Sadly, there is more blood on the hands of the FDA. Its as if these people care only about their regulatory program rather than the lives and health of people. Of course, that couldn’t be – that would be unspeakable.
The Wall Street Journal has a piece on a drug that is capable of dramatically helping some people with multiple sclerosis. The drug has been approved in “30 countries, including Canada, Australia and all members of the European Union. But on Dec. 27, Food and Drug Administration reviewers at the division level (subject to a final decision by top officials) rejected an application to use the drug here to combat MS.” Sadly, the division level reviewers are rarely if ever overruled.
Why did the FDA lords refuse to approve the drug?
The primary reason FDA reviewers gave for rejecting Lemtrada was that the studies demonstrating the drug’s efficacy did not conform to the agency’s standard requirement of double-blind, placebo-controlled drug trials—where some patients, unbeknownst to themselves and their doctors, receive placebo treatments. There are excellent reasons for the standard approach, but only up to a point. Lemtrada and many established MS treatments have immediate side effects, such as nausea and headaches, that are well known to doctors and patients. A double-blind trial would not really be blind. Patients on a placebo would promptly discover that they were the “controls,” and many would decline to participate further—scrambling the statistical comparison with patients receiving real treatments.
The article continues:
For Lemtrada, the FDA reviewers announced that the trials were not “adequate and well-controlled.” They are now demanding another round of trials, with somewhat different procedures, that would take years and cost at least $100 million. Given the magnitude of the results of the already completed trials, the additional trials could add nothing to answering the regulatory question of whether Lemtrada is suitable for clinical use against MS.
If one of my family member needed this drug, I would be protesting. I would find out who the peope were who turned it down and I would protest in front of their house with dramatic images of the infirmity this disease causes. Let these doctors explain their actions to the children and their neighbors.
The Republican Party is supposed to be the party that resists regulation but alas they are pretty lame. There ought to be bills being introduced in Congress to address these problems. Here are a couple of places to start:
1. Off label uses – where the drug is approved for treatment A, but not treatment B – should not only be allowed but should be permitted to be advertised. The drug is approved as safe and the medical industry is perfectly able to determine whether it is effective for other uses. The FDA goes after advertising of these off label uses with a vengence, denying truthful information to people.
2. Drugs that are approved in a certain percentage of a group of other countries, such as in Canada and Europe, should be presumptively approved. The FDA should only then be able to disapprove the drug if it can show that there is a problem.
These reforms are to my mind no brainers, but they will be resisted by the FDA and others tooth and nail. It is time to start the battle.
And I know the perfect person to lead the charge. There is a libertarian Senator who is a doctor who has a talent for publicizing important matters. This should be Rand Paul’s cause.