Vice President, Sites and Patients Center of Excellence, PPD Clinical Research, Themo Fisher Scientific

Rodrigo Garcia, MD, MS

Drawing insights from industry news and the findings of a 

 of over 150 biopharma and biotech leaders conducted by the PPD clinical research business of Thermo Fisher Scientific, the following rise to the top as four notable lessons learned in drug development over the past year and what they might mean for 2023.

Lesson No. 1: Decentralized trials have redefined what is possible, but have yet to reach their full potential

As stakeholders across the drug development industry would attest, decentralized clinical trials (DCTs) have been a point of much interest over the past several years. COVID-19 highlighted the value of remote approaches in many fields, but DCTs offered an opportunity beyond the needs of the pandemic: to make clinical research easier for patients to participate in and more accessible to a wider population not served by traditional study design.

The use of decentralized and hybrid clinical trials has increased considerably—51% of respondents in our survey reported that the COVID-19 pandemic encouraged their organization to decentralize clinical trials. Those whose organizations already use DCTs anticipate their use increasing by 24% over the next two years. DCTs offer a potential solution to mounting concerns over patient recruitment and retention, as 

 that decentralized and hybrid models may yield enrollment 1.3 to 2 times faster than traditional approaches and retain those patients at a 90% higher rate. Patients also are enthusiastic about the opportunities offered by these trials. In 

one survey published in JAMA Network Open

, 80% of cancer patients and survivors reported being open to remote interventions, and 77% said they’d join a trial if it were at least as easy to access as regular care.

While technology is expanding rapidly to support the unique needs of these trials, sponsors and sites often face decision paralysis in the face of so many tech options. Many excellent tools are available, but sites and sponsors generally need multiple tools to carry out a study—each of which has dozens of potential options that may not work well together or with the tech at partnering organizations. Good training, user-friendliness and seamless operation are key to making a strong DCT tool, although no perfect option yet exists. The field has a great deal of work to do for DCT tools to reach their full potential. It is also important to note that deployment of technology and decentralized clinical trial design should not take a “one-size-fits-all" approach. A best practice is to perform an intentional, thoughtful assessment for each protocol to determine what makes sense for specific patient populations, schedules of events and procedures, disease areas, and trial objectives.

In 2023, we can anticipate that decentralized and hybrid clinical trial models will continue to expand as technology and infrastructure improve to support them. DCTs also may be a critical factor in meeting 

 to recruit and retain a more diverse patient population. To address the needs of busy staff of decentralized and hybrid trials, clinical trial technology will likely move to consolidate functions and focus more on total workflow solutions rather than tackling single elements of a trial. In our survey, 32% of respondents said their organization has trouble keeping up with trial technology and innovation. Increasing integration of different platforms and focusing on usability will streamline workflows for sites and sponsors, paving the way for decentralized, hybrid and digital clinical trial approaches.

Lesson No. 2: The value of real-world data goes beyond payer applications and can accelerate drug development

Advances in data science and analytics have demonstrated the value of RWD for stakeholders beyond health care payers. RWD is now essential to drug development. As an example, RWD can be leveraged to overcome clinical trial recruitment and retention issues. Our survey found that:

59% of organizations are adopting RWD and RWE in their studies

45% of respondents consider RWD/RWE the top opportunity in the drug development sphere

Clinical trial sponsor companies can best leverage RWD for their trials by using electronic medical records and census data in their trial planning and site selection, which will allow them to better locate patients in the population of interest and streamline outreach to qualified candidates. With 55% of survey respondents naming recruitment as their top challenge in clinical development, there is a clear need for new approaches to reach patients.

It’s inevitable that the use of RWD and RWE will become more commonplace in clinical trial design, facilitating improved outcomes and drug development timelines. In addition to using these data to inform recruitment strategies and trial parameters, drug developers also can leverage RWD and RWE in single-arm clinical trials as an external control arm, a practice that will likely grow as RWD sets become more accessible.

