As we approach the 40th anniversary of the Orphan Drug Act, the rare disease community is again facing a potential threat to the transformational progress this landmark legislation helped propel over the last several decades. Last year, as part of the efforts to pass a budget reconciliation bill, a policy proposal emerged that would negatively impact the rare disease community. While the reconciliation efforts did not advance, Congress is now discussing a revitalized reconciliation bill that could contain major changes to a core component of the Orphan Drug Act.

The proposal would change the Orphan Drug Tax Credit (ODTC), limiting the credit to apply only for clinical testing expenses for the first approval of a rare disease drug. This provision would fundamentally alter the rare disease pipeline and discourage companies from pursuing further clinical programs to test approved therapies in new disease areas. Investment in further research improves the lives of the most vulnerable rare disease patients and offers clinicians and insurers the evidence needed to guide treatment recommendations.

Our patient communities need more options, more hope, and more investment in rare disease research. The proposed change to the Orphan Drug Tax Credit will not result in any benefit to patients and stands to cause irreparable harm to the progress and innovation in rare disease therapeutic development.

As an advocate for patients with rare diseases, using your voice can make a difference. As the Congress continues to debate the reconciliation bill, we urge you to speak up and let them know the rare disease community does not want changes to the ODTC.

After taking action by emailing, calling and/or sending them a Tweet, your job is not done! The final step is to share the action alert with your family, friends, co-workers and any other people that might be interested in taking action on behalf of the rare disease community.  Thank you for joining with other rare disease advocates to fight this harmful change.