The “Five Big Shifts”​ Coming Next to Clinical Trials

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Many today are discussing how clinical trials will look differently after the pandemic. Innovative approaches that had been developed in recent years but were stalled in “experimentation” have been rapidly adopted over 2020 as pandemic counter-measures, and many organizations are now making commitments to embed these approaches into their clinical trial pipelines going forward.

These areas — telemedicine, decentralized trials, risk-based monitoring, remote monitoring, and others — were the “next big shifts” five to ten years ago. While it will take time and resources to fully embed and scale these approaches into development organizations, these are the solutions of today.

For those considering planning, strategy and investment, the question must then look beyond: Based on our experiences during 2020, what are the next big shifts coming to clinical trials?

Based on our experiences during 2020, what are the next big shifts coming to clinical trials?

 

1. Participant Experience Gets Personal

A majority of sponsors, CROs and sites have indicated that decentralized trials will be a part of their model for study conduct going forward, and 75% have indicated an expectation that trials will be “hybrid” with a blend of virtual in site-based approaches. Today’s hybrid trials, however, are still highly “protocol-centric” in dictating to the patient how and where their engagements will take place.

Looking ahead, research participants will have more choice and personalization in how they participate. As sponsors continue to invest in digital endpoints and other strategies that provide more confidence in reliable data regardless of location, participants will have more flexibility to adjust how they engage on a visit-by-visit basis based upon their own patient journey.

For some visits the patient may have other obligations in their life and find it impractical to travel to a site, opting instead for an encounter via video with remote data sharing. For other visits the patient may have a clinical concern or simply look forward to the high-touch engagement provided by study coordinators and site staff. This concept of personalization is hardly new — we are all experiencing such choice in how we engage in shopping, education, and healthcare today. Research participants will bring these new expectations as they engage in studies, and it is up to those leading trials to meet those expectations.

There will always be examples that are unique and require a visit, but the pandemic has shown us that in-person visits are not essential in a majority of instances. As sponsors build confidence with local labs, local imaging centers and more reliable endpoints, flexibility and personalization for visits to meet participant experience will continue to expand.

2. Site Technology Goes BYO

Investigator sites want to invest in technology, but struggle to recover their investments in the context of today’s multi-center trials. For example, most all clinical centers in 2020 have adopted some form of telehealth and video visits yet when they are participating in a sponsored trial the sponsor and CRO often push down their preferred video platform. Many view this as a necessity to ensure control and confidence in the systems being used, but these actions are creating an environment reminiscent of the days of sponsors delivering stacks of laptops at the doorstep of sites early in the adoption of electronic data capture.

Looking ahead, sponsors and CROs must look at site technology similar to how we look at -80° freezers. Research sponsors want the site to have a freezer, but we cannot all push preferred freezers down upon the site and expect them to embed these tools into their workflow and environment.

Instead we must rely upon baseline quality standards — what makes for a qualified and capable technology — and focus on interoperability of key operational or clinical data. Doing so will allow sites to invest in the technology that fits their workflow, improving quality through familiarity while allow sponsors to avoid redundant cost by paying for technology that already exists and only provisioning technology when needed by the site. From telehealth to eConsent to eSource, standards initiatives such as those being launched by IEEE will enable sites to “bring their own” (BYO) technology, recover investments, lower cost, and improve quality.

3. Equity Meets Commitment Through Incentives

Lack of representation in clinical trials is hardly a new revelation. Most every research sponsor has had workstreams and task forces through the years, and FDA has done their part by exposing diversity (or lack thereof) in posting Drug Trial Snapshots for approved medicines.

It has taken generations to embed the lack of trust that diverse and underserved communities hold for research participation, and it will take long-term commitment to make a meaningful change in making studies more inclusive. There are no silver bullets, and there will be no sustaining quick-fixes during the current heightened awareness and conversation of the need for representation in trials.

We have a proven model for making change when there have been important gaps in our research ecosystem. In years past when it was appreciate that orphan indications were not receiving adequate attention, legislation enabled the FDA to create incentive structures to fuel changes in medicine development by the private sector. The same use of “carrots and sticks” have changed pediatric drug development.

