I was recently interviewed by Context Matters on Expedited Reviews and the Current State of Drug Development. Here’s Part One of the conversation
Regardless of what you want to call it, whether it’s expedited review or special protocols, limited use medicines, the theory is, “How can the FDA help bring important therapies to the market faster?” And, there are a lot of pieces to that puzzle.
At the end of the day, the agency still has to meet with the sponsor, who still has to have data. They have to meet and decide how to move forward.
There are two issues that also have to come into this conversation:
- The patient voice
- Benefit / risk profile
Those are two things that really are not usually discussed. Generally speaking, in this type of conversation what comes about in the discussion is time. How quickly can something happen? Obviously faster is better than slower, but you don’t want to ever sacrifice proper review.
Blood From a Stone: Expedited or Standard, It’s All the Same FDA Resources
A key issue not being widely talked about is that the FDA isn’t hiring a whole new team to deal with expedited reviews. It’s the same people. So does an expedited review take precedence over a non-expedited review? How does the agency determine who gets the resources at what point in time?
Obviously, if you’re going to file a regular old-style NDA, you get a PDUFA (Prescription Drug User Fee Act) date and the agency is responsible for that. How do you weigh that against “critical medical need”?
The answer is it’s on a case-by-case basis. You can’t say, “Hey, we’re not going to make the PDUFA date because we’re reviewing someone else’s drug,” that’s a non-starter. You have to view expedited review situations in the broader context of everything else the agency has to do.
And then the agency is then going to define (on a case-by-case basis) what it feels is most important to the public health. Here’s an example: MS (multiple sclerosis) or IPF (Idiopathic pulmonary fibrosis) – Are either of those more or less important from an expedited review / resources perspective than a new anti-infective? It depends who you talk to. If MRSA issues are on the top of your agenda, then it’s one thing. If it’s orphan diseases, it’s another or if it’s pain-mitigation it’s something else again. Where you stand on these issues depends on where you sit.
I don’t really think having people specifically tasked to working on expedited reviews is the way to go, unless you have more people. And then the question is, where do they come from and do you take the best and brightest away from standard review, to put them there? How do you weigh the general importance of staff time and agency resources? Do you want it to be for a disease that is not an orphan disease that’s an important therapy, or do you want it to be on an orphan disease that can have a profound difference on people’s lives? Those are really philosophical questions, but it comes down to resources. I don’t think you can have dedicated teams when you don’t have people that can dedicate their time exclusively, otherwise it is just rhetoric. It’s just another committee on which the same people sit.
Comparing the Agencies
What might be interesting (and is generally not part of this conversation) is a move towards greater FDA / EMA harmonization on certain disease categories. There’s been a lot of chatter on this issue, but at the end of the day nothing really happens. I think the EMA would be interested in that, probably more than the FDA, because one of the main differences is the type of data accepted by the EMA. The EMA casts a wider net in terms of the types of data they accept for any given application.
One of the reasons the EMA has gone beyond the FDA is that their national economies are so much more financially strapped than we are here. It is important to remember that EMA decisions are only the first part of gaining market access. After the EMA, drugs go on to be evaluated by different country Health Technology Assessment (HTA) agencies to determine reimbursement and pricing in various markets. And from a reimbursement perspective, the HTA agencies are moving away from the QALY (quality-adjusted life year) towards a more value-based insurance design.
From a review proposition, from the FDA’s perspective, obviously they don’t get involved in cost issues, only clinical issues. I think what innovator companies are going to find out (especially with the expedited review pathways) is that the risk of getting a drug yanked quicker (meeting a quick death) rises. So they are going to have to understand, from a business perspective, the risk of asking the FDA for these special medical use reviews – it could backfire unless you understand what you are asking the FDA to do. The FDA is trying to signal that there are multiple ways to get an important new therapy to market quicker, but that has to be based on a public health need and not on a marketing strategy.
