L Last month the Food and Drug Administration took the unusual step of admitting that it had made a mistake 25 years ago when it was given opioid addiction treatment called Orphan Drug Status conferred buprenorphine (Subutex).
The original use of Orphan Drugs was based on the claim by Indivior, the manufacturer of Subutex, that the company would probably not make a profit by marketing the drug. Opiate addiction was not the widespread scourge it is today.
In its reversal, which I called for earlier in the year in STAT, the FDA said its original decision was wrong because the manufacturer made billions of dollars from the drug.
When the FDA admitted its mistake, it was brave, but also an important step for public health, since every year lives were lost due to opioid addiction and the families it destroyed.
The original orphan designation prevented Brixadi, a sustained release injection of buprenorphine, from entering the market, as did any other buprenorphine that tried. By lifting orphan drug status for buprenorphine, the FDA has opened the door to other opioid addiction drugs, expanding options for patients with opioid addiction and hopefully lowering treatment prices by creating a competitive market.
I am a lawyer for people with rare diseases, not a lawyer, and it is admittedly difficult to explain this problem. But here goes.
Already in 1994, Indivior referred to a rarely used provision in the 1983 Orphan Drug Act, according to which the FDA can give a drug the status of an orphan drug if the manufacturer can prove that the drug is unlikely to make a profit. At the time, no one had imagined that a drug for the treatment of opioid addiction would ever be in high demand, and the FDA agreed to grant Subutex orphan drug status, and thus seven-year commercial exclusivity.
A legal loophole – and this is the real problem – states that orphan drug status and marketing exclusivity can be expanded for future versions of the same drug without the manufacturer having to continue to demonstrate that the drug is unlikely to be profitable is. When Indivior asked the FDA to authorize a once-monthly injection of buprenorphine called Sublocade in 2016, the company received the original exclusivity, although it now generated billions by selling buprenorphine.
The FDA reversal has looked at the specific situation in which this opioid addict occurs. However, the potential for this continues – the Orphan Drug Act has not been changed to prevent this. In this way, a manufacturer can still gain orphan drug exclusivity based on a prediction of unprofitability and then get extensions for future versions of the same active ingredient without having to prove that the drug is actually not profitable.
This is an example of playing the system using a legal provision that increases profits for a pharmaceutical company.
I have spent almost 20 years working for 30 million Americans with rare diseases. The Orphan Drug Act is a good law. The development of drugs for rare diseases has been successfully promoted.
However, the buprenorphine case has uncovered a legislative error that needs to be remedied. I encourage Congress to make this small but important legislative change: for a manufacturer to continue to be given an orphan designation due to lack of profitability, it must demonstrate that the drug was unprofitable and will likely not be profitable in the future]
The Orphan Drug Act has very successfully encouraged companies to develop treatments for rare diseases. However, the existence of the gap in the nonprofitability provision could be used to undermine the integrity of the law itself. And that would be a shame.
One possible approach to fill this gap is the adoption of the Law on Fairness in Orphan Drug Exclusivity (HR 4712) by the representatives Madeleine Dean (D-Pa.), Marc Veasey (D-Texas) and Earl L. "Buddy" was introduced. Carter (R-Ga.) And David McKinley (RW.Va.). According to this law, any company that receives an orphan label for a drug as part of the nonprofitability determination would have to demonstrate that all subsequent drugs that are eligible for the label have no reasonable expectation to reimburse research and development costs.
Drugs that exist thanks to the Orphan Drug Act have saved lives, and future lives depend on this law continuing to function well. We cannot allow disaster and chaos to stand in the way of the law's mission and the incredible results it brings to people across the country.
It is time for Congress to close this gap.
Diane Dorman was the vice president of public policy for the National Organization for Rare Disorders, which represents patients and patient organizations with rare diseases, for 15 years.