Convergence: Listen to patients when developing treatments for unmet medical needs, experts say

LONG BEACH, CA – Listening to patients and communication across disciplines are key to developing treatments for unmet medical needs, according to a panel of experts who spoke at RAPS Convergence 2024 on Wednesday. They also noted that healthcare product makers need to engage with patients and their caregivers continuously to understand how their conditions affect their lives.

Panelists included Cheryl Coon, vice president of the clinical outcome assessment program at the Critical Path Institute; Allison Komiyama, vice president of medtech innovations at RQM+; Karl-Heinz Huemer, a consultant at khhconsulting; and Justin West, president of the KCNT1 Epilepsy Foundation. João Duarte, a vice president, Head of Global Regulatory Affairs Excellence at Ipsen, moderated the panel.

According to Coon, collaboration is the most important element in developing treatments for unmet needs. While it’s important to have scientists conducting research, developing the right endpoints, collecting the right data, it’s critical that all stakeholders communicate across their silos.

“They don't need to be at the same table doing their work together, but they need to be having conversations,” said Coon. “If you're not able to have your conversations with the people who are discovering the drug and the people who are designing the study and the people who are actually creating the recruitment criteria, you can't turn that into the evidence-based package that needs to be submitted for review and for ultimately hopefully success.”

The panelists also acknowledged the difficulty of defining unmet medical needs, and some noted that the definitions are often arbitrary. Huemer said he isn't optimistic that there can be a standard definition of unmet need because the term needs to be adaptable and modifiable. He added that it is particularly difficult to define unmet medical needs for children and that endpoints used in pediatric research should be adaptable.

Based on his experience as a patient advocate, West said he’s noticed a significant disconnect between drug developers and patients and their caregivers. Drugmakers often have their own ideas of what success looks like, which may not be what is important to patients and could result in significant barriers to participation in trials. He advised that after pharmaceutical companies decide what diseases they plan on developing treatments for, they should contact their respective disease foundations to understand what patients actually need.

"You're not going to recruit me to a drug that you think is important, but that doesn't resonate in my household. It turns into a waste of time and money,” said West. “The fact is you probably should have built a team that represents every element that needs to go into a drug development package.”

Huemer agreed, and said that oftentimes, researchers will ask for patient input and think they have covered the topic. However, he said that it’s critical to continue talking to patients and their caregivers to constantly modify how researchers think about the products they are creating based on the feedback.

"It's completely different if you really had a dialog, that you listened to them, that you learned, and that's something that's not exactly the same,” he added.

West said he would take it a step further and encouraged key stakeholders to meet patients on their turf, including patient advocacy conferences and even their homes to understand their day-to-day struggles.

West also touched on the relationship between patients and regulators and noted that the patient community often sees regulators as adversaries and hurdles to getting treatments. He said that relationship has to change and added that it’s important to get everybody involved in developing a treatment for an unmet need of a stakeholder early on and start interactions to help build trust.

Convergence: Listen to patients when developing treatments for unmet medical needs, experts say

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