supplemented with electronic circuits, chips, and sensors—can track and verify medication intake till the individual pill or unit level and trigger alerts in case of inconsistencies to patients and sites.

 As an instance, in a Phase II Alzheimer study using smart blisters, a participant performed a wrong intake (he erroneously doubled his intake since he had forgotten his previous intake), that was immediately picked up and alerted by the system to site who could then connect and intervene directly with the patient.

Such advanced medication tools can also be used to trigger motivational messages based on thresholds, or prompt specific questions in case of incorrect dosage or intake outside the allowed time window, or even identify behavioral changes and prevent potential cases of dropout.

Age and use of technology are often also raised as an area of concern. While younger people are believed to be more familiar with technology, studies have shown that elderly patients too are quite open and supportive of these technologies even if they have had limited to no prior experience with smartphone.

 Easy-to-use and simple features supported by clear and easy-to-understand training material is an important pre-requisite for elderly patients. In such population, medication compliance also remains very high (+90%) even after six months as exhibited in a recent study conducted for the elderly Asian population, by leveraging personalized patient notifications and support.

And lastly, involving relatives or caregivers of a patient, and providing them with a read-only app or allowing them to perform certain activities on behalf of patient has also found to be very useful and highly recommended.

The erstwhile medication process is fractious involving several stakeholders, isolated systems and is often guided by a paper-based, manual approach involving hand-offs between multiple stakeholders. These include Contract Manufacturer Organizations (CMOs), depots, pharmacies, couriers, Interactive Response Technology (IRT) 

 Randomization & Trial Supply Management (RTSM) suppliers, medication destruction facilities in addition to clinical site personnel for dispensing and the patients—the most critical stakeholders—for intake.

The process silos introduce a lot of inefficiencies and gaps in the process and are very labor-, time-and cost-intensive. What if there was a mechanism to integrate and interconnect all systems and stakeholders and ensure automated and seamless flow among diverse activities? What if all partners could have a real-time view of the medication process—where, what, and when? Or what if medication packages could automatically control parameters such as temperature, storage condition, etc. and alert sites and patients in case of concerns?

Recent advancements have shown that the above futuristic scenarios aren’t mere concepts anymore but are already becoming a reality. Collaboration between various technology, medication suppliers and pharma companies has enabled the creation of unified and integrated web portals and apps for patients and sites with real-time data flows, analysis, and insights.

System integrations too can improve the data quality and integrity and pick up errors that might hitherto have gone undetected. For instance, in a recent study conducted for top-three pharma, a real-time RTSM integration with a barcode medication scanning tool resulted in an elimination of 10 kit assignment errors per month.

Smart packages, that can measure temperature and storage conditions in real-time, or individual pill / unit removal as explained in previous section, can also have a significant impact with eliminating many of the manual, time, and cost-consuming activities. For example, the entire drug accountability overview or specific forms such as site medication destruction forms or prescription forms (a regular ask for direct to patient supplies) can be automatically generated from the system with one click. Reports have shown that ‘Temperature Out of Range’ error in shipments can count for 13 billion/year leading to a total loss of up to $35 billion/year considering the cost to replace and labor involved.

While anytime, anyplace real-time insights on medication flows can bring significant value, having a secure stakeholder-based access management protocol in place is also an absolute necessity. For example, in direct-to-patient medication shipment scenarios, patient’s address and contact details can be securely stored in the system after participant’s consent is obtained but access must be strictly controlled. Only specific stakeholders such as courier services and home nurses can have access to these Personally Identifiable Information (PII), which at the end of the study should also be permanently deleted from the system.

Flexibility is a key word that we have learnt and put in practice at all levels and in a record time during the COVID-19 pandemic. Patients who were unable to travel owing to the social distancing norms, still needed to have access to life-saving drugs. As a result, alternative approaches combining technology and partnerships were put in place.

Tracking and verifying kit flows between different entities using simple technologies such as kit barcode or QR scanning have been implemented along the entire medication flow (e.g., Contract Manufacturing Organization, depot, sites, local pharmacy) even till the level of the patient confirming the right kit delivery by the courier using his/her own smartphone. Even implementing uber-like real-time notifications—providing insight into where the medication kit is—are items that are being considered.

Also offering sites the capability to define, even at an individual visit level whether the medication kit will be picked up or provided at the site, or shipped by courier to the patient’s home, or the local pharmacy, is another example of flexibility being introduced.

