Long-term, strategic engagement with patients and advocacy can then be harnessed to build a mutually beneficial relationship. Image credit: Shutterstock / PeopleImages.com – Yuri A.
As advocacy efforts bolster the need for patient-focused drug development, amidst regulatory encouragement, the value of listening to patients is being realised in a more actionable manner.
“You can paint a fuller picture by hearing directly from patients,” said Richie Kahn, co-founder and COO of Canary Advisors.
Kahn was speaking at the ongoing Outsourcing in Clinical Trials Southeast 2025 conference, being held 1–2 April in Cary, North Carolina. Kahn, who has participated in clinical trials, advocated for more people in the field to do the same to understand the patient perspective more accurately.
The sector’s reaction to patient-focused drug development (PFDD) has evolved as new regulations have come to the forefront, starting with the first guidance by the US Food and Drug Administration (FDA) on patient-focused drug development in 2011. The 21st Century Cares Act went a step further and through the 3002 and 3004 mechanisms, outlined how the agency would use the patient experience data, and report on the use of patient experience data in regulatory decision-making.
This guidance, similar to many issued by the FDA, however, is not prescriptive, said Kahn. In his role at Canary, he partners with a variety of stakeholders to ensure better alignment between clinical development and patient needs. Patient engagement does not need to be limited to surveys or listening sessions, but can occur through other mediums such as focus groups, advisory boards, patient-focused drug development meetings, and more, said Kahn. The advisory boards are set up to gather experience information.
Across all these mediums though, data should be collected from all patients, and Kahn noted the importance of not cherry-picking cases and cautioned against embellishing or over-emphasising results.
Why early patient engagement matters
While much of the public discourse has been centred on the role of patient voices in ensuring access following a drug’s approval, Kahn noted the value of patient-focused strategies much earlier in development.
For example, if a biotech has promising preclinical data but a finite amount of capital, such data can help in tasks such as indication selection, said Kahn. Long-term, strategic engagement with patients and advocacy can then be harnessed to build a mutually beneficial relationship, he explained.
Information collected from these patient engagement avenues can be varied. Kahn is also a visually impaired clinical researcher by trade, who has also become a patient advocate. He talked about pairing the quantitative with the qualitative by talking to patients and understanding what they need. Restoration of vision for patients suffering from vision loss may still be a goal that is several years away from clinical research. However, Kahn gave the example of a patient who wanted his vision loss to be stabilised enough to do certain tasks.
Even post-approval, there is an opportunity for patients and families to speak to drug review boards.
“Those with first-hand experience often have a fervent desire to help others get access to those treatments,” said Kahn.
Last year, the patient community rallied to prepare for the FDA Advisory Committee meeting held to discuss Stealth Therapeutics’ drug elamipretide for the ultra-rare disorder Barth syndrome. The committee eventually voted 10-6 in favour of an approval decision, which is now expected by 29 April.
While convincing companies, especially small biotechs with limited budgets to incorporate patient voices earlier in the development process can be challenging, Kahn stepped back to give the big picture. He said approximately 86% of candidates fail to meet their development timeline, and patient recruitment is the biggest reason for that. The cost of developing that drug would far outweigh the cost of supporting patient engagement early in the process, which can help support candidates, he added.
Kahn spoke at a time when the FDA has battled several developments in the last few weeks, which he acknowledged at the beginning of his talk. On 1 April, planned cuts for thousands of jobs at the FDA were implemented across offices, with Endpoints News reporting some employees learned of their termination when they reached their offices.
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