Ringing in the New Year with decentralized clinical trials

Pamela Tenaerts, M.D., Chief Scientific Officer, Medable

Prediction 1: In a year, evidence will replace hypotheses in decentralized clinical trials (DCTs).

By the end of 2024, the lifesciences industry will replace hypothetical surveys about the impact of decentralized methodologies in clinical research with hard evidence. The Tufts Center for the Study of Drug Development has formed a pre-competitive consortium of more than 50 companies that will collate metrics to understand how decentralization impacts protocol performance such as cycle times, patient recruitment, and retention rates. The project was funded by the Reagan-Udall Foundation for the Food and Drug Administration (FDA) and Medable and will be a multi-year effort, with the goal to publish its first data set by spring of 2024.

The consortium will conduct a granular analysis of actual data to gain a better understanding of the impact of specific clinical trial innovations to better inform future protocol design. With this information, the industry can take a more fit-for-purpose approach for determining how to incorporate decentralized elements into more trials. During COVID, we tried everything. Now, with more evidence, we can get better at matching decentralized innovations to populations, diseases, and specific study designs. This could have the add-on effect of decreasing complexity. For example, rather than trying to implement eCOAs, wearables, and home health nurses in the same trial (which may be overkill), we can carefully pre-plan to use just the technologies that are most likely to improve outcomes.

As we continue to capture evidence of DCT value, we will increasingly adopt a nuanced approach, but this progression will move at the pace of maturity of each DCT innovation.

Prediction 2: The acronym “DCT” will die away as decentralization becomes the norm.

By the end of 2024, the lifesciences industry won’t distinguish DCTs from simply clinical trials anymore. As noted by the FDA, the modernization of clinical trials is an ongoing evolution. In the United States, it will require an act of Congress to change the definition because the term DCT is used in provisions published by the Food and Drug Omnibus Reform Act of 2022 (FDORA) that amended the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act. Other regulators are already talking about trials with decentralized elements (i.e., EMA’s recommendations paper) rather than DCTs.

Even with existing regulatory nomenclature, the acronym DCT will become less of a call-out as a distinct type of unique clinical trial – and that is a step in the right direction. The term suggests that a decentralized trial is uncommon and requires special extra adjustments, but that is not true. Recent FDA and other global regulatory agency guidances explicitly note that decentralized trials simply need to comply with existing regulations for clinical trials – nothing extraordinary. There may be some data quality, privacy, and security considerations, but the existing playbook still applies to trials leveraging decentralized innovations.

By this time next year, decentralized clinical trials will simply be clinical trials (with certain parts appropriately decentralized) and the impact of this model will be much more prevalent.

 

Alison Holland, Executive GM of Customer Value, Medable

Prediction 1: We will stop focusing on the “what” of decentralized trials and focus on the “how.”

Over the last 18 months, the industry has spent a lot of time trying to define decentralized clinical trials and debating what a DCT consists of but now we are focused on outcomes. DCT methodologies give us more options around evidence collection, opportunities for data capture in real time – all with greater integrity and less friction for the patient. Now, as we ring in a new year, we are simply working to decide what elements should be decentralized to get the desired outcome. With more choices about how to package a clinical trial ecosystem, we can make research more of a consumer-like process.

Prediction #2: Participation diversity in clinical research will become a bigger priority.

For 2024 and beyond, there will be an increase in focus on patient diversity in clinical trials. The difficulties of a lack of patient representation in clinical research bubbled up to the surface to a raging boil during the pandemic’s vaccine trials and that issue isn’t going away. Some of the approaches may change from what we have learned – for instance, there is far more capacity now to connect with broader patient populations using new digital technologies. Given the convenience of these tools, more patients who become increasingly comfortable with technology will take advantage of digital opportunities to connect to a trial and remain engaged through until the end. This will also help ensure patient compliance for the full duration of a trial even when patience and perseverance start to wane in the later weeks, making it harder to capture consistent data.

Prediction #3: A competitive pharma marketplace will compel increased trial innovation.

The pharmaceutical marketplace is more competitive than ever, particularly in the race to be first-to-market. For instance, the time between the first FDA approval for a vaccine for the respiratory ailment RSV to the fourth FDA approval for a similar vaccine was less than six weeks. The first-mover advantage is dramatic, so clinical trial sponsors are hyper-focused on making sure they are maximizing every second and not wasting time. And, to that end, large pharmaceutical companies are setting lofty goals of 50 percent reductions in cycle time next year – meaning, the status quo won’t allow them to reach such significant new goals. Sponsors will need to make commensurate changes to accelerate drug development.

The DCT methodologies will now be part of the standard toolkit, embedded into most trial processes and operational decisions because sponsors need new ways to engage with patients in a competitive marketplace and collect data reliably. In 2024, the shift to DCT methodologies will be transformative and wholesale, bringing DCT elements into the organization at the portfolio level rather than trial by trial.

Here is why: decentralized methodologies allow for higher fidelity data collection and greater objectivity in signal detection, removing skews and biases. This enables a more informed understanding of an investigational medication’s safety and efficacy, which allows companies to reduce the sample size – and fewer participants means less patient recruitment time and lower costs. Roche reduced its sample size by 70 percent this way – helping to speed drug development and time to market.