Flexibility to use alternative processes in case of issues is another example. For example, in case scanning technologies are not working, typing manual kit numbers should be available. Or in case the patient cannot confirm the receipt of courier delivery using his/her smartphone, the site can perform this activity remotely.

Lastly, introducing flexibility in the medication process was also a key ask even prior COVID. For example, providing sites with the ability to postpone treatments based on results (e.g., oncology treatments) and having digital engagements automatically updated to temporarily pause medication reminders and diary completion. Or even providing sites with the ability to combine medication handouts in line with patient’s holiday plans are just a few examples of flexible medication processes.

The possibilities of combining technology and partnership are endless but as with all novel processes, change management cannot be underestimated. Involving all stakeholders early and through the entire process i.e., from the design of the tool is highly recommended and proves extremely valuable in the long run.

Understandably, it won’t be a one size fits all approach and human interaction remains an important cog. Technology can never replace the site-patient interaction but can play a pioneering role in improving this relationship. Technology also doesn’t possess a magic wand to resolve all challenges, with medication regimen sometimes just not being realistic, requiring life science companies to go back to the drawing board and rethink.

In addition, to level up with the dynamic environment, new capabilities such as voice enabled packages, drones for drug delivery, personalized medicine, are being constantly explored and prototyped.

All of us understand that adoption of digital technologies in activities pertaining to medication in clinical trials has lagged for multitude of reasons including GMP ecosystem, lack in maturity of modern technologies, lack of guidelines etc. However, the COVID-19 pandemic situation has compelled us to break traditional silos and identify and implement novel approaches for carrying out medication management, placing patient at the center of care. Going back to the days of yore with manual approach governing limited patient and site support, fragmented ecosystems, non-integrated and rigid systems is a thing of past! As we enter the next generation of medication management fueled by innovative technologies and symbiotic partnerships, we can realize preventive, augmented, and embedded smart medication management. Let’s come together and go for it!

is the Head Industry leader, Life Sciences and Healthcare, for

Joann Lindenfeld, Mariell Jessup; EUR J Heart Fail; National Library of Medicine; 10-Sep-2017; 29-Jul-2021; 

https://pubmed.ncbi.nlm.nih.gov/28891126/

Beena Jimmy, Jimmy Jose; Patient Medical Adherence: Measures in Daily Practice; National Center for Biotechnology Information; 26-May-2011; 01-Aug-2021; 

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3191684/

Adherence to medications: insights arising from studies on the unreliable link between prescribed and actual drug dosing histories. Blaschke TF, Osterberg L, Vrijens B, Urquhart J. Annu Rev Pharmacol Toxicol. 2012; 52:275-301

BIMO Metrics; FDA; 09-Feb-2021; 02-Aug-2021; 

Classification and analysis of the GCP inspection findings of GCP inspections conducted at the request of the CHMP; EMA; 01-Dec-2014; 02-Aug-2021; 

https://www.ema.europa.eu/en/documents/other/classification-analysis-good-clinical-practice-gcp-inspection-findings-gcp-inspections-conducted_en.pdf

Stefan Dürr; Meeting the clinical supply challenge with IRT; Applied Clinical Trials; 14-Sep-2017; 04-Aug-2021; 

https://www.appledclinicaltrialsonline.com/view/meeting-clinical-supply-challenge-irt

Jodi Smith-Gick, Nicola Barnes, Rocco Barone, Jeff Bedford, Jason R. James, Stacy Frankovitz Reisner, Michael Stephenson; The Near-Term Viability and benefits of eLabels for patients, clinical sites and sponsors; SAGE Journals; 02-Apr-2018; 04-Aug-2021; 

https://journals.sagepub.com/eprint/6Y3TQ6sz38kNTnmK23Wp/full

Hilde Vanaken; Digitally Enhanced: Janssen drives effort to bring suite of “smart” clinical trials into practice in 2017; Applied Clinical Trials; 02-Dec-2016; 04-Aug-2021; 

https://www.appliedclinicaltrialsonline.com/view/digitally-enhanced-smart-trial-platform

Hilde Vanaken; Smart Tools Enabling Patient Engagement; Conference Forum; 04-Aug-2021; 

https://theconferenceforum.org/wp-content/uploads/2019/01/PatientsEU2019_HildeVanaken_SmartTools.pdf

Bernard Vrijens; The Role of Electronic Monitoring in Maintaining Medication Adherence; Contract Pharma; 14-Oct-2020; 05-Aug-2021; 

https://www.contractpharma.com/issues/2020-10-01/view_features/the-role-of-electronic-monitoring-in-maintaining-medication-adherence/#:~:text=Electronic%20monitoring%20shows%20that%20poor,adherence%20to%20the%20study%20medications.

