Congress: Keep the nation’s drug safety system intact

Using the same backlash politics as some presidential candidates, generic drug lobbyists are pointing to the despised former CEO of Turing Pharmaceuticals, Martin Shkreli, as the reason Congress should weaken drug safety protections, called Risk Evaluation and Mitigation Strategies (REMS), which have allowed Americans with cancer, multiple sclerosis, and other serious diseases to be treated effectively with medicines that would otherwise not be on the market today.

At issue is the most stringent type of REMS, called “Elements to Assure Safe Use,” where the U.S. Food and Drug Administration can require a restricted distribution system if a drug carries a severe risk, such as a terrible birth defect, organ damage, or death, when not administered with utmost care. Due to the safety issues involved, FDA can only approve these medications with strict controls, which is why it is time to separate fact from fiction so these medicines will remain available to patients.

The first fiction is that somehow Shkreli is relevant to the debate over REMS. He is not. Shkreli instituted a controlled distribution system for a needed medicine for which there is no generic copy and then hiked up the price by 5500 percent to maximize profits.  While this action is reprehensible, the drug in question, Daraprim, is not subject to REMS safety protections and the limits on distribution were not mandated by the FDA. Additionally, Daraprim’s patent expired in 1953, meaning any generic company could have developed a copy long ago. This didn’t happen because the market for the drug is small and the original price of $13.50 a tablet was insufficient to spur generic development.

Another fiction is that REMS safeguards, and especially restricted distribution systems, are used to block generic manufacturers from getting the samples needed to create cheaper alternatives, resulting in billions of dollars in avoidable costs. As evidence, lobbyists point to a questionable 2014 study funded by the Generic Pharmaceutical Association, which claims at least 40 unidentified brand drugs worth $5.4 billion fall into this category. However, this projection is not based on a review of published data but, rather, is the result of polling some association members who identified the drugs in question with no explanation of the criteria used.

Yet, even with this questionable methodology, only 16 of the 40 drugs in the study have REMS controls and the other 24 have “non-REMS restrictions” that are not described. Moreover, the study attributes over half the sales of these 40 brand drugs to four medicines but fails to state if any carry FDA-mandated REMS requirements.

What then are the facts about the purpose and use for REMS? Quite simply, Congress gave FDA the authority to require REMS so important medicines that carry high risks are prescribed and dispensed appropriately by physicians, pharmacists, and other health professionals and taken correctly by patients. This requirement is especially critical for drugs known to carry the greatest risks where restricted distribution systems ensure these medicines are not taken by the wrong person or not taken exactly as directed.

However, because very few drugs cause these “serious risks,” FDA-mandated restricted distribution systems are rare. Out of the more than 1,100 brand drugs approved by FDA, approximately 30 require restricted distribution systems as part of REMS to control access and use of the drug at every step in the process.

Finally, REMS safeguards have not been a barrier to generic drug development, despite claims to the contrary. According to statistics documented on the FDA website, nearly a dozen medicines marketed with REMS safety precautions have gone generic, including nine subject to strict controls, such as restricted distribution programs. Additionally, several new generic medicines carrying REMS controls are under FDA review and are likely to reach the market.

When the facts are separated from the fiction, it is clear that the REMS program envisioned by Congress and implemented by the FDA is working to advance patient safety and give patients access to innovative medicines that would otherwise not be available. That’s why this matter is too important for backlash politics.  What is needed are legislative policies that support generic drug development while maintaining the drug safety protections REMS makes possible.

Worthy is director of Public Policy at the Aimed Alliance.