Policy Summit

Engage Patients Early to Ensure Successful Therapeutics

Rachel Salzman, DVM - October 05, 2021

Increasing patient input will contribute to developing and delivering transformative medicines to benefit all of society, says Rachel Salzman, DVM.

“Patient centricity” and “incorporating the patient voice” are phrases that we hear more and more often in gene therapy development, and with good reason—there is significant meaning behind them that should be associated with calls to action within the ASGCT community. During the ASGCT Policy Summit, speakers during one patient-focused session emphasized the need for drug developers to engage patients in the earliest stages of product conception.

The session “Prioritizing Patients in Gene Therapy Development” included two speakers representing disease-specific patient advocacies: Erin Ward, president at MTM-CMN (Myotubular and Centronuclear Myopathy) Family Connection, and Pat Furlong, founding president and CEO at Parent Project Muscular Dystrophy. The guiding principles and key learnings Ward and Furlong shared with the audience were highly relevant across all areas of genetic and cellular therapies, ranging from early stages through late-phase clinical development.

True patient engagement is not always as easy as it sounds,” Ward pointed out. She explained that sponsors should be committed to transparent bi-directional communication, which includes a commitment to sharing and learning “even when things go wrong.” The patient journey begins well in advance of clinical trial enrollment, and thus drug developers should be mindful to engage patients and their representatives in clinical trial design from the earliest stages of product conception. Doing so would include discussions around determining meaningful endpoints and outcome measures and inclusion/exclusion criteria. Regarding the challenges of the latter, Furlong observed that there are now five companies pursuing gene therapy for DMD, which is an exciting development for the patient community; however, she also pointed out that most of those companies are limiting enrollment to four to seven-year-old patients, which unfortunately excludes the majority of adolescent and young adult patient populations. 

In terms of pending legislative matters, Ward brought up the BENEFIT Act, which would amend the Food, Drug and Cosmetic Act to ensure that the patient experience, patient-focused drug development (PFDD), and related data be considered as part of the risk-benefit assessment. This would include information developed by a product sponsor or a third party, such as a patient advocacy organization or academic institution.  Furlong’s organization, PPMD, is another primary supporter of the bill.

In the session’s last presentation, Benjamin Forred, director of clinical research for the Coordination of Rare Diseases at Sanford (CoRDS) Project, shared information about Sanford’s patient registry. CoRDS is a no-cost, rare disease-agnostic, and patient-reported resource. Patients, caregivers, and close relatives can also upload information, which can ultimately better capture the broad burden of serious rare diseases. As an important note to researchers, CoRDS has an IRB-approved protocol and is considered a clinical trial. Furthermore, to support the pre-competitive space and advance therapy development in rare and underserved populations, the CoRDS registry is highly accessible. Participants can enroll for free, and researchers can access it for no charge as well.

The panel discussion, moderated by Lesha Shah, MD, assistant professor at Icahn School of Medicine at Mount Sinai, closed with multi-stakeholder acknowledgement of both the upcoming challenges and opportunities associated with bringing complex treatments, such as gene therapies, to people living with serious unmet medical needs. Additional policies can be put into place to spur progress, and success will ultimately lie in transparent communication supported by empowered commitments. Increasing patient input will contribute to developing and delivering transformative medicines to benefit all of society.

Registered Policy Summit attendees can watch all the sessions on demand on the virtual platform through October 24. Still want to register? You can do so here.

Dr. Salzman is EVP, portfolio, external affairs and development, at Alcyone Therapeutics and an ASGCT member.

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