Call Your Bluff

The rare disease space has been much in the news lately. There have been attacks on the pharmaceutical industry. There have been attacks on pricing. There have even been attacks on rare disease advocacy. And there is truth in all of these articles. There are companies that abuse the Orphan Drug Act. There are companies charging astronomical prices on orphan drugs. There are advocacy organizations that are barely disguised as such. There are so many things wrong in the world of rare disease, but nothing is more wrong than playing the patient card when you don’t mean it.

Recently, Senator Grassley began an investigation into the Orphan Drug Act. Of course, we should have seen this coming as early as Martin Shkreli hit the newsstand. Now, the one piece of legislation that has done the most good for rare is under the gun. Who is to blame? Is it industry? Is it the FDA? Is it advocacy organizations? Yes, it is.

This afternoon I called Senator Sanders’ health aide, believing he was working with Senator Grassley. We both agreed that the ODA needs work and pricing is an issue. I was grateful for her passion on behalf of the rare disease community. She stated how wrong it was for people to lose their home because they are trying to pay for an exorbitantly expensive treatment. It is wrong, but I had to call Sen. Sanders’ bluff. So I asked his health aide why Sen. Sanders was so focused on the 2% that have a treatment and not the 98% that don’t. The rest of us, without treatments, are also losing our homes because of medical bills. Our marriages, our families, our friendships and our neighbors too often leave our lives because we are complex. We cannot devote attention to everything we need to, so we work off our list of priorities and there so many priorities. The financial burden of having a rare disease, treatment or no treatment, is huge. Living rare is uniquely challenging, truly unimaginable. We are medical outcasts in a country rich with medical technology, with the best doctors, and with the best facilities to treat the ill.

So, Senator Sanders we appreciate your concern for the 2% of the rare disease community you are addressing, but we respectfully ask that you look into the lives of the 29,400,000 people without a treatment living with a rare disease. When you have tens of millions of people, half of them children, suffering and dying without any treatments, that is a health crisis. Addressing pricing for rare disease treatments is important, but addressing pricing and not addressing the other 98% without a treatment seems disingenuous.