In response to patient demands for access to unapproved medicines, the US Food and Drug Administration and the Reagan-Udall Foundation, an independent, nonprofit created by Congress to help the agency accomplish its work, will hold a public meeting next week to create a navigator. What is a navigator? An online tool that would help patients and doctors find drugs that quality for an FDA program called expanded access, or compassionate use. These are drugs not currently approved, but are being tested and may be suitable to help someone who has a fatal illness. The notion seems helpful, but Alison Bateman-House, a bioethicist and public health researcher at the New York University Langone Medical Center, believes the navigator would be a Band-Aid. We asked her to explain…
Pharmalot: What is a navigator supposed to do?
Bateman-House: That’s still being decided. There are a number of options on the table. It could be a portal to get information on expanded access, to follow up a request for a medicine, or to follow up to see if a medicine was clinically helpful or not. It may also tabulate requests so we can have data.
Pharmalot: To what extent do you think this would be necessary or helpful?
Bateman-House: Some aspects are really good and some are potentially replicating things we already have. The goal is to increase transparency and access to information and help people understand the process better. I’m all in favor of that. It’s difficult to find information now. We have occasionally asked our research assistants to role-play. We tell them, pretend you have kidney cancer and X drug is in the pipeline and may treat your form of cancer. We ask them to navigate and find companies to talk to, how to contact them, phone or email. The majority of the time, they can’t figure out how to request a drug before it’s approved. And when a person goes to the FDA website and looks up compassionate use, they’re told to talk to doctors. But when I talk to doctors, they frequently don’t know how to do it either.
But there’s another issue. People aren’t trying to get educated. They want a drug in hand. Just creating a navigator will help inform people, but if people are informed and then denied access, it’s not a very satisfactory outcome. The FDA is getting anger and pushback because people can’t get access to drugs, but giving them a path that allows them to get an address isn’t going to help anybody. The FDA says it will have a disclaimer that it will be an informational site. But what’s the point if people get a “No” from companies? It will fall short of providing them what they really want, which is access to a drug.
Pharmalot: So what do you propose?
Bateman-House: We should create a navigator, but also have the FDA look at its own policies, so they can turn a no into a yes. Remember, it’s not the FDA but companies that get to say yes or no. So the real issue is if there’s anything FDA can do to encourage companies to say yes.
Pharmalot: How does that happen? Which policies?
Bateman-House: First, the FDA needs to write and publish a statement assuring companies that the agency understands serious adverse events can and will happen in compassionate-use settings and that such events will not constitute a “kiss of death” in the eyes of the regulator.
Pharmalot: What do you mean by kiss of death?
Bateman-House: Companies are afraid if someone dies using their drug in compassionate use, either their drug will be blamed, or — while FDA is trying to decide if the drug or an underlying disease caused a problem — their clinical trials will be halted. If you’re a drug company, you don’t want trials halted — it costs time and money. You don’t want people thinking your drug kills patients, and you don’t want FDA to think your drug needs extra scrutiny when it comes up for approval. They need to write this down — it should be a stated policy.
Pharmalot: But how often does a trial get halted?
Bateman-House: We don’t know. FDA needs to do an audit so they can maintain an online database so that companies can know. This can give them a grasp of the risk in providing compassionate use. If they see it’s a small number of cases, companies would feel safer. Such an investigation and report would go far toward helping companies feel reassured that compassionate use, even among the very sick, will not result in the derailment of their costly and lengthy drug development process.
If companies understand that FDA will not automatically penalize them for serious side effects … but will rather use discretion in deciding which side effects to investigate, will all companies open their doors to those seeking access to their investigational drugs? No. But this one factor is more likely to expand patient access than building a navigator or any other proposal currently on the table.
Pharmalot: You told me you don’t think FDA or Reagan-Udall should run it, though. Why?
Bateman-House: The pharmaceutical industry may be the best suited to run such an informational clearing house, either by itself or in concert with a broad-based patient advocacy organization that specializes in patient education. I’m concerned that FDA is always understaffed, never has enough money, and is subject to political pressures.
But the main argument … is logistical. We do not want a webpage that has thousands of links to company webpages that will go inactive and need to be maintained. We do not want a webpage with lists of phone numbers that go out of service when small companies are acquired by larger ones. We do not want a webpage that promises assistance only to result in frustration. We already have one of these, and it is called ClinicalTrials.gov. And that should be modified so it provides a user-friendly way for doctors and patients to navigate up-to-date information. Right now, any patient trying to find something would not know how to do it.