Antoine Pironet; Medication adherence monitoring and management: a closer look at the role of data science and understanding patient behaviour; Applied Clinical Trial; 17-May-2021; 05-Aug-2021; 

https://www.appliedclinicaltrialsonline.com/view/medication-adherence-monitoring-and-management-a-closer-look-at-the-role-of-data-science-and-understanding-patient-behavior

Claire Swedberg; NFC helps track medication use in clinical trials; RFID journal; 31-Jan-2019; 06-Aug-2021; 

https://www.rfidjournal.com/nfc-helps-track-medication-use-in-clinical-trials

Shaping the future of Decentralized Trials; TCS; 06-Aug-2021; 

https://www.tcs.com/decentralized-clinical-trials-patient-centric-webinar

Marissa F Dockendorf, Bryan J Hansen, Kevin P Bateman, Matthew Moyer, Jyoti K Shah, Lisa A Shipley; Digitally Enabled, Patient-Centric Clinical Trials: Shifting the Drug Development Paradigm; National Library of Medicine; 30-Nov-2020; 07-Aug-2021; 

https://pubmed.ncbi.nlm.nih.gov/33048475/

Allison Proffitt; Building A Winner: Janssen on Collaborating on a Connected Clinical Trial Platform; Clinical Research News; 22-Oct-2019; 07-Aug-2021;

https://www.clinicalresearchnewsonline.com/news/2019/10/22/building-a-winner-janssen-on-collaborating-on-a-connected-clinical-trial-platform

Henry Kerali; The implication of cold chain failure; Clinical Trials Arena; 03-Mar-2017; 08-Aug-2021; 

https://www.clinicaltrialsarena.com/news/the-implications-of-cold-chain-failure-5755057-2/

What the next generation medication management in clinical trials should entail and insights on smart technologies and synergistic partnerships—backed by real-life implementation use cases.

 Smart New Normal in Medication Management—Personalized, Integrated, and Flexible

Clinical trial inclusion in the biopharmaceutical industry had been driven primarily by federal initiatives. However, there seems to be a renewed strategy in big pharma to incorporate diversity inclusion in clinical trials. For example, 

Eli Lilly has discussed its clinical trial diversity inclusion strategies

, which seems to be driven by social injustice. Now, Genentech/Roche is taking on diversity inclusion as a socially responsible strategy. In this interview, Nicole Richie, Global Head Health Equity and Population Science at Genentech/Roche, will discuss her perspectives on the topic.

Moe Alsumidaie: Can you tell me why Genentech formed the Advancing Inclusive Research Site Alliance, and what was the motivation behind this? What sparked this initiative, and why now?

 We started this work several years ago with the appreciation that clinical trials are homogeneous and that the majority of patients included in clinical trials are of one race, and ancestral background—only five to 15% are not Caucasian. A couple of reasons this was front of mind was, for one, the United States demographics are shifting—we are becoming a more diverse and minority-majority nation. The second element that caused more of an increased focus internally was that we thought about personalized healthcare and invested in that as a pharmaceutical company. With that came this burgeoning awareness in the clinical and scientific communities that genetic and clinical data are homogeneous.

All of the information that’s readily available to us is limited. And the implications of that as we try to develop more and more personalized treatments are significant. It undermines our ability to have more personalized care. And so there was just this kind of bolus of publications and literature around this.

That tipped us into having more of a discrete focus on this work back in 2017. As it evolved, we realized that one of the mechanisms to have more representation in research is by having sites in the right location, with the right capabilities and the proper outreach into patient communities. That is where this focus on an Alliance model was born. It was intended to create an infrastructure that will allow for sustainable and durable commitment and a building of partnerships to have more representative patients in our trials and our research environment. We started with four sites, with the aspiration of having a network that ultimately serves these cohort centers of excellence and partners with us to achieve our aspirations for diversity in clinical research.  

MA: How does that fit with Genentech’s overall product development strategy? You mentioned personalized medicine, and of course, Genentech is big on oncology. Does that have something to do with that? Or is it more of a federal or social mandate that you’re following?

 It is to bring more representation into our clinical trials and to bring greater real-world data cohorts, both clinical and omic, into our strategies and planning and our process that informs our personalized healthcare approach. And then to partner strategically across the healthcare ecosystem with the appreciation that this is a big challenge, and we have to come together in different ways to integrate new solutions.

