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GULFO: ‘Right to try’ just another bandage on a chronic wound

New initiatives can’t mask need for closer look at FDA

- - Friday, July 11, 2014

"Right to try" laws aim to broaden access to experimental drugs for people who are terminally ill. Colorado's law was enacted in May, and in Louisiana and Mississippi, the laws are waiting on the governors' signatures. In Arizona, right-to-try will be put to referendum this fall.

Right-to-try gives hope to patients who have none. The public wants right-to-try laws, but the FDA does not like them. In a recent editorial in USA Today, FDA Commissioner Margaret Hamburg wrote, "The column 'Right to try experimental drugs: Column' misleads the public on the Food and Drug Administration's approach to allowing access to experimental drugs The agency is an important part of the process, helping to ensure that patients are protected from potentially harmful drugs or one that doesn't work ..."

The FDA response is at the heart of the problem. Notice the commissioner's use of the word "protected." When a patient is dying, who or what are they trying to protect? The benefit-to-risk ratio is overwhelmingly favorable in this setting with virtually any experimental product. According to Section 1003, Subpart 1 of the Federal Food, Drug, and Cosmetic Act, "The Administration shall promote the public health by promptly and efficiently reviewing clinical research and taking appropriate action on the marketing of regulated products in a timely manner "

Oddly, this is not what the FDA says about itself. Instead, the FDA website states: "What We Do. [The] FDA is responsible for protecting the public health by assuring the safety, efficacy and security of human and veterinary drugs, biological products, medical devices ."

"Promoting" and "protecting" are not synonyms that can be freely interchanged; they represent completely different ideologies. Protecting serves to further preserve the antiquated medical status quo. It assumes current medicine is always safe, when that is often not the case.

What about misleading the public? In a May piece in FDA Voice, the commissioner touted the fact that in 2013, the approval times for drugs was faster in the United States than in Europe and Asia. From 2003 to 2014, the median approval time in the United States was 304 days. The article omitted the fact that these are "review days," as opposed to "calendar days," that the FDA starts and stops the review clock at its discretion and that the statutory requirement is 10 months for every drug, not a median performance metric for all drugs. This is the reason for incessant calls for FDA reform.

The commissioner went on to laud new legislation that was passed in July 2102 called FDASIA (FDA Safety and Innovation Act), which included the new Breakthrough Therapy Designation. Since the enactment of this piece of legislation, 44 products (of the 178 that applied) were granted the designation and six products were approved. The piece highlights the approval of "a late-stage lung-cancer drug" four months ahead of its goal date (as defined by PDUFA — Prescription Drug User Fee Act).

The details about this product (Zykadia) were not mentioned. The drug is approved to treat patients with lung cancer that possess the ALK mutation, which make up 5 percent to 7 percent of all non-small-cell lung cancer, and who have failed or are intolerant to Xalkori. This is a very small number of patients — so small that the drug received Orphan Drug Designation. Not only that, the drug also received Priority Review and Accelerated Approval, rendering Breakthrough Therapy quite moot.

As Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, stated in May at a Friends of Cancer Research meeting, Breakthrough Therapy is intended for products that show "spectacular results" and would be "game changers." The circumstances in which this would most likely occur are in niche settings where there are no alternative treatments so that the benefit-to-risk ratio would be overwhelmingly positive.

Sure enough, 34 percent of drugs receiving this redundant and superfluous designation have been targeted cancer drugs. The travesty is that this program and the others encourage drug makers to develop products for small niche claims.

Americans want drugs for all conditions approved faster — that's what right-to-try is all about. They want the FDA to promote health, not to protect the medical status quo; to review all drugs quickly, not just those that could provide "spectacular results" in the estimation of the FDA.

Right-to-try is the latest Band-Aid on the chronic wound of FDA performance. Bandages before it that have failed include PDUFA, MDUFA, Breakthrough Therapy and the Transparency Initiative. The reason for failure is because none of them address the fundamental problem causing the wound; that is, FDA's ideology.

As with chronic wounds, when the fundamental medical condition is not addressed, amputation is the unfortunate next step to save the patient.

Joseph V. Gulfo, M.D., is the author of "Innovation Breakdown: How the FDA and Wall Street Cripple Medical Advances" (Post Hill Press) and CEO of Breakthrough Medical Innovations.