Here’s a significant potential PDUFA addition – adding patient perspectives to the FDA’s analyses of drug application reviews.
In the minutes of the 11/22 PDUFA negotiating session, the FDA said that information about patients’ understanding of existing treatment tools is considered valuable, but is not consistently available during the review process.
This new initiative would place in front of frontline reviewers the views of patients who, for example, are willing to live with more serious side effects or safety concerns because they have failed on previous treatments and have no other options.
The agency said it wants to host meetings with review divisions and relevant patient advocacy groups to talk about available treatment options and unmet needs. That information would be used to help draft guidances for specific indications.
This is a real advance and will significantly enhance the role of the FDA’s Office of Special Health Issues. Bravo.
And speaking of greater input, industry is keen to bring more clarity and transparency to the approval process. Specifically, industry officials want a more structured risk-benefit formula included in PDUFA V and asked that the agency begin using it in its decision-making (similar efforts under way at the EMA).
CDER Director Janet Woodcock, “We’re all for that and we’ve been working on that, the industry’s been working on that, other academic parties have been working on that. So I think we will come up with something there.”
Office of New Drugs Director John Jenkins has said on more than one occasion that the framework should be simple and support sound expert judgment, not replace it.
According to Jenkins, such a model could improve predictability and consistency, support more structured discussions and provide concise descriptions of evidence and the risk-benefit implications.
Amen.
It's time to see all these good words turned into legislative deeds. And the sooner the better.