This work, in general, is about having more representation in our research programs. Roche is fully supportive and synergistic with our aspirations for personalized healthcare, as they align with many of Roche’s vision as a pharmaceutical company to impact patients’ lives. By moving into a more comprehensive solutions-based approach, and not a transactional medicine, we we can impact patient care in an end-to-end manner.

Then, we also think about what our impact on society is. So we are asking questions like, “How do we reduce the burden for society?” Healthcare costs are unsustainable, and this is not a new problem, but as institutions and stakeholders continue to move more and more towards value-based care, these types of solutions and integrating the totality of health becomes front and center to what a pharmaceutical company needs to focus on.

So as such, this is a crucial component to broadening the lens on who informs our science and medicine and who benefits from our innovations. And it’s also coupled with our “Bench to Bedside” efforts that are happening across the organization. So it is the research endeavors, but also making sure that we partner holistically with the access pathways to ensure that we’re bringing not just research, but enabling science understanding in under-studied patient populations and ensuring that patients get served in new ways and have access to these innovations.

MA: What does the diversity currently look like with Genentech’s trials, and what milestones do you have to increase the diversity, and what does that mean for clinical trials?

 Currently, you can look at the last new molecular entities that were published on FDA Snapshots. The metrics are getting better, and the most recent NMEs were approved with the caveat that these are global trials and comprised of different countries enrolling at different rates. So what used to be upwards of 90% of all European or white patient demographics has moved to around 75%, and in some cases even less than that.

So that population that’s growing in some cases is through China. We are doing more and more studies in China, and we’re bringing in more Asian patient populations. We still have much work to do, I would say, in bringing in African American and Black patients and Latin and Hispanic patients, both in the US and Latin America, for example.

The demographics of the studies have to be viewed within what recruitment occurs in the US versus outside of the US if we compare to US metrics. So, for example, in our most recent geocentric approval, the representation of US recruitment is very much in alignment with what you would expect for the epidemiology of disease. However, if you look at the whole global population, it gets diluted that the minority of patients come from the US.

It is contextual as we think about benchmarking and baselining. We are using epidemiology as our metric of what we’re trying to drive towards. We are putting together an understanding of where we were before, so looking at that historic recruitment is a first them. Then, our goal will ultimately be to align to the epidemiology of disease and what we would expect based on demographics. Those are the metrics we’re trying to achieve as we think about what “good” looks like. This is a long process because recruitment, depending on what therapeutic area you’re talking about, takes several years of integrating mitigations or implementing new tactics that, over time, move the needle.

We have seen some great examples in our EMPACTA study. The study was conducted during the COVID-19 pandemic, and it included patient populations in the US. Approximately 84% of patients were non-White and from places worldwide, including Latin America and Africa, and in the US like the Navajo nation and New Orleans, where patients were disproportionately affected by the disease.

We had to show with that study that it’s possible, and we can find these patients. If we can do it in a global pandemic, I’m sure we can think about how to do it more holistically. So many learnings have come from that. There have been several proofs of concepts that have shown what we can achieve. Now it is a matter of how we bring it into the whole totality of the lifecycle of development.

I understand you have global partner trial sites. Can you tell me more about those partner trial sites and how they are working successfully to include diversity while also de-risking it as much as possible?

 Absolutely. That is undoubtedly the plan. We launched the first-ever oncology industry study in Kenya this year. So there are sites that have that high rigor and that fidelity that is necessary, but we don’t typically think about them in that way. From the global vantage point, we’ve been thinking about where those patient populations are particularly understudied. So you end up in Latin America, Africa, India, these vast regions. With the appreciation that, to your point, we have to have an infrastructure that serves us.

In a similar manner that we have established the Advancing Inclusive Research Site Alliance model in the US, we are looking at how we can do something that’s probably quite different in the actual tactical planning but has the same intentionality. So that we again create this infrastructure that is a center of excellence and has fidelity, conveys trust, and creates partnerships that can be forged over time and then maintained.

The other thing we’re looking at is how Europe and the UK are rapidly diversifying, in certain regions more than others. So really, it takes much global strategy with local implementation and information, which is typically how we do things. So it fits right into our current operating model.

MA: Can you tell me about the disparities in clinical trials? What can the pharmaceutical industry essentially do to bridge that gap? Or at least try to bring that gap together through bringing those innovative and breakthrough medical products to those patients?

 I think there are a couple of elements. As a pharmaceutical company, we can intervene in many ways from a research and data generation perspective. We are moving into real-world data and other forms of information that we can use to supplement and facilitate our understanding of populations that have almost entirely been understudied and left behind by science and medicine.

