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I don’t think very highly of Senator and Republican Presidential candidate Ted Cruz, but his proposal to loosen the drug approval process is at least worth discussing. If you haven’t heard, Cruz’s RESULT Act proposal is as follows:
- FDA to grant reciprocal approval of “life-saving” drugs and devices from regulatory agencies in developed countries including EU, Israel, Australia, Canada and Japan. FDA would have only 30 days to review and approve;
- Congress can override FDA decisions with a majority vote.
Basically he is trying to make it easier to get drugs and devices on the market. There are a number of problems with this approach, which are pointed out by my most of the respectable analysts. Objections include:
- FDA may just reject the applications anyway since 30 days is not a long enough time to review and they may object to procedures used elsewhere;
- Inserting Congress directly into the process undermines the scientific basis of the decision;
- Trial sponsors may seek the jurisdiction with the lowest standards or fastest review times, imperiling safety for Americans;
- It would hurt US economic development by shifting development resources overseas;
- We would be ceding our sovereignty to foreigners;
- FDA is already pretty fast and responsive and has programs for compassionate use and acceleration of the approval process when warranted.
These are all reasonable, and yet I was struck by the fact that almost all the commentators use the Thalidomide debacle as their one and only example. See for example STAT, The FDA Group Blog, and Harvard Law Blog, which are the top articles that come up under a Google search for “Ted Cruz FDA proposal.”
Thalidomide was approved in Europe as a sleeping pill and for morning sickness but rejected in the US. It caused serious birth defects, with thousands of people affected in Europe but not the US. Great example but we’re talking 1956. Remember 1956? I don’t. But to give you some perspective it was just two years after food rationing was lifted in the UK and nine years before Medicare was established. Soviet troops rolled into Hungary to crush the uprising there.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
I don’t hear people citing 1956 examples about anything else in healthcare.
A more serious discussion could be held on the balance between safety and efficacy in FDA approvals. Cruz’s impulses might be better directed to that debate. Maybe it’s ok for FDA to approve any product that’s safe, and not force sponsors to prove efficacy. Sponsors would still want to demonstrate efficacy, but they’d need to do so for healthcare payers, not the FDA.
That’s not as simple as it sounds either. Here are some of the challenges:
- The biggest payers are Medicare and Medicaid, so even if you take FDA out of the business of judging efficacy the government is still involved;
- In many situations, safety is relative, not absolute. I might accept a modestly effective cancer drug that kills 1 percent of those who take it, while I wouldn’t accept a cure for toenail fungus with the same death rate. But it’s also important to protect cancer patients from dangerous drugs. A recent NYT article about the death from cancer of the wife of the FDA’s head of oncology included this important and under-appreciated point: “Cancer medicines not only often fail to save patients but can accelerate their deaths and make their last weeks far more painful”;
- Safety and efficacy are individualized. (Cruz seems to recognize this.) Some drugs will be effective for only a small population; similarly safety issues don’t always apply across the board. I would want access to a drug that could cure me even if it could kill someone else, especially if there were a companion diagnostic to sort out who will be helped and hurt.
Overall I think the FDA is doing a good job of managing the conflicting pressures it faces. Some parts of FDA (such as the groups with responsibility for HIV and oncology) do a better job than others.
I really would like to see Cruz’s proposal spur a more productive debate and not simply be dismissed out of hand.