Lesson No. 3: Taking a transformational, partnership-focused approach to work with communities and patient advocacy groups can be a benefit both for sponsors and patients

As part of their effort to increase diversity and equity in clinical research, organizations have changed howthey work with patients by 

 with communities and patient advocacy groups. Companies are taking a more holistic approach when starting a research program, engaging stakeholders early and often in decision-making processes and trial operations. These relationships can be a significant value add for both parties.

Working with advocacy groups enables sponsors to access an enthusiastic and appropriate candidate pool while increasing patient access and participation. Additionally, patients and patient advocates offer a unique and credible perspective for clinically meaningful input disease burden and trial design. This is especially valuable for the sparse populations targeted by rare disease trials, which 39% of our survey respondents noted as a priority therapeutic area.

Importantly, welcoming and actively gathering input from patients and advocates during clinical trial design also can help to create more patient-centric studies. This not only makes participation easier and more pleasant for patients but also can yield better insights. By seeking counsel from patient communities and advocacy groups, investigators can select study endpoints that are truly meaningful to patients’ experiences rather than focusing on traditional outcomes. Instead of measuring 25-foot walking speed, for example, a study could choose endpoints more reflective of a patient’s daily function and lived experience.

We can anticipate that collaboration between drug developers and advocacy groups will continue, especially as we start to see returns in the form of positive study conclusions and promising drug approvals. The dedication of advocacy groups will likely keep pushing sponsors to build on progress made thus far, furthering growing relationships that benefit everyone involved.

Lesson No. 4: COVID-19 increased awareness of clinical research, but public perceptions haven’t improved as much as expected

While trials for the COVID-19 vaccines brought clinical research into the public spotlight and motivated thousands of newcomers to participate in clinical research, increases in study enrollment have not persisted as anticipated. A 2021 

 by the Center for Information and Study on Clinical Research Participation found that, while overall awareness of research had increased, self-reported understanding of clinical trials and willingness to participate has decreased since 2019. In fact, between 2019 and 2022, the percentage of respondents stating they were “very willing” to participate in a clinical trial dipped from 34% to 30%.

While the report found that trust for research centers, government research organizations and regulatory agencies increased from 2019 levels, distrust of pharmaceutical companies persists 

even among clinical research participants

, which may be a limiting factor in growing outreach.

Despite this perceived lack of trust between sponsor companies, patients and advocacy organizations, ongoing efforts to strengthen community engagement and improve research tools likely will help foster wider positive awareness of clinical research. The continued growth of DCTs certainly will add to this trend. By meeting participants where they are and becoming part of the community, sponsors can employ decentralized approaches to build trust and reduce patient burden. Additionally, as trial digitalization increases and technology improves to support clinical trial processes, enhanced engagement and convenience will make trial participation a more patient-friendly experience.

, MD, MS, vice president, sites and patients center of excellence, PPD Clinical Research, Thermo Fisher Scientific

Articles

DCTs and RWD among topics for industry to focus on moving into 2023.

Lessons Learned in 2022 and the Impact on Drug Development in the Year Ahead

The clinical trial landscape is more crowded than ever, with nearly 60,000 studies launched worldwide in 2021 alone.

 This expansion of clinical research will undoubtedly bring thousands of patients with life-altering conditions closer to therapies and cures. However, the busy research landscape is also demanding that sponsors and key stakeholders transform how to set up their clinical trials up for success.

The boom in trial volume presents new challenges to clinical trial organizers and administrators throughout the industry. More than 80% of clinical trials fail to meet enrollment goals on time, leading to significantly prolonged study timelines, protocol amendments, and increased costs.

 As drug developers prioritize more advanced, targeted therapies, competition to recruit from limited candidate pools within specific research indications also has increased dramatically.

Despite these challenges—in addition to high rates of turnover among clinical research staff and increasing trial complexity—the launch of new therapies hinges on sponsors’ ability to accelerate their trials and ensure they run efficiently.

 So how should biotech and biopharma organizations adapt their clinical trial strategy in an increasingly busy environment?

In this article, we’ll discuss five key areas to consider from the onset of a trial to make your study a priority in this crowded field.

1. Engage with clinical trial stakeholders from the start

What is one simple way of understanding what trial sites and participants need for a study to run smoothly? Proactive communication. It may seem obvious, but sponsors that seek and incorporate perspectives from sites’ staff and potential participants as early as possible in the trial planning and startup process are more likely to succeed in operationalizing truly patient- and site-centric trials.

