ORPHAN Cures Act
ORPHAN Cures Act: Supporting Access to Affordable Treatments for Rare Diseases
The Impact of the Inflation Reduction Act on Rare Disease Treatment
The Inflation Reduction Act (IRA) introduced reforms for drug pricing to with the intention to reduce healthcare costs. However, the IRA includes an exemption from price negotiation for orphan drugs, which are medications specifically developed to treat rare diseases. While this exemption was intended to aid in making these drugs more affordable, it has had unintended consequences, potentially discouraging future research and investment into life-saving therapies for these patients.
Understanding the Orphan Drug Price Negotiation Exemption
Orphan drugs serve a critical role in providing treatment options for individuals with rare diseases, which are defined as diseases affecting fewer than 200,000 people in the United States. Given the high costs and complex development processes, these drugs are often priced higher than other medications. Under the IRA, orphan drugs are currently exempt from Medicare price negotiations if they treat a single rare disease and do not have additional FDA approvals for broader uses.
While this exemption may seem beneficial for patients today, it can actually de-incentivize pharmaceutical companies from expanding research or seeking additional indications for existing orphan drugs, as they may be able to retain higher prices indefinitely without the need for competitive pricing. Much of the rare disease development pipeline and the progress over
the last 40 years has relied on identifying new applications, or second uses, for existing drugs that could address unmet needs for patients living with rare diseases.
The Added Burden on Cancer Patients
Orphan drugs, or drugs for rare diseases, most directly impact oncology (cancer) therapies, which make up 92% of second orphan indications (the second use of the drug). This puts the usual approach to expanding access to cancer drugs at risk. In cancer medications more than half of drugs have multiple indications and its important those are able to be continued.
And while there are also many rare diseases that are important to our community like ovarian cancer, cervical cancer, fallopian tube cancer, etc., It’s equally important to look at not just these primary rare disease diagnoses, but the fact that many common permanent side effects of anti-cancer treatments are rare diseases themselves. Examples include neuropathy, kidney disease, liver disease, etc. So even if a cancer patient is not diagnosed with a rare cancer, they will still likely be in need of a rare disease treatment during their treatment experience.
The ORPHAN Cures Act: Encouraging Innovation and Access for Rare Disease Treatments
The ORPHAN Cures Act has been proposed as a solution to address these gaps left by the IRA, fostering research for rare diseases while ensuring affordability. This act aims to adjust the IRA’s orphan drug policy to balance fair pricing with sustained innovation. By allowing Medicare to negotiate prices for orphan drugs under certain conditions, the ORPHAN Cures Act seeks to encourage pharmaceutical companies to continue their research and develop new treatments for rare diseases without sacrificing patient access to affordable medications.
The ORPHAN Cures Act aims to bring about meaningful change by removing the price negotiation exemption under specific conditions to balance affordability and access and by incentivizing drug companies to pursue more extensive research into rare conditions by removing the second indication penalties. This will help ensure fair pricing for patients who rely on orphan drugs for critical treatments, promoting equal access to life-saving therapies.
The ORPHAN Cures Act is crucial for patients and their families who rely on orphan drugs to manage life-threatening and often debilitating conditions. For these individuals, the price negotiation exemption has meant limited options, as treatment costs remain beyond reach or are only partially covered by insurance. By addressing the gaps in the IRA, the ORPHAN Cures Act empowers patients to access the medications they need to manage their conditions, helping to lift financial burdens while ensuring that innovation in rare disease treatment remains strong.
We Need Your Voice!
Your support for the ORPHAN Cures Act is vital to ensure that all individuals, regardless of their condition’s rarity, have access to affordable, effective treatments. Together, we can ensure that affordable, life-saving treatments are within reach for everyone, empowering more patients to thrive.
- Send an Email: Reach out to your Representative and urge them to co-sponsor the ORPHAN Cures Act (H.R. 5539/S. 3131) HERE.
- Share Your Story: If you or a loved one has been diagnosed with a rare disease, whether as a primary diagnosis (ex. ovarian cancer, cervical cancer, fallopian tube cancer) or experienced one as a side effect of anti-cancer treatment (ex. neuropathy, kidney disease, liver disease, etc.) your story will make a difference! Your personal story can help make the need for this bill real to Congressmembers and your community members. Share your story HERE.
- Schedule a meeting with your representative: Find a toolkit for reaching out, scheduling and having meetings with your congressmembers HERE.