There is a commitment to leveraging more considerable data assets to generate insights and contribute to scientific discourse in a new way. We’re trying to achieve population-specific medical elements based on the appreciation that disease affects populations differently. For example, there are well-known variations in clinical course and even response to therapy due to therapeutics attributed to race, ethnicity, and genetic ancestry.

As a science company, we have to get the science right. But, we also have to think about health equity, access, and the totality of health. I think that’s deeply at our intersection of how we think about personalized healthcare.

Because research is just one piece of the broader health continuum, there is a leadership element to showing and setting industry standards on addressing health equity.

We must partner in new ways across the ecosystem, which we currently do in many different avenues through charitable giving, for example. We also do so through our External Council on inclusive research and health equity, which includes key opinion leaders that are very established in the health equity space representing healthcare professionals and patient advocacy groups and organizations.

Our role as a biotechnology company is twofold. Firstly, it is to get the research and the science right, increasing the clinical utility and results generation. Secondly, we need to make sure that we address access deprivation and the channels needed to ensure that patients are getting access to our medicines.

We also have to put a line in the sand with public declarations of our intention, hoping that we will instill that in other companies. Because it will take all of us coming into this space to achieve more remarkable health outcomes for more patients. Ultimately it’s also to mobilize the industry and enterprise at large.

MA: The Advancing Inclusive Research Site Alliance is one of the many examples of what Genentech is doing as a trailblazer in the industry. How do you anticipate that the biopharmaceutical industry will also change to address diversity?

 Hopefully, this is setting the standards. We are very active in pharma trade organizations and other cross-industry collaborations, and we are even publishing our approach and methodology. For example, the Advancing Inclusive Research Site Alliance announcement shared how we’re thinking and what infrastructure and foundational elements we’re putting in place are serving us well.

One of the things I like about our approach is that it is very much in Genentech fashion to partner with experts. So this is where again the External Council comes in. We have these incredible minds who have been in this space for decades and can help challenge us and inform our process. So there are tangible outcomes from that.

For example, on the council, we have an incredible multiple sclerosis (MS) clinician who has observed that the clinical course and outcomes look different in Hispanic and Black patients. So, as a result, we now have a study called CHIMES to address this specific hypothesis in Black and Hispanic patients. It goes to show that in pure Genentech fashion, we’re harnessing the best and the brightest because we know that this is bigger than just one company. We understand that this is a substantial multifaceted challenge, and we have the humility to say that that is the case.

There is no magic bullet. This takes much learning and much iteration, and it’s probably different across disease areas, as we’ve come to appreciate as well.

I think the leadership element of this also comes in the fact that we resource it. I have a team of people that focus on this that serve this work globally. Quita Highsmith, our Chief Diversity Officer, has a strategic pillar concentrated on this. There’s a US medical affairs group focused on health equity and inclusive research. Both of our research institutions in Genentech and Roche have cross-functional teams that have come together around this. So I think it all comes back to giving it time, space, money, and funding.

, 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.

Nicole Richie, Global Head Health Equity and Population Science at Genentech/Roche, discusses a renewed strategy in big pharma to incorporate diversity inclusion in clinical trials.

Clinical Trial Diversity Inclusion is Becoming a Socially Responsible Initiative

Clinscience, on September 15th signed an investment agreement to acquire 60% of the digital CRO—EXOM Group. Headquartered in Milan, EXOM carries out its activities in the design, supervision, monitoring and management of Clinical Trials based on its own digital eco-system of more than 20 applications unified in the multi-modular platform Genius Suite.

EXOM Group's proprietary Genius Suite system will allow Clinscience to continue its digital transformation and conduct Clinical Trials with a focus on the needs of both Investigators and Patients and ultimately Sponsors.

Clinscience Digitizes Clinical Research by Investing in EXOM Group

Syneos Health has announced the acquisition of StudyKIK, a technology-enabled clinical trial recruitment and retention company. StudyKIK provides eConsent, Telemedicine video calling, Phone Pre-Qualifications, and Patient Retention application solutions. The company manages over 1,000 indication-specific patient community groups and has enrolled more than 5,400 protocols supporting over 3,800 research sites across the globe. The platform encompasses more than 6 million patients through social media.

Syneos Health Acquires StudyKIK

 as the Global Head of Clinical Operations.

Premier Research announced three new oncology leadership team hires: 

, has joined the company's oncology team as Vice President, Global Head of Medical Affairs. 

