By Amy McKee, Vice President Regulatory Consulting Services, Parexel
“Cancer did not pause with the rest of the country.” This poignant observation from Kristen Seamans, a patient representative for the rare-cancer advocacy group Sarah’s Cure, seems to capture the challenges facing patients, clinicians, drug developers and regulators alike during this unprecedented crisis.
Indeed, how has the pandemic affected patients in the search for treatments for rare disease and cancer? How have clinical trials and regulatory activities been impacted? What have we learned about trial conduct, patient recruitment, risk mitigation and the pace of regulatory approvals? How are patients coping? This article outlines some of the findings from a recent virtual discussion with Parexel experts and representatives from the patient advocacy group, Sara’s Cure.
Sponsors: Pausing, modifying and continuing clinical trials
As the pandemic forced shutdowns in every corner of the globe, some clinical trials went into a temporary holding pattern, including both pausing trial enrollment and suspending clinic visits. Within a matter of weeks, though, sponsors had re-tooled and were finding new and innovative ways to ensure safety, mitigate risk for patients, and identify sites in areas where there is less transmission of the virus. Most of the trials involving rare disease and oncology that were underway continued uninterrupted. Sponsors and sites quickly implemented key adjustments, reducing in-person participation and conducting treatment remotely wherever possible.
These changes have raised many questions about modifying trial conduct and gathering evidence in ways that are acceptable for regulatory submissions, which the regulators have diligently endeavored to answer. In fact, the Federal Drug Administration (FDA) and its global counterparts quite clearly understand the immense challenges that sponsors and patients are grappling with, and have proactively issued guidance since the early days of the pandemic. The FDA’s Oncology Center of Excellence (OCE) published “A Message to Patients with Cancer and Health Care Providers About COVID-19,” stating unequivocally that the agency is doing its utmost to address critical issues and identify priorities.
Regulators: Modifying guidance, ready to be flexible
Our colleagues at global Health Authorities (HAs) are working as hard as they ever have, most in home offices just like the rest of us. There are no slowdowns; they conduct meetings online to expedite oncology product development and have approved multiple new therapies in the past few months alone. Research on using real-world evidence to inform decision-making is ongoing, with data gathered in the patient’s own environment ultimately promising to reduce the burden on sponsors, patients and sites. Health Authorities are striving to balance scientific rigor with flexibility. For example, some HAs adjusted compliance mandates so that scans don’t have to be conducted at the same site using the same techniques.
Patients: Adapting, adjusting and rising to the challenges
But what about the patients? How are they adapting to the many complications? Beyond the inherent uncertainties and anxieties of cancer treatment, they now must contend with sudden disruptions in travel, severe limits on participation by their families, inability to meet with their physicians in person – to name just a few of the adversities. Their experience in navigating the healthcare system has been difficult, to say the least.
In our view at Parexel, it is incumbent upon us, as a company running clinical trials, to be creative in figuring out ways to get patients treated in this environment. We need to partner with physicians, patients and patient advocacy groups – an underutilized resource – to learn how we can make it as easy as possible for them to participate in clinical trials, especially for rare diseases. What can sponsors do to get them to sites safely? What aspect of treatment can be done remotely to reduce the stress of traveling to the site?
The biggest challenge for sponsors is to determine where can they safely open trials and where patients will be willing to come in. But we hear from patient advocacy groups and from the patients themselves that they will do whatever it takes to continue their participation in the trials. We have seen that they bravely continue to rise to the challenges. They have confidence in us to keep drug development programs on track and meet regulators’ requirements despite the disruption and wait with great hope for treatment.
Parexel recently hosted a roundtable, “Patients Can’t Wait: Advancing Rare Disease and Oncology Drug Development During Covid-19,” that explored these issues from the perspective of Kristen Seamans and Lenny Woods of Sarah’s Cure. We invite you to watch it on-demand. Alternatively, read this short article as we answer some of the common questions about trial conduct, patient recruitment, risk mitigation, the pace of regulatory approvals, and how patients are faring in this space.