A similar path can be charted for embedding a commitment to diversity and inclusion in clinical trial. Looking forward, we can leverage similar proven incentive pathways to encourage regulatory submissions for new medicines to include studies powered to understand differences and outcomes for various subgroups (race/ethnicity, gender, or otherwise). The FDA’s system of incentives would create long-term commitments toward studies including representative patient populations, while providing providers and patients with vital data to have confidence in the potential for new medicines.

4. The Next Warp Speed

For years, leaders in the “C-Suite” at major pharmaceutical companies believed that research and development could go faster. CEOs and CFOs across the industry have looked toward their R&D chiefs pressing for more speed, often being countered that science and processes can only go so fast. The pace of medical breakthroughs during the pandemic has proven otherwise.

Through parallel processes and removing governance, initiatives such as Operation Warp Speed have proven that medicine development can make a step change in speed for an vital health crisis with significant unmet medical need.

It is not feasible to expect entire R&D organizations to shift to “Warp Speed” for their entire pipeline — such initiatives bring high execution risk, consume vast resources and would burn out most any engine.

But we should expect to bring similar Warp Speed urgency to therapeutic areas beyond COVID. Looking ahead, what will be our pathway for “Warp Speed: Alzheimer’s”, “Warp Speed: ALS”, and beyond?

Executives have seen that their organizations can make go exponentially faster, and we should expect to deploy that sense of urgency for other vital health needs.

5. Clinical Trials Meet the Shared Economy

We often think of the “shared economy” with examples such as AirBnB and Uber — instances where we do not own a capability but are able to access the capability from others based on our needs. Clinical trials have long been a shared economy — pharmaceutical companies rely upon a network of technology capabilities, research sites, and study participants that they do not own, employ or control.

A step change in the shared economy has been the adoption of master protocols, enabling more rational testing of new medicines with individual protocols exploring multiple indications or multiple interventions concurrently under a single study framework and platform. Examples have emerged from Oncology to Rare Diseases to Alzheimer’s, and the FDA has been extremely vocal in their support. Many have appreciated that master protocols were a sorely underutilized resource during the pandemic — while most medical learnings have come from COVID-19 master protocols such as SOLIDARITY and RECOVERY, the US has instead been lead by a cacophony of disconnected and often underpowered research studies competing for resources and patients.

We can look toward a different model by considering “all-comer big-data observational studies” such as the NIH’s All of Us or Google/Verily’s Project Baseline. These studies are open to most all Americans and are enrolling from tens of thousands to over a million participants, each sharing rich and diverse data spanning electronic health records, molecular profiles, self-reported, as well as sensor-derived data. That diversity of data often reflects the same or more breadth of data that we seek for any individual trial of a new medicine.

A future model on the horizon will lie at the convergence of master protocols and “all comer” studies and create a next generation of shared economy for clinical trials. Models that go beyond enrolling a patient into a single trial for one indication will evolve to simply invite patients to “participate in research”. These study experiences may begin as observational, but then invite individuals for different interventional arms based upon existing data. Rather than attempting to migrate the patient out of their existing study experience and shift them into a different and unfamiliar set of study tools, the research experience will remain centered around the patient and allow them to continue sharing rich data just as they did when the study was only observational. When their period on a new intervention is complete, the participant can continue to share observational data once again. Patient engagement and data capture tools will no longer need to be launched on a study-by-study basis, but instead can be more widespread and universal by simply adding custom questions based upon specific medical concerns.

By rapidly evolving to sustain clinical trials throughout the pandemic, those leading and executing medicine development have proven they can be resilient and adaptable. Committing to these changes in the coming months will continue to challenge organizations, but will be vital for business continuity, advancing science and meeting the needs of patients.

We must also look beyond the solutions at our fingertips — those that were developed but waiting for adoption — and look ahead to the next big shifts on the horizon so that our strategies and investments are pointing toward a future realizing our fullest potential in improving the delivery of new treatments and cures.

 

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