People impacted by rare diseases face significant hurdles including limited treatment options, difficulty accessing specialized care, and quality of life issues. An effective policy environment that prioritizes innovation and considers the specific needs of these individuals is essential to improving their outcomes.

Connie's Story: A Personal Perspective
Take Connie Morales, a 27-year-old aspiring author living with thymidine kinase 2 deficiency (TK2d), an ultra-rare mitochondrial disorder characterized by progressive muscle weakness. In addition to her physical limitations including the need for a wheelchair and a ventilator as well as difficulty speaking, Connie needs intellectual, emotional, and independent living support.

Connie’s story is unfortunately not unique. With an estimated 30 million Americans living with rare diseases, 95% of which lack FDA-approved treatments, these challenges are widespread. The feeling of being forgotten and unheard compounds the difficulties they face.

Addressing the Hurdles: Shaping the Policy Landscape
A supportive policy environment is vital for fostering innovation and promoting access to treatments for people impacted by rare diseases. While the Orphan Drug Act of 1983 made significant strides, the current landscape remains challenging.

Jed Perry, Head of U.S. Public Policy and Government Relations at UCB, shares his insights about policy solutions to improve outcomes for people impacted by rare diseases.

How does UCB determine which policies to advocate for to best improve patient outcomes?
At UCB, we focus on addressing the needs of people impacted by rare diseases by continuously listening to and learning from patient and caregiver sessions. These insights help us identify unmet needs and develop solutions, ensuring we cater to this underserved community. By prioritizing patient experiences, we aim to improve access to treatments and support, empowering individuals like Connie.

We are launching the Aspire 4 Rare Report, which outlines actionable policy solutions and provides a global framework for systematic change.

What is a policy priority for 2025?
We are actively advocating for the Joe Fiandra Access to Home Infusion Act, which provides Medicare reimbursement for home-administered infusions by healthcare professionals. Many individuals with rare diseases face physical limitations that make traveling for treatment difficult. This legislation would be life-changing, providing at-home options that increase access and improve quality of life.  Last year, the U.S. House of Representatives passed the bill. Our goal is to pass the bill into law in 2025.

How does the Inflation Reduction Act (IRA) impact individuals with rare diseases?
The IRA’s Medicare Drug Price Negotiation program exempts drugs with a single orphan indication from negotiation, but drugs with more than two or more orphan indications lose that exemption. The narrow exclusion for orphan drugs poses challenges by potentially stifling innovation. To counter this, we support the ORPHAN Cures Act, which aims to amend the IRA to ensure orphan drugs treating one or more rare diseases or conditions are excluded from Medicare price negotiations, which would support investment into orphan drug development.

What is UCB doing to advocate for this bill?
UCB has joined the Save Rare Treatments Task Force to amplify our advocacy for rare disease support. We engage in initiatives like Capitol Hill meetings to raise awareness about the importance of supporting individuals who live with rare diseases. Most recently, UCB’s executive leadership team met with key members of Congress to advocate for policies that support investment in treatments for rare diseases. Our goal is to involve more patient organizations and educate Congress about the bill, aiming to passing it in the next Congress.

Are there other rare disease policy priorities this year?
In addition to the ORPHAN Cures Act, Congress must act to ensure the Orphan Drug Act (ODA) is not deluded. The legislation is crucial for development of drugs to treat rare diseases by incentivizing support for research and innovation by providing tax credits, waiving the Prescription Drug User Fee and extending market exclusivity for 7 years. Any reduction of these incentives could have detrimental effects on rare disease research and treatment development.

Any closing thoughts?
We are privileged to contribute to the cause of rare diseases and strive for better outcomes. By empowering patients like Connie and advocating for supportive policies, we aim to create a landscape where every individual has access to the treatments and support, they need to thrive. Together, we can drive positive change and improve the lives of millions affected by rare diseases.