The Centers for Medicare & Medicaid Services (CMS) is looking to hear from our community about how the Inflation Reduction Act (IRA) should work.
The IRA was signed into law last August and now, for the first time, CMS will start negotiating the price of certain prescription drugs. The way CMS has interpreted the law could have significant impacts – some positive, some potentially problematic – for the more than 25 million people living with a rare disease.
To learn how the IRA could affect the rare disease community, visit this page on our website, which includes NORD’s position listing five positives & concerns.
Thank you to all the advocates who have already spoken up on this issue! Here’s a look at what our community is saying:
- As an individual caring for some with adrenoleukodystrophy, a rare disease, I urge you to implement the Inflation Reduction Act in a way that works for the rare disease community. This issue is important to me because my son who is only 2 years old deserves a chance at life that is affordable.” – Jessica H.
- “As an individual living with a conglomerate of rare diseases, I urge you to implement the Inflation Reduction Act in a way that works for the rare disease community. Weekly I hear from my desperate friends who have lost access to drugs due to cost, impossible pre-authorizations or the Medicare waiting period.” – Maria P.
- “As an individual living with a rare disease, I urge you to implement the Inflation Reduction Act in a way that works for the rare disease community. My rare disease, Complex Regional Pain Syndrome (CRPS), has very few FDA approved treatments. We are lucky enough to have a few medications approved for treatment of CRPS. The drugs and treatments that make the most difference in the disease process, with a higher rate of remission, remain experimental. This means that the full cost falls on the burden of the patient despite having medical coverage. This means these treatments at out-of-reach for many.” – Erin M.
- “As an individual living with a rare disease, I urge you to implement the Inflation Reduction Act in a way that works for the rare disease community. Myself and my two children benefit immensely from an orphan drug which is the only FDA approved treatment for Periodic Paralysis. While the cost is high, the manufacturer and insurance have worked very well together to ensure no cost to their patients. Very few people need this life changing medication, and I fear the demand is so low that drugs for similarly small populations may not be developed if the financial incentive is capped.” – Marti S.
- “As an individual living with a rare disease, I urge you to implement the Inflation Reduction Act in a way that works for the rare disease community. My neurologist and I like for me to try [a certain medication], but my insurance will not approve it, and attempting to purchase it OTC is expensive, since I am on both Medicare and Medicaid. Since I have a Rare Disease (Cerebellar Ataxia), there are no FDA approved meds, so I have to experiment with meds for similar disorders that share symptomology. – Brandon H.
- “As an individual living with a rare disease, I would urgently ask you to implement the IRA in such a way that those of us living with rare diseases could benefit as much as possible. Currently I am on Social Security disability but must pay over $1,000 a month (after reaching the catastrophic phase) for my medication. I am very grateful for the Social Security benefit of $1,900/month but am sure that you understand that there is no way I can pay all bills (including mortgage, utilities, insurances etc.) when I spend half that amount on medication. Key provisions in the IRA, including the $2,000 annual and amortized monthly caps on out-of-pocket costs for Medicare Part D beneficiaries that start in 2025 will ensure that more rare disease patients will be able to afford the life-altering therapies they need.” – Astrid H.
- “Diagnosed with my first rare disorder at the age of twelve, I have personally spent the majority of my life struggling with this disease. I consider myself one of the lucky few in the rare disease population, because some breakthrough treatments were discovered – and FDA approved – in the early years of my diagnosis. Thankfully, with insurance and ‘patient access programs’ my family was able to help ensure that I got these critical medications when I needed them. Without such treatments, I never would have succeeded in medical school, to become the physician I am today. However, my work as a Primary Care Physician has underscored how lucky I have been, how unique my opportunity was. I have seen countless patients struggle to afford their essential medications, at times sacrificing basic needs to get them. Sadly, I’ve seen even more patients with rare disorders who have no approved treatment available at all. I join those patients, and everyone in the rare disorder community, in putting my hope in future research outcomes.” – Kristen K.
- “As an individual living with multiple rare diseases, some genetic and some of unknown cause, including hyperekplexia, Alport Syndrome, and low gamma globulin, all rare diseases, I urge you to implement the Inflation Reduction Act in a way that works for the rare disease community. It took me four neurologists and nine months to be diagnosed with hyperekplexia, because the doctors simply didn’t know what to look for even though I was seizing for up to eight hours a day. While there is a relatively easy maintenance drug for this condition, Alport syndrome is just starting an investigational treatment; right now, its only partial cure is kidney transplant, and the eye and ear problems continue to progress even so.” – Elizabeth S.
- “As an individual living in Boston with 9 rare diseases and conditions, I urge you to implement the Inflation Reduction Act in a way that works for the rare disease community. I depend on Medicare and Medicaid for healthcare, yet I still have to borrow money, every month, simply to cover my medically necessary medications and supplements.” – Lynn C.
Exactly how this new law will be implemented is critically important because the vast majority of the 7,000+ rare diseases still don’t have FDA-approved treatments. NORD believes the law’s success hinges on a careful balance between greater affordability and continued industry investment in rare disease drug development.
Just as we have for the past 40 years, NORD is taking a leading role to ensure the law ultimately benefits patients and families living with rare disease.