A Welcome Letter from Graham Owens

DECEMBER 14, 2023

Welcome Letter from Graham

By Graham Owens—Executive Director.

December holds special significance for me this year as I close out my first month as the RDCC Executive Director. I’m grateful for having been surrounded by so many incredible people who have welcomed me in, and I am looking forward to what comes next. But before we dive into 2024, let us first take a look back at all the Coalition accomplished over the past year.

This year, the RDCC carried out three campaigns to highlight our 2023 policy priorities. These campaigns focused on the need for protecting innovation, increasing access to therapies, and enabling earlier rare disease diagnosis. Each of these campaigns shed light on these critical issues and provided policy recommendations to help policymakers address the unique challenges and opportunities of the rare disease community. The RDCC is extraordinarily grateful for the support of long-time champions and new allies in rare disease policy this past year and looks forward to building on our strong foundation in 2024.

I am thrilled to lead the RDCC into the new year as we focus on our two policy pillars: promoting innovation and research & development, and enabling access to rare disease treatments. We will work to move the needle on critical issues like the Rare Disease Pediatric Priority Review Voucher Program reauthorization, consistent and appropriate flexibility at the regulatory level, and the ORPHAN Cures Act. I look forward to working with RDCC member companies and key stakeholders to harness opportunities and allay challenges for the rare disease community.

To everyone who has welcomed me over the past month, thank you, and to those who I haven’t already met, please reach out and say hello. I wish you all a wonderful holiday season and a happy new year!

Graham Owens

Executive Director

Learn More About RDCC Leadership

Looking Forward: Our Policy Priorities

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Supporting Robust Development and Innovation

This year, we celebrated the 40th anniversary of the Orphan Drug Act (ODA), a seminal piece of legislation that empowers biotech companies to invest in rare disease R&D that would otherwise be impractical or impossible to pursue. The ODA established a strong foundation for the incredible breakthroughs in rare disease research over the past 40 years - but there is much work to be done. The RDCC urges policymakers to protect the existing policies that provide incentives for rare disease R&D, and to establish new incentives that will facilitate additional breakthroughs. 95 percent of patients living with a rare disease still lack an FDA-approved treatment - and while our member companies remain committed to discovering, developing, and delivering new therapies — policymakers must protect and build on the policies that have enabled progress for rare disease patients, not erode them.

Ensuring Accessibility to Treatments

While it’s critical to protect the ability for rare disease companies to develop innovative new therapies, these treatments are only as effective as they are accessible. We must advance policies that facilitate timely access so patients can benefit from treatments as soon as they are available. From restrictive coverage policies, to inconsistent access to experts, and to employer-sponsored health plan discrimination, patients living with a rare disease face numerous obstacles to access the therapies they desperately deserve. The RDCC urges policymakers to remove barriers for patients and increase access equity for patients.

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In Case You Missed It – Risks of Rare

Our Q4 advocacy campaign was focused on “The Risks of Rare: Obstacles and Opportunities in Rare Disease Drug Development.” The campaign focused on the realities of rare disease drug development and the role public policy plays in catalyzing or stemming critical investments. The campaign highlighted findings from a recently-released white paper produced by the Health Capitol Group titled, “Rare Disease Companies in the Public Markets: Challenging Performance Against a Backdrop of Policy Uncertainty.”

Recent public policy changes at both the state and federal levels have substantially reduced investments in rare disease companies. This investment is critical for rare disease companies to survive - and without the necessary funding, rare disease companies will be unable to continue discovering, developing, and delivering treatments to patients living with a rare disease. A strong, predictable policy environment is necessary to offset the inherent risks of rare disease drug development and attract investors. The RDCC continues to urge policymakers to recognize these risks and reverse course on blunt policies that will have a devastating and disproportionate impact on already-underserved rare disease patients.

Earlier in the fall, the RDCC hosted a Congressional briefing on Capitol Hill, convening representatives from the research, investor, and patient communities to discuss how policy plays an integral role in incentivizing investment in rare disease R&D. Panelists emphasized that seemingly small policies and proposals can have hugely consequential impacts on the rare disease community, especially in times of such financial uncertainty.s.

Interested in learning more? A summary and full recording of the briefing has been made available here.

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