 Early engagement ensures the goals and parameters of the study are realistic for those who develop, manage, and participate in clinical trials.

For example, site staff can provide valuable insight regarding the targeted patient population, potential recruitment numbers, and best practices and lessons learned at their site, which then influences development of feasible enrollment timelines and inclusion parameters. Additionally, a recent mandate by FDA requires sponsors to submit a “Race & Ethnicity Diversity Plan” for every study to ensure adequate representation of traditionally underrepresented groups in clinical trials.

 By seeking input from the target patient population in the conceptualization of their trial development and diversity plans, sponsors

Can create effective strategies to reach patients from diverse and underrepresented backgrounds;

Develop more inclusive, culturally appropriate trial plans; and

Potentially avoid costly protocol amendments and delays.

2. Take a data-driven approach to identify new sites and patient populations

Many sponsors lean on the same sites for study after study, leaving other potential sites, physicians, and patient populations unknown or untapped. A data-driven approach, leveraging multiple data sources, offers drug developers potential competitive advantages such as: trial optimization; expanded site and patient networks beyond “usually” crowded and constantly tapped sites; and new patient populations.

The feasibility process conducted by a clinical research organization (CRO) is crucial to introduce new sites to clinical research and have them join the clinical trial landscape. CROs must conduct extensive feasibility analyses and follow detailed processes prior to finalizing site identification and selection for an upcoming clinical trial. This process is executed in close collaboration with sponsors and even vendors involved in the clinical trial.

With access to deep and transparent site data, CROs have a proven, data-driven methodology to guide sponsors through the site-selection process. This wealth of data is paramount not only to identify and highlight well-equipped sites, but also to identify underutilized sites with the potential to bring value to the clinical trial. For example, if a feasibility analysis reveals that a potential site has a strong patient pool but lacks the staff or specialized equipment required for the trial, CROs and sponsors can work to fill these resource gaps.

3. Leverage new technology—but do so intelligently

As the clinical trial landscape grows, so does the range of new study tools, capabilities and approaches enabling a trial to become “decentralized.” The PPD clinical research business of Thermo Fisher Scientific recently surveyed more than 150 drug developers across the globe on pharmaceutical and biotech R&D trends and found that, among organizations currently using decentralized trials, usage is expected to jump 24% over the next two years.

 Even organizations not yet using decentralized trials anticipate 27% of their clinical trials will employ this approach by 2024.

However, many trial sites are struggling to cope with the implementation of these decentralized methodologies. While technological developments enable the collection of novel and more complex data and improved data integrity, they also burden already overwhelmed site staff and site support mechanisms. The Society for Clinical Research Sites recently found that 60% of sites use more than 20 systems on a daily basis, and that some sites have turned down studies based on protocol technology.

To enable efficiency, when planning a clinical trial, take time to evaluate the applications and capabilities the study requires and how they fit into the larger site and patient ecosystem. Additionally, consider investing in training, virtual enablement and resources for site staff to ensure they are comfortable with tools and applications. Not only does this provide sponsors valuable feedback about future selection of applications and devices, but it also strengthens and promotes a site-centric mindset from sponsors and CROs.

4. Simplify the trial ecosystem for patients with an inclusive mindset

Patients who evaluate clinical trial participation as a therapy do not base their decision on the intervention alone. They also assess how trial design and procedures may fit into their lives, as well as the lives of their families and caregivers. Our survey on the state of the drug development industry found that patient recruitment, including patient retention and patient diversity, is the biggest pain point for biotech and biopharma companies, with 55% citing it as the top challenge faced by their organization.

To assure that your study is truly patient centric, consider multiple factors that may affect patients participating in the clinical trial, such as: number and duration of on-site visits; distance between sites and patients’ homes; and the need for patient transportation and family support (such as child care). Partially or fully decentralized visits can make participation easier for potential patients who cannot find alternative child care or take time off work. Similar to a well-rounded site engagement strategy, consider user-friendly training and support resources that help patients easily participate. At the same time, it is important not to over-engineer the protocol or over-utilize technology at the expense of accessibility for the target patient population. For example, will older adults be comfortable using tablets or biometric devices for a study, or are the demands too daunting?

