Is it Time to Sound the Alarm?

Unknown-1     Is it Time to Sound the Alarm?

Patricia Weltin

The Orphan Drug Tax Credit came into being in 1983 after a group of determined rare disease parents pushed for legislation to incentivize rare disease research. The Act gave a categorization of “rare” to diseases that affect fewer than 200,000 people. Further, the Act created incentives for pharmaceutical companies to research treatments for rare or “orphan” diseases like tax credits and an extended patent life.

Fast forward thirty years. There are 7,000 rare diseases with 5% having a treatment. Not a very impressive statistic, but lately things have been looking up. There is a real interest to create treatments for small population diseases. Dare I hope for a treatment for my children’s disease? I did, that is until I watched the President’s State of the Union address and heard of his Precision Medicine Initiative.

Precision medicine and what it means to the rare disease community has been weighing heavily on my mind for months. Precision medicine will allow treatments to be tailored to a person’s specific genetic make-up. This type of targeted treatment is already being used to provide more effective treatments for breast cancer, lung cancer and leukemias. Basically, precision medicine will create better treatments, but in the process will make all diseases “rare.”

If you read the press release on the President’s Precision Medicine Initiative put out by the White House, you will see that one of the main objectives is “more and better treatments for cancer.” While I believe this to be wonderful news, having lost my mother to ovarian cancer and soon to find out if I carry the gene, I believe it sends a message to those of us already “orphaned” that we are likely to stay that way.

This is where I sound the alarm. We are looking at a two-pronged hit to the rare disease community. The first is the President’s overlooking the rare disease community in his Initiative. The second is the future of the Orphan Drug Tax Credit. What becomes of the Orphan Drug Tax Credit when all diseases are considered rare? Both of these things are alarming, but not something we can’t overcome. Like the generation before us, we will take the lead in our health and come up with new ways to incentivize rare disease treatments. We are creative, resourceful and determined!