How COVID-19 Renews Clinical Trial Vigor for Patients and Their Advocates

Deborah Collyar
Patient Advocates In Research (PAIR)
Amy Leitman
Nontuberculous Mycobacteria Info and Research (NTMir)

nfortunately, the already complicated lives of existing patients have become more so. While people around the world grapple with new realities from COVID-19, millions of patients with comorbidities, weakened immune systems, or long-term side effects from prior treatments are considered at high risk for infections and complications and continue to deal with daily dilemmas that have no right answer. This “new normal” concept, which now describes how society will function post-COVID-19, is common for many patients: Their “new normal” began the moment they were diagnosed.

But the pandemic also presents an opportunity. For example, the current rapid evolution of clinical trials for COVID-19 therapeutics and vaccines enables us to evaluate new design elements and to determine which of these new elements can be implemented permanently to both improve the clinical trial experience for patients and help streamline the development processes. For example:

  • COVID-19 has prompted many modifications to keep clinical trials operating.
  • Many of these changes help reduce patient burden and can increase receptivity to clinical trial participation in the future.
  • Patient advocates want key clinical trial modifications caused by COVID-19 to become permanent and are ready to help make that happen.

Clinical Trials Learn to Adapt

The clinical trials system has adapted at unprecedented speed to deal with COVID-19 and prevent clinical trials from grinding to a halt. Many of these innovations also benefit patients in other areas of clinical research and treatment. Because of this, patient advocates are working to make these changes permanent when they better meet patient needs.

Shortcuts to accelerate trials do not always translate to better patient results, however, and patients must be part of endpoint discussions that determine what will be studied. This is also a time when patient experiences and Patient-Reported Outcomes (PROs) must be carefully recorded in clinical trials to learn more about important information that leads to critical epidemiological and other factors to fight COVID-19. This includes various details about patients and how they feel about their symptoms, recovery, and long-term repercussions. Adding patient-focused endpoints also becomes easier when digitally facilitated (e.g., telehealth) which can lower data capture costs while making future trial more relevant and important to patients than what is often measured with traditional methods.

Patient engagement—from development through regulatory approval—can make or break a clinical trial. Too often, trial design does not take into account the patient’s burden when dealing with medical conditions and diseases, and this lack often progresses into the clinical setting after commercialization.

What Do Patients Want?

While different patient communities have specific needs, they all want more effective therapies (not just more drugs) and better therapeutic development. Given the large amount of real-time data being collected in COVID-19 trials, it would be a shame to miss the opportunity to develop more patient-focused drug development (PFDD) and clinical trials, both for COVID-19 and other medical conditions.

Patients want convenience and ease of use while participating in a clinical trial. They also want researchers to share data to learn what works best as well as the ability to share their own data in case they need to enroll in another clinical trial. Mitigated side effects are also paramount, so they can make good decisions based on real tradeoffs that impact their lives.

Specific System Improvements Need to Stay, For the Patients’ Sake

Current trial modifications can be harnessed to streamline clinical trials, improve the patient experience during trial participation, and foster receptivity for future studies.

Many clinical trial sites include major institutions, often referred to as “centers of excellence.” For patients who do not receive treatment there, this creates additional doctors’ visits for various procedures, tests, and labs. Decentralizing clinical trial sites by enlisting more community physicians helps relieve burden, making it easier for patients and sites to participate in clinical trials.

Adaptive clinical trial design is a key element that has lagged trial advances for various disease states, even though evidence has proven its effectiveness since 1969. Adaptive clinical trials often help fill knowledge gaps, such as determining at an earlier phase which groups of patients might actually benefit from an investigational agent. Patient communities are also highly receptive to carefully planned adaptive trials that learn in real time and adapt based on that knowledge. Platform trials are also more appealing to patients who must make decisions no matter how much uncertainty exists about treatment; these patients prefer more choices than traditional randomization gives them.

The COVID-19 pandemic has also brought the paucity of available technology in medicine and clinical trials into stark relief. Virtual visits and home delivery of medications, devices, and supplies have become the norm and should remain a first-line option in a post-COVID-19 world. This is critical as technology evolves at near light speed, which can offer more benefits to patients and real time data, as long as patient needs, issues, and preferences are also accounted for in clinical trial designs. Again, it becomes more economically sound to include patient experience data in every clinical trial with technological tools that are now accepted, thanks to COVID-19.

By combining these elements with other changes such as technology-ready home infusion and off-site monitoring, we potentially make clinical trials faster and safer for patients by reducing their risk of exposure to infection in medical settings.

From Alpha to Omega: What Really Matters to Patients?

Engaging patients in clinical trial design from start to finish means the clinical trial can focus on what really matters to the ultimate end user. What do end users want in a therapy? What changes should be measured during the clinical trial to see if patients get improved health and function?

Endpoints often focus on a “cure” or treatment, whether or not the patient survives. These do not meet patients’ needs, yet many fields continue to design trials this way. Instead, patients want endpoints that help measure a longer life expectancy or better quality of life. Identifying tradeoffs of side effects in exchange for a therapy that, while imperfect, may lead to some improvement is also critical for reimbursement as well as better patient communication and care.

Tradeoffs are questions only patients can answer, and to obtain these answers the use of PRO tools need to be better integrated into clinical trials. Having these data and properly analyzing them will give a greater understanding of results that patients seek.

Let’s Make it Happen Together

The time to “get real” is now—made all the more urgent by the fact that clinical trial participants may be barred from visiting trial sites even though they wish to continue. We all need to find creative ways to make that happen.

It is heartening to see nascent discussions on these points. We’ll need many more once the focus shifts slightly from the current COVID-19 crisis. Patient advocates stand ready to help build solutions. Now is the time to plan with trial sponsors, investigators, providers, patients, regulators, and payers. Together, we can turn regulatory mandates such as PFDD from FDA into more than a checklist. Let’s actually create a clinical trial environment that improve people’s lives for both efficacy AND safety. This is what patients want.