Leveraging Patient FDA Engagement to Increase Your Chances of Approval

rare disease research

Patients are the backbone of clinical trials, playing an essential role in the drug development process. However, patients also play a vital role in engaging directly with the FDA. This engagement is often less understood and is underutilized by sponsors, meaning a significant element of the trial and drug experience is missed during sponsor engagement with the FDA.

To spur more frequent engagement between patients, sponsors, and regulators, Richie Kahn and Jenn McNary co-founded Canary Advisors, a patient engagement consultancy that works to better align development programs with patient needs.

A public health professional by training, Kahn has spent the last 13 years working to reduce the time it takes to bring promising, new treatments to the patients that need them most. Back in 2019, he was working in full-service ophthalmology when he learned he was going blind due to a rare and orphan disease called Wolfram syndrome. He and McNary began working together several months later.

McNary, who has three children with rare diseases, is perhaps best known as one of the moms who single-mindedly lobbied politicians and the FDA for years in support of children with Duchenne muscular dystrophy, ushering in the current age of patient advocacy.

Earlier this year, McNary participated in a panel discussion at a rare disease conference that focused on the importance of including patients during FDA engagement. Her fellow panelists represented a diverse group of stakeholders across clinical research, including patient advocates, sponsors, sites, and the FDA itself.

During the discussion, a biopharma CEO panelist shared his opinion on patient involvement in the FDA engagement process, stating that it can create problems for sponsors. He said that it should only occur with heavy coaching and once their objectives have been aligned with the sponsor’s established goals.

However, patient involvement can provide innumerable benefits to the sponsors, helping them to create well-designed and strongly executed trials. “When sponsors focus on learning about the unique experience of the patient and their disease, as well as their experience with the tested drug versus the general data, they gain meaningful insights,” McNary says. “The utility of data that patients bring to the table based on their lived experience can’t be overstated.”

How the Patient’s Role in FDA Engagement Benefits Sponsors

When the FDA, patient community, and sponsors can successfully collaborate, an investigational product’s chances of receiving regulatory approval and gaining market acceptance may improve.

“We focus on patients as equal stakeholders who need to be involved in discussions early on in the development process to develop a path forward together,” McNary says. “Getting early and ongoing buy-in prevents miscommunications later in development. It also means sponsors are less likely to waste time and resources developing programs that don’t work for patients.”

Patients included in FDA discussions can convey the value your compound has to the patient community and its impact on the lived experiences of those with the disease. It can also provide a chance for the FDA to better understand how the market will react if the drug were to be approved and if the patient community would welcome the approval.

“When designing patient-focused trials, sometimes you’ll make decisions that work best for the patient, and having the patient at your FDA engagement meetings can validate why you made the decisions you did,” McNary explains.

Ways Patients Can Engage with the FDA

The FDA offers numerous patient engagement methods, and both sponsors and patient advocates need to understand the rules associated with the different types of feedback.

“There are as many ways to engage with the FDA as there are people who want to engage, and we have trained many patient advocates to attend sponsor meetings and provide effective feedback,” McNary says. “But what you can’t do is have a sponsor telling patients and patient advocacy organizations what the messaging is and writing them a script.”

Kahn agrees. “Much of the work we do at Canary is focused on ensuring that patients can clearly and authentically communicate their reality, what matters most to them, and speak to topics FDA wants to hear about,” he says.

Patients should be encouraged to be honest and open during FDA engagement. If a program has considered the patients’ needs and engaged with the community appropriately throughout the development process, then the program will align with community and patient expectations, eliminating the potential of unexpected or negative feedback.

The FDA has three primary mechanisms for hearing from patients:

  • Written feedback: Patients and members of the public may independently send in commentary about a particular drug or study but will not receive any reply as the FDA is not authorized to engage directly with patients regarding specific studies or drugs. This feedback is unsolicited, and sponsors will not be involved in this feedback, but the FDA can consider this feedback when evaluating a program.
  • Sponsor discussion: Sponsors can invite patients and other guests to sit down with the FDA for a give-and-take conversation about a specific drug or study. This avenue is the only way sponsors can directly solicit the patient’s experience for FDA engagement.
  • Advisory committee public hearing: These public hearings are held when the FDA reviews a specific drug for potential approval. Patients and sponsors can attend and engage openly in discussion.

How to Ensure Your Trial Has Patient Support

Ultimately, positive patient feedback to the FDA comes down to designing a program that works for your patients, starting from the very beginning of your trial.

Canary Advisors recommends including patient advisory boards, which a sponsor, CRO, or patient advocacy organization can create in the early stages of clinical development. Kahn explains these in-depth: “Before you have a clinical trial design finalized or sometimes before indication selection, you want to meet with patients,” he says. “This is done in a regimented, structured way to ensure patients, as well as the organization sponsoring the patient advisory board, receive maximum benefit. A minimum of two-thirds of the time is spent hearing directly from patients, learning more about their unmet needs, preferences, and quality of life impacts.”

Not only can the patient advisory board help sponsors determine how to design a trial appropriate for the patients in the given indication, but it can also allow them to use their time and resources more effectively throughout the entire process.

“You can convene this patient advisory board at multiple time points throughout clinical development,” Kahn says. “It can start in preclinical while you’re working on indication selection, but you can meet again to talk about clinical trial design, endpoints of key interest, and perhaps even engage in patient experience data collection to learn how trial participation impacts how patients and their families feel and function.”

Engaging with Canary Advisors

Based in Durham, NC, in the heart of the famed Research Triangle, Canary Advisors serves biopharmaceutical, nonprofit, and regulatory organizations, helping them to better understand disease impacts, unmet needs, and patient preferences to inform drug and device development.

“Our mission is a lofty one. We’re working to change the whole drug development paradigm,” Kahn says. “We’re looking to change the belief that things are done to patients. Instead, we dream of a future where patients are truly seen as valued collaborators and co-creators.”

Canary Advisors’ services include:

  • Early-stage patient advocacy, such as building out patient advocacy strategy or processes for engagement
  • Clinical trial patient support and white glove patient concierge services
  • Regulatory patient advocacy, like externally led patient-focused drug development meetings and Hill Day work
  • Patient experience data collection to support regulatory submissions
  • Working to ensure that access and reimbursement are never a source of delay

With more compounds being discovered every day, incorporating the patient perspective could be the deciding factor in whether your development program succeeds or fails. Let’s talk about how Worldwide can help your rare disease program succeed by partnering with patient advocacy.

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