The U.S. Food and Drug Administration today proposed to withdraw approval of the drug midodrine hydrochloride, used to treat the low blood pressure condition orthostatic hypotension, because required post-approval studies that verify the clinical benefit of the drug have not been done.Phone lines were flooded. Doctors scratched their heads because the "options" for alternate therapies to replace this drug amounted to exactly... none.
Patients who currently take this medication should not stop taking it and should consult their health care professional about other treatment options.
Two days later on 18 August 2010, Shire pharmaceuticals announced they will stop manufacturing midodrine 30 September 2010, adding fuel to the patient backlash of the FDA's prior announcement:
Shire plc (LSE: SHP, NASDAQ: SHPGY) acquired ProAmatine (ed's note: trade name for midodrine) as a part of the acquisition of Roberts Pharma in 1999 and Shire conducted and completed the post marketing trials that the FDA required. The FDA, however, viewed these trials as inconclusive and required that additional trials be conducted for ProAmatine to maintain its marketing authorization. As a result, Shire elected to withdraw the product effective September 30, 2010 and notified the FDA in November 2009 and healthcare professionals earlier this year of this decision. Shire's withdrawal of the NDA was not related to any concerns regarding the safety of ProAmatine.Doctors scrambled. Patients complained. Finally, news about the FDA recanting came via a New York Times article on 3 September 2010:
The agency’s flip-flop demonstrates the difficult choices regulators face in policing the nation’s drug market. Cracking down on drug makers sometimes means stranding desperate patients. And now that Congress has given the Food and Drug Administration greater powers to insist on better information about life-saving medicines, such disputes may become more common.Sadly, not until a 10 September 2010 press release from the FDA could the information published in the New York Times be confirmed and a plan of action taken to assure patients and their physicians aren't blind-sided by the FDA's actions on this matter:
Since the agency notified the manufacturers of the proposed withdrawal of midodrine, we have heard from professional organizations, doctors, and patients about their concern about losing access to midodrine. We have also heard from organizations that have expressed interest in conducting the clinical studies necessary to establish effectiveness, as well as doctors and patients who support the conduct of such studies. FDA intends to work with Shire, the generic manufacturers, and other organizations to discuss the data that are necessary to establish the efficacy of midodrine. FDA will post this information in a public docket.So what have we learned with this regulatory fiasco?
First, once the FDA approves a drug with strings attached, they have an obligation to assure to the public that those strings mean something long before a drug goes generic. Why impose regulatory requirements if they're not going to enforce those requirements in a timely fashion? We are left to wonder how many patients might have been harmed by their inaction had there been a problem.
Secondly, news agencies now appear to be our best source of new information about updates on drugs and their availability. This should be of considerable concern to physicians who need to verify the legitimacy of information published in the mainstream media. Admittedly, the New York Times got the story right, but such is not always the case with media reports about drugs and innovations in medicine. At the very least, the FDA should provide midodrine updates at the same time this information is provided to the media. Verifiable information is increasingly needed in our new era of web-enabled patients and doctors.
Third, this was one small example of the power of government to intervene on our patients' care rapidly and without warning to the populace at large. Fortunately, the public outcry over their action was not ignored, but patients should be aware that litigation against the government should harm arise as a result of such an action would be very difficult and expensive to prosecute.
Hopefully, the FDA has learned a few lessons with this fiasco.
One thing's for sure: you can bet our patients did.