In 2020, life sciences manufacturers, providers, researchers, and other health care stakeholders renewed their interest and investment in clinical trial innovation. Spurred by limitations of Covid-19 shutdowns, many organizations had to quickly adapt and introduce telehealth, e-consent, direct-to-patient drug shipping, and remote monitoring to support ongoing trials. And clinical trial innovation companies, such as Science 37, VirTrial, and Medable, saw an explosion in interest and demand from their life sciences partners.
While these innovations were great to see, solutions like decentralized or virtual trials are not new. In fact, clinical trials have long been an area ripe for disruption. The challenges and inefficiencies with clinical research are well known; clinical trials are expensive, burdensome for patients and caregivers, and often fail to recruit a diverse set of trial participants. Life sciences companies have experimented with incorporating elements of virtual or decentralized trials into existing trial designs, but most efforts to innovate have been fragmented or have occurred on an ad-hoc basis.
So, if the challenges and solutions are well understood, what is standing in our way? To answer that question, we came up with a list of seven things we'd like to see that could truly drive transformational change in the clinical trial space.
What the health care industry must do to accelerate clinical trial innovation
1. Academic medical centers and providers become active proponents of virtual and decentralized clinical trials.
In the past year, life sciences companies and contract research organizations (CROs) have committed to advancing clinical trial innovation. Notably, the newly formed Decentralized Trials and Research Alliance (DTRA)—which aims to make clinical trial participation widely accessible by advancing policies, research practices and new technologies in decentralized trials—already has more than 50 members spanning life sciences companies and CROs.
In 2021, academic medical centers (AMCs) and providers must also embrace clinical trial innovation. Today, providers have expressed interest in decentralized and virtual trials, but it has not been top-of-mind—especially as they navigate other Covid-19 challenges. However, providers have already realized many benefits of decentralized trials throughout the pandemic. For example, many cancer trials were successful after having to quickly adapt decentralized protocols during the pandemic.
Decentralized and virtual trials will become increasingly important to AMCs and providers, especially for emerging pipeline treatments, such as gene therapy and rare disease drugs. Studies for these products will require providers and researchers to follow up with a small number of dispersed patients over a long period of time. Remote patient monitoring, telehealth, and other decentralized tools will be essential for trial continuation and success in the long term.
2. Advancements in digital data collection improve the validity and usefulness of digital endpoints used in decentralized and virtual clinical trials.
Over the last few years, life sciences companies and researchers have experimented with using wearable devices and digital tools to collect data during clinical trials. For example, Apple and Stanford Health's Heart Study used the Apple Watch to monitor and detect Atrial Fibrillation. Novartis launched the FocalView app to allow researchers to track ophthalmology disease progression using data collected from patients' smartphones. The Digital Medicine Society even created a Library of Digital Endpoints to crowdsource the number and kinds of digital endpoints used in clinical trials.
Despite the momentum toward digital data collection, few stakeholders have embraced a standard framework through which they can assess and validate digitally collected endpoints. However, many efforts are underway to regulate digital data collection and endpoint selection. For example, the Clinical Trials Transformation Initiative has developed a series of frameworks and pathways for developing novel endpoints from digital health technologies to improve clinical trials.
These frameworks are a key first step in advancing industry-wide clinical trial innovation. In 2021, researchers, trial sponsors, and regulatory groups must align on a standard framework to ensure the quality, consistency, and reliability of data generated through digital trials. This will enable industry leaders to embrace new types of data collection and to gain confidence generating evidence through innovative clinical trial designs.
3. FDA and regulatory agencies develop guidelines that encourage post-pandemic clinical trial innovation.
At the beginning of the pandemic, FDA issued new guidance for conducting clinical trials during Covid-19. This guidance allowed for flexibility in trial conduct during the pandemic and led to increased use of telehealth, e-consent, remote monitoring, and other tools for decentralized and virtual clinical trials.
FDA has already shown early signs of openness towards clinical trial innovation, and the agency recently published a guidance document on the conduct of complex innovative trial designs, such as master protocols. Next, FDA must capture lessons learned from decentralized and virtual trial design during the pandemic and establish guidelines that encourage their widespread use in the future.
4. The health care industry addresses technology disparities that currently mitigate remote trial accessibility.
Decentralized and virtual clinical trials can expand care to underserved populations, improve diversity and inclusion in trials, and advance health equity. And many industry leaders have already committed to improving diversity and inclusion in clinical trials. However, disparities over access to technology used in decentralized and virtual trials mitigate the potential for these goals to succeed.
In 2021, researchers, providers, life sciences companies, and other industry stakeholders must prioritize addressing underlying causes of trial participation disparities—such as insufficient access to the internet, technology, and transportation. This will enable clinical trial innovations to succeed and improve accessibility and health equity for all.
What life sciences companies must do to accelerate clinical trial innovation
5. Life sciences companies begin incorporating elements of virtual and decentralized trials into their existing clinical trials.
Clinical trials do not have to be entirely virtual or decentralized to be successful. Incremental changes or additions to existing trials, like virtual check-in visits, e-consent, direct-to-patient drug shipping, or remote patient monitoring, can add value to patients and providers and improve their experience with trials—without completely changing existing protocols.
By starting to innovate within their existing trial designs and testing new technology, life sciences companies can begin to gain comfort with decentralized and virtual trial and identify innovations that have the most impact.
6. Life sciences companies invest in proof-of-concept decentralized and virtual trials to build confidence in their applicability and use.
Launching a fully virtual or decentralized trial for a new drug or intervention may be risky for life sciences companies' pivotal, pre-launch clinical trials. However, life sciences companies can begin to invest in lower-stakes, proof-of-concept decentralized and virtual trials for Phase IV studies or utilize trial innovations to measure the efficacy of non-drug solutions—such as beyond the pill services or digital health apps.
Proof-of-concept studies can help life sciences leaders build comfort with new processes and protocols for data collection and patient communications and anticipate future roadblocks and obstacles in trial innovation. They can also help life sciences' provider and researcher partners acclimate to different kinds of virtual data collection and trial designs.
7. Medical affairs, market access, HEOR, commercial, and regulatory colleagues within life sciences companies work together to overcome internal roadblocks to trial innovation.
Company siloes have traditionally hindered clinical trial innovation and adoption of new trial designs. However, Covid-19 has demonstrated how internal siloes within life sciences companies can work together to innovate trials and overcome internal barriers.
At the beginning of the pandemic, some life sciences companies set up cross-functional SWAT teams to implement components of decentralized and virtual trials into trials impacted by the pandemic. Now, they are turning those SWAT teams into a formalized function,recognizing that different siloes within a life sciences organization can contribute to, and benefit from, clinical trial innovation.
For example, medical affairs and HEOR teams can identify post-launch evidence needs to inform endpoint selection and trial design and advance Phase IV/real-world evidence studies. Research and development (R&D) can utilize clinical trial innovation to improve the patient and provider experience in pre-launch clinical trials, increase diversity and representation in trials, and collect digital endpoints and patient-reported outcomes to strengthen regulatory submissions. And key accounts, commercial, and market access colleagues can use evidence generated in trials to strengthen relationships with key customers.
Interested in participating in Advisory Board's research on clinical trial innovation? Email Pam Divack (email@example.com) and Manasi Kapoor (firstname.lastname@example.org) to discuss.