What are the opportunities and challenges for the regenerative medicine field as the world recovers from COVID-19?

We have all been inundated by webinars, podcasts and Zoom calls focused on identifying and navigating COVID-19 challenges. Based on what we have learned from the first five months of shelter-in-place and quarantine, what are the opportunities that can benefit our patient community as it pertains to clinical trials?

This discussion focuses on the Rare Disease community as they comprise the majority of current cell and gene therapy clinical trials. COVID-19 has redefined how we engage and treat patients currently enrolled, or in the process of enrolling, in clinical trials. COVID-19 has called for small, mid and large Pharma, CROs and patients/caregivers to become agile and flexible in this unprecedented environment. The safety of patients, the quality and integrity of clinical trial data, and the timeliness of treatment options guide the work of all clinical trials. These trials have now been paused, even stopped, so protocols can be reviewed for viability in the COVID-19 environment.

To put this situation in perspective 44%, or over 2600 clinical trials, were stopped according to a May  18, 2020 article in Centerwatch. Trials that were not stopped experienced slow enrollment, cancellation of appointments by patients, site closures, and lack of investigator availability impacting clinical trials in a way never experienced before.  As an industry, we pivoted.  IQVIA reported on August 4, 2020 that the use of telemedicine in clinical trials increased from 59% to 80+% from April through June of 2020.  88% of patients accepted the telemedicine appointment option and 92% found the experience to their liking. Telemedicine was accompanied by home health visits for clinical trial continuation when patients were not comfortable or did not have the option of visiting the site for a protocol specific appointment.   Institutions that haven’t traditionally been open to remote review of electronic medical records (EMR) also pivoted to accept and implement remote processes to maintain clinical trial continuity.

This follows the FDA Guidance on Conduct of Clinical Trials of Medical Products During COVID-19 Pandemic. The agency provides a number of considerations for ongoing trials and recommends that sponsors evaluate alternative methods for participants when in-person visits are not available. 

COVID-19 pushed into the spotlight additional key areas worth considering for inclusion in clinical trials moving forward.  The COVID-19 burden will not lessen immediately, so it is timely to consider these alternatives. 

  1. Simplification of study protocols to streamline the studies.
  2. Decreasing the number of participants needed in the study.
  3. Decreasing the number of patients who are required to receive the placebo.
  4. Remote monitoring of patient data.
  5. The use of historical control data in clinical trials with rare disease indication.

Will our learning cause a shift to more patient-centered clinical trial designs and rewriting protocols to allow for remote patient monitoring accompanied by in-home delivery as cited above? We can’t answer that yet; however, I believe it is worth consideration.

The greatest learning from COVID-19 may be increased patient engagement outside of traditional clinical trial participation. Patients, families, clinicians, and Pharma have worked together to provide safe, efficacious, and acceptable protocols for continuation.  All efforts aim toward increasing approvals every year for rare disease therapies.  What a difference a few months can make for patients and their loved ones.

My blog is one of many covering this topic as part of Signal’s fifth annual blog carnival. Please click here to read the unique perspectives of other bloggers.

Susan B. Nichols

President and CEO
Falcon Therapeutics, Inc.
snichols@falcontherapeutics.com
falcontherapeutics.com

Chairman of the Board
Rare Disease Innovations Institute
snichols@rdii.org
RDII.org

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