, MS, MBA, has been named Vice President, Medical Oncology Strategy, and 

, has joined as Executive Director, Oncology Strategy.

 has been selected to co-lead the Decentralized Trials & Research Alliance’s Initiative 4B: Collaborate on Regulatory Gaps.

 was recently named the winner of a Gold Stevie Award in the Executive of the Year Health Products & Services category and a Silver Stevie in the Woman of the Year category in the 18th Annual International Business Awards.

 to the post of Chief Marketing Officer.

Clinical Research IO (CRIO) announced that 

 has joined them as Senior Vice President of Sales.

 was named a top leader of life sciences operations and services to the global biopharma industry by Everest Group’s latest report.

 has received a patent for the technology underpinning its Patient Network Explorer. The patent, EP 2015/059415, “Patient Recruitment System And Patient Recruitment Method,” was published in the European Patent Bulletin 2021/30 on July 28, 2021.

 announced that they have launched a new brand identity and name change to 

 Simcyp COVID-19 Vaccine Model was named a finalist in both Informa Pharma Intelligence’s 2021 Citeline Awards in the Excellence in Innovation in Response to COVID-19 - Clinical Trial Activities category and R&D World’s 2021 R&D 100 Awards in the Software/Services category.

 has been named as one of the top five 2021 Best Places to Work by the 

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Business and People News —9/21

Egnyte has announced Egnyte for Life Sciences eTMF, a product for the clinical development functions at life science companies to manage trial master file data. Egnyte for Life Sciences eTMF is 21 CFR Part 11 compliant solution that aims to reduce document processing time while maintaining compliance and audit readiness.

Egnyte for Life Sciences eTMF solution enables clinical development professionals to:

 built on Egnyte’s secure collaboration platform, get full visibility into study milestones, allowing clinical teams to communicate with sites to stick to timelines.

 Egnyte’s governance features allow teams to flag missing source documents, leverage AI and ML features for constant monitoring for misclassified documents or embedded PII/PHI.

 meeting GxP and FDA 21 CFR Part 11 requirements, Egnyte allows companies to stay audit-ready and maintain compliance in a cloud-based solution.

Egnyte Announces Solution for Trial Operations

In an important step for modernizing drug formulation and biopharmaceutical development, FDA is launching a program to test the safety and suitability of novel inactive ingredients for use in new drugs and biologics. This final initiative, first proposed two years ago, supports a two-year pilot program to test up to four new excipients likely improve the effectiveness and safety of important new drugs.

 The aim is to encourage sponsors of clinical trials to formulate innovative therapies with those new excipients that FDA finds acceptable for use under this program.

FDA spelled out the pilot program in a Federal Register notice published Sept. 8, 2021.

 The agency explains that the initiative is open to novel excipients that have not been used previously in food or in FDA-approved products and how excipient manufacturers looking to participate should submit information by Dec. 7, 2021 on the novel inactive ingredient and its potential for enhancing public health. The program aims to evaluate four selected novel excipients likely to advance drug development, such as by increasing solubility for an oral formulation taken chronically. Those ingredients authorized under this program then may be used in the formulation for an experimental drug slated for clinical trials, provided that the sponsor demonstrates that it is safe for use in that specific treatment. After FDA approves a new drug or biologic utilizing the new excipient, that ingredient then would be added to FDA’s Inactive Ingredient Database, making it available for use by other manufacturers, including those producing generic drugs.

Manufacturers of drugs and biologics, as well as excipient developers, have long sought a process for FDA to evaluate new inactive ingredients prior to use in a new drug or biologic to support the development of innovative formulations without fear of delay or complications in FDA review and approval of the new product. Biopharma companies support this effort to avoid difficulties in formulating innovative drugs with available, established excipients, for example, in looking to stabilize proteins in new formulations. Excipient makers similarly acknowledge that they would be more willing to invest in production and characterization of novel excipients if it’s possible to gain FDA review of the product’s benefits outside of clinical testing for a new molecular entity. The initiative, as well as ongoing efforts to set international standards for excipient use, have potential to enhance drug formulation and support modern manufacturing and quality control efforts.

https://www.fda.gov/drugs/development-approval-process-drugs/novel-excipient-review-pilot-program?utm_medium=email&utm_source=govdelivery

https://www.federalregister.gov/documents/2021/09/08/2021-19335/center-for-drug-evaluation-and-research-office-of-new-drugs-novel-excipient-review-pilot-program

FDA is launching a program to test the safety and suitability of novel inactive ingredients for use in new drugs and biologics.

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