In addition, sites find more success in carrying out protocol tasks when they properly communicate the potential therapeutic offering of the clinical trial to all patient populations. Sponsors should regularly evaluate whether their materials convey the significance and value of patients’ involvement with inclusive, non-biased, culturally appropriate and health-literate language.

5. Leverage partnerships to strengthen your trial

Community organizations and patient advocacy groups can expand access to care and therapies in their communities. For them, a partnership with trial sponsors and CROs offers an opportunity to open a new “road” to new therapies. For sponsors, this valuable partnership provides access to honest and patient-centric feedback. Partnerships also enable trial organizers to tap into supportive resources, such as transportation to study visits, which can broaden the pool of potential participants for a study. Ultimately, an investment in relationships that enhance community health access will create long-lasting collaboration among sites, patients, sponsors and CROs well into the future.

Another critical partnership is between CROs and stakeholders (e.g., sponsors, patient organizations, vendors). CROs serve multiple clients and hundreds of clinical trials, and can deliver a deep and dedicated base of knowledge and experience to draw from throughout the planning and execution of a trial. Do you want to know precisely what does and does not work in a trial, pertaining to a particular indication? Tap into the expertise, tools and networks of your CRO. An ideal partnership model with a CRO incorporates shared values, governance, and honest and transparent early engagement to prioritize and co-manage the sponsor’s pipeline.

 Even as the clinical trial ecosystem becomes increasingly complex, experienced trial partners can provide valuable guidance to set your study apart from the beginning.

The expanding clinical trial landscape is promising for the development of lifesaving therapies. And new strategies and technologies—such as adaptive trial design and the use of real-world data and evidence to complement clinical trial data—offer exciting methods for trial organizers and administrators to advance the future of medicine.

However, with so many trials competing to enroll participants and navigate similar timelines, the rapid increase in research has created a daunting, constantly changing reality for those looking to claim space in the field.

Within this crowded ecosystem, leveraging the right input, data, tools, capabilities, and partnerships will position your trial for success.

, MD, MS, Vice President, Sites and Patients Center of Excellence, and 

, Vice President, Head of Development Operations-Biopharma; both PPD Clinical Research, Thermo Fisher Scientific

Number of clinical trials by year, location, disease and phase

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https://www.who.int/observatories/global-observatory-on-health-research-and-development/monitoring/number-of-trial-registrations-by-year-location-disease-and-phase-of-development

Desai, M. (2020). Recruitment and retention of participants in clinical studies: Critical issues and challenges. 

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Mayo Clinic Proceedings: Innovations, Quality & Outcomes

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Contemporary Clinical Trials Communications

https://doi.org/10.1016/j.conctc.2020.100608

Vat, L. E., Finlay, T., Jan Schuitmaker‐Warnaar, T., Fahy, N., Robinson, P., Boudes, M., Diaz, A., Ferrer, E., Hivert, V., Purman, G., Kürzinger, M., Kroes, R. A., Hey, C., & Broerse, J. E. W. (2019). Evaluating the “return on patient engagement initiatives” in medicines research and development: A literature review. 

Diversity Plans to Improve Enrollment of Participants From Underrepresented Racial and Ethnic Populations in Clinical Trials; Draft Guidance for Industry; Availability

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/diversity-plans-improve-enrollment-participants-underrepresented-racial-and-ethnic-populations

Trends in Pharmaceuticals Research and Development

Closing the Gap on Clinical Tech for Sites

https://myscrs.org/resources/sites-now/closing-the-gap-on-clinical-tech-for-sites

The Growing Role of CROs in Clinical Trials

https://www.ppd.com/blog/growing-role-of-contract-research-organizations-in-clinical-trials/

A more crowded space is presenting new challenges for stakeholders that must be addressed to improve efficiency.

Rodrigo Garcia, MD, MS

Articles

Lessons Learned in 2022 and the Impact on Drug Development in the Year Ahead

Make Your Clinical Trial a Priority in a Busy Landscape