How to Improve the Rare Disease Treatment Pricing Problem

During Rare Disease Week on Capital Hill, on behalf of Lyme and dysautonomia, in addition to ALL rare disease, I lobbied my congressman to support the newly introduced (kind of) OPEN Act.

The OPEN Act would allow six months exclusivity to drugmakers if they repurpose already approved drugs to help treat rare and neglected diseases. This was originally part of the 21st Century Cures Act which was passed last year, but was taken out at the last minute. One of the main goals of the OPEN Act is to make drugs more affordable and available to those of us suffering from rare disease. This comes at a time when big pharma is getting a lot of flack recently for producing life-altering drugs to address rare disease, but hiking up the prices to the point where we can’t even come close to purchasing them.

The high price of rare drugs is a complex issue. Even personally I have cognitive dissonance about it. On the one hand, I think “We get it. Big pharma needs fair return-on-investment when introducing these drugs to market.” On the other hand, I think “Wow, if being sick/in pain/having a fatal illness isn’t bad enough, you want families and patients to go broke by merely trying to survive a disease they never asked to battle.”

In thinking about all this, I read an article by Meg Alexander that I wish every pharma company would read. In it, she has three main tips for them in moving forward with investigational rare medicines.

  1. Pricing. Patients need access to treatments, and if pharma companies price a drug at a cost that is theorectically doable for an ideal ROI but is unfair to consumers, there’s no amount of PR that will help them recover from the backlash and public scrutiny that pricing has earned them.
  2. Context. While financial assistance is occasionally available to rare disease families, the actual cost should be communicated where rare discussion exists. And perhaps insurers should be asked to provide a clearer picture of the cost burden rare treatments have on society so that their accuracy is ensured.
  3. Collaboration. Big pharma and drug makers should collaborate with both the patients and their advocacy groups, and the government to better fulfill the precision medicine ideal.

Check out her entire article here.


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