By Kelsey Jakee, PA Consulting
Clinical research is primed for transformation more than at any time before. The events of 2020 have reverberated globally across sectors, but COVID-19 has specifically given developers of new medicines a distinctive role in rising to the occasion. The promise of new innovations developed by the biopharmaceutical industry is now front-of-mind across the public — a feat that no prior industry PR campaign has been able to accomplish so swiftly. And industry leaders are increasingly vocal about a new template for research and development, one that fully embraces the lessons and opportunities revealed this year.
COVID-19 raised new challenges for researchers. Lockdowns and social distancing required reimagining the way existing clinical trials were conducted, often with less travel to clinics by participants. Belief in the speed at which vaccines could be developed skyrocketed. And public attitudes toward the promise of new treatments and vaccines were often polarized, ranging from tentative to trusting. When the whole world is watching, transparency in research takes on a new meaning and a new set of responsibilities.
Last year, shortly before the pandemic, we predicted that clinical trials of the future would be more open, more human-centered, and more integrated in the healthcare ecosystem, providing guideposts for how industry will adapt along these three themes. We asserted that over the next decade, R&D leaders would need to make end consumers (patients, care-givers) the focal point of their business, engineer innovation speed and agility into their organizations, and participate in cooperative, systemic transformation outside company walls. COVID-19 has both accelerated the pace of this change and highlighted new challenges for R&D leaders to address to continue making an impact on health, well-being, and a more positive human future.
COVID-19’s Effect On 3 Emerging Clinical Trial Trends
We have described open clinical trials as those involving the 3 Cs: research infrastructure that is increasingly connected through networks, operations that are more consistent, and stakeholders who share in research more cooperatively. In 2020 these themes have been accelerated, most notably through increased use of trusted research networks, a surge in adaptive platform trials, efforts to share and harmonize protocols, and industry alliances focused on information sharing. For example, Health Data Research UK launched the International COVID-19 Data Alliance, at the center of which is a digital research environment enabling authorized teams to share, request, and analyze data from a single workbench.
Platform trials such as SOLIDARITY, RECOVERY, iSPY COVID, REMAP-CAP, and several organized through the NIH-ACTIV public-private partnership have scaled up quickly, albeit with a general lack of cross-coordination. It seems the energy and momentum to reduce inefficiency and duplication through networks have given rise to a further patchwork of networks. Even with new consortia dedicated specifically to biopharma’s pandemic response (COVID R&D Alliance), coordination proves challenging, highlighting further opportunity to streamline the operation of complex trial designs across industry. Still, the successful launch of these novel designs, particularly ones using existing platforms such as Quantum Leap Healthcare Collaborative’s iSPY, demonstrates the ability to repurpose networked infrastructure across a variety of therapy areas. As trials begin to read out, large troves of data are becoming available for analysis and the sharing of this data across entities is facilitated by nonprofits like TransCelerate and Vivli.
The pandemic has also substantially changed the experience of trial participation for enrolled patients. Human-centered trials are oriented toward offering value to the participant, resulting in experiences that are more personalized, seamless, and information-rich. Seamless experiences require striking a balance between human connection and wise application of technology — but what happens when we have new limits placed on close human interaction? Increased uptake in telemedicine and remote care during the pandemic has offered new flexibility to patients and will have a lasting impact on patients’ expectations of possible care experiences, extending to clinical trial participation. Sponsors have also looked to virtual trial options to ensure continuity during the pandemic and have managed to demonstrate real-world feasibility of remote data collection, remote patient contact, and even shipping clinical trial supplies directly to homes. Following this progress, the #nogoingback movement was created specifically to acknowledge strides made in clinical trial conduct; a variety of industry, CRO, site, and tech vendor organizations have already signed the pledge.
Meanwhile, as COVID-19 gathered steam and social justice issues garnered increased public attention, a new spotlight has been placed on underrepresentation of minority groups in clinical trials. Human-centered objectives of personalization and information-rich experiences with clinical trials require that clinical research appropriately represents the populations in which new therapies are intended to be used. Particularly for groups disproportionately impacted by a disease, as was evidenced with the black community for COVID-19, greater attention is being paid to trial representation gaps that have long existed but with little traction to address systemically in the past. The FDA recently issued new guidance to enhance diversity in clinical trials, PhRMA launched its first ever industry-wide principles on clinical trial diversity, and companies BMS and Johnson & Johnson are each investing $100 million toward trial diversification and health equity efforts. The events of this year may be enough to spur a tipping point for change that transforms the representativeness of trials for years to come, basing enrollment targets on real-world burden of disease across sub-populations.
