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

Applied Clinical TrialsApplied Clinical Trials-02-01-2023
Volume 32
Issue 1/2

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

Drawing insights from industry news and the findings of a survey 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 findings from the Mayo Clinic suggest 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 FDA and industry goals 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 fostering closer partnerships 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 report 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.

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

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