The global Covid-19 pandemic has left families and communities around the globe grieving for lost loved ones, exhausted from prolonged social isolation, and anxious about their financial future as unemployment reaches levels not seen since the Great Depression.
But it is also driving an unprecedented transformation of the global medical research ecosystem through the search for effective new therapies and vaccines that can help ease symptoms and prevent death among people with Covid-19, restart economies, and ultimately protect people from the disease.
As the first truly global public health crisis of the internet era, cutting-edge medical research and data are being shared and analyzed in near real time, which will help slash drug and vaccine development timelines. With some luck, over the next 12 to 18 months we will see more repurposed antivirals like remdesivir (Gilead’s formerly Ebola-focused drug) and potentially antibody drugs and vaccine candidates promising enough for emergency use in health care workers even as late-stage clinical trials are ongoing.
There is no silver lining to this pandemic, which has cast a pall over much of the world. It has, however, sparked a global commitment by the research community that is accelerating many promising trends in biotechnology that will not only help us prevail against Covid-19 but turn the tide against other life-threatening illnesses that annually claim the lives of millions of people in the United States and around the world.
This acceleration is fueled by international regulators, companies, academic researchers, hospitals, and others collaborating to break down silos between medical research and patient care. They are creating an adaptive research network that can harness the latest findings from patients treated in frontline settings and use that experience to help select and test the most promising new therapies and vaccines in clinical trials.
Three major tools in this effort are modernized regulatory science, digital medicine technologies, and adaptive clinical trial designs. Together, these approaches can dramatically shorten the time and cost needed to generate the essential safety and effectiveness data that regulators need to review and approve new medicines and diagnostics.
The first tool, modernized regulatory science, is being adopted by regulators around the world. In the U.S. over the last few months, the Food and Drug Administration has released in record time vital new guidance for drug developers. This guidance, which normally takes the FDA years to issue and update, contains critical features for conducting clinical trials and good clinical practice for investigational drugs.
The FDA has also launched the Coronavirus Treatment Acceleration Program, an expedited program for drug development. It can generate significant efficiencies by coordinating senior agency staff and expertise from multiple drug review divisions to give sponsors rapid advice on how best to test and develop promising new therapies.
Digital medicine technologies represent a second vital tool. Digital tools are helping advance medical research when many patients can’t go to a physician’s office or hospital for even routine assessments. For instance, telemedicine visits using tablets and smartphones make it possible to remotely monitor patients, reducing the risk of patients and physicians’ being exposed to coronavirus in health care settings while also reserving scarce personal protective equipment for higher-risk settings.
Collecting data via electronic health records, wearable sensors, and apps was already occurring before the emergence of the coronavirus pandemic, but trial sponsors, clinicians, and regulators are enthusiastically embracing digital strategies to help streamline trials and collect more information about how patients feel and function day to day. Additional recent federal regulation on interoperability for health data will strengthen efforts to integrate real-world data into drug development and regulatory decision making.
Third, regulators, researchers, and industry are embracing new master protocol trials to simultaneously test multiple drugs or vaccines using shared clinical trial protocols and common control arms. Master protocol trials, especially those with adaptive or learn-as-you-go elements, can add promising new treatments or drop those that miss key endpoints as the trial progresses, shifting patients to treatments that are more likely to be successful based on evolving evidence and sophisticated statistical designs.
These trials are key to unlocking the full potential of precision medicine by testing targeted drugs in specific patient populations to see what works in a learning-while-doing environment. More than a half-dozen master protocol trials are underway testing coronavirus drugs and vaccines. The National Institutes of Health and nearly 20 drug companies formed a public-private partnership called Accelerating Covid-19 Therapeutic Interventions and Vaccines (ACTIV) that includes scientists from the U.S. Food and Drug Administration, the European Medicines Agency, the Centers for Disease Control and Prevention, and the U.S. Department of Defense to expedite testing of the most promising candidates and share real-time clinical data. On May 18, ACTIV announced it would be moving six repurposed drug therapies into master protocols by the end of May.
The Covid-19 crisis is testing us and will continue to test us. In response, the scientific community is rallying as we’ve never seen before to find new therapies. And as its efforts unfold, it is making the future of medicine a reality today.
To paraphrase Winston Churchill as he addressed the House of Commons in 1940 not long after the British disaster at Dunkirk when Britons were losing hope: Let us brace ourselves to our duties and harness science to defeat this pandemic in a way that people will still say in a hundred years or a thousand that “This was their finest hour.”
John F. Crowley is the chairman and CEO of Amicus Therapeutics. Kush M. Parmar is a managing partner at 5AM Ventures, one of the founding partners of the COVID R&D partnership.