As trials have become more open and human-centered in 2020, meager progress has occurred in making clinical trials and research results better integrated in the healthcare ecosystem, understood, and applied. Many in the public have been hungry for trusted information on the symptoms, spread, and progression of COVID-19. And while the media has paid more attention to the drug development pipeline in 2020 than it ever has in recent decades, common myths about clinical trials still persist and general public understanding of the high bar of evidence required for new therapies does not seem to have improved. The Center for Information and Study on Clinical Research Participation (CISCRP) fielded its annual survey in June/July and compared to the same results one year prior, clinical trial literacy and trust declined dramatically in unprecedented amounts (for example, willingness to participate in a trial shifted from 85% in 2019 to 49% in 2020). It is possible a void of sources for clear and plain-language information on clinical trials, the research process, peer-review standards, and the like has paved the way for negative perceptions. Trust in clinical trials and drug development appears to be waning, not unlike a decline in public confidence in institutions.
How Can R&D Leaders Be Prepared To Lead And Act Moving Forward?
End Consumers as a Focal Point
Understanding the preferences, information needs, and outcomes desired by end consumers is more critical than ever to navigate the changing landscape of clinical research and development. Though dispelling myths and misunderstandings about research and drug development has proven challenging throughout COVID-19 and a chaotic political environment, industry has already taken a leadership role in fostering trust. In a rare public statement made in September, a group of nine pharmaceutical companies made a collective commitment to “make the safety and well-being of vaccinated individuals our top priority.” It is alarming that this point required reassurance, but the action was a small and meaningful step toward creating greater familiarity between the industry and the end consumers it serves. A chief lesson from 2020 may be a renewed appreciation by industry of reaching consumers as people, like other sectors’ track record in developing intimacy with customers. Vast opportunity exists for R&D leaders to play a more prominent role in the public’s information landscape for clinical research and drug development, including bolstering efforts to build trust with communities that are underrepresented in trials.
Likewise, R&D leaders have a role to play in meeting goals for personal health, wellness, and prevention. It is possible COVID-19 will leave a lasting impression on consumers’ expectations for fast, reliable diagnostics that serve to protect themselves, their families, and their local community. Consumers increasingly expect intuitive tools and mobile apps that help them track or predict the onset of symptoms. Treating and preventing disease alone is now only part of the battle; supporting the more holistic health and wellness journey before, during, and after a diagnosis will separate industry leaders from the pack.
Innovation Speed and Agility
In our 2019 predictions on the future of clinical research, we emphasized the importance of organizational agility to innovate with speed and drive transformation at pace. This year has demonstrated that industry can adapt rapidly and let go of perceived barriers to accelerating clinical trials, such as geographic location or the confidentiality of trial data. With the backing of the COVID R&D Alliance, 20 companies committed to sharing data from COVID-19 trials with each other through the TransCelerate consortium, which designed and launched a new COVID-19 data-sharing solution in a matter of weeks. Companies are increasingly capitalizing on rich clinical trial data from their competitors to become better, faster learners and inform design of trials in their own development pipelines.
Another chief lesson from 2020 may be a redefinition of what agility means for the industry, viewed now in terms of preparedness to respond to a variety of “what if” scenarios, including global risks to public health. These “black swan” scenarios can disrupt the normal course of business or strategic priorities. Furthermore, agility can no longer be viewed solely in terms of an organization’s own approach, but in its ability to move with speed in collaboration and partnership with others, including competitors. Organizational agility must translate to sector-wide agility not only in the time of a pandemic — in honor of the progress and collaboration fomented this year, the industry can work in greater partnership as a sector within many other areas that continue to have high unmet need, such as the diverse and fragmented territory of rare diseases.
Cooperative, Systemic Transformation
Just as one vaccine alone will not be enough to address the COVID-19 pandemic, one company alone will not systemically transform clinical research and drug development. 2020 has given this call to action a renewed focus while also magnifying critical gaps (e.g., diversity in trials, cross-industry coordination). Industry is still learning and adapting to a new frontier of “coopetition” and open sharing across research networks and centralized platforms. To make continued progress, industry must continue to collaborate with regulators as a united front to guide new policy. Companies must work more efficiently with research partners to more quickly activate adaptive platform trials with master protocols. Competitors must share information more broadly to minimize unnecessary duplication and failures across the sector. And stakeholders across the health and life sciences ecosystem must cooperate to build more sophisticated standards-based networks that enable fluid movement of data and power the next generation of clinical trials.
R&D leaders rose to the challenge to address the pandemic with a mindset that “we are all in this together.” Going forward, we shouldn’t wait for a global crisis to justify increasingly open collaboration on shared goals. Sustained future innovation requires collaboration. For improved health, well-being, and a positive human future, R&D leaders must carry this torch into the next decade.
About the Author:
Kelsey Jakee is a life science expert at PA Consulting. She is experienced in R&D strategy and transformation, patient-centered drug development, and operating model design. Connect with her on LinkedIn.