The EveryLife Foundation for Rare Diseases is grateful to Congress for their vision and leadership which has successfully spurred investment and innovation in rare disease therapy development since passage of the Orphan Drug Act in 1983. However, we strongly oppose the recently proposed changes to the Orphan Drug Tax Credit (ODTC) included in the Build Back Better Act passed by the House Ways & Means Committee which threatens to roll back this progress. We urge Congress to ensure this dangerous provision is removed from the final reconciliation package.
The proposed changes would result in potential savings at the expense of the more than 30 million Americans living with rare diseases, 93-95% of whom have no approved treatment options.
Limiting the Orphan Drug Tax Credit to the first approval 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 that wouldn’t otherwise be available.
There are countless examples of rare conditions that have benefited from investment in further clinical programs after one approval was obtained.. The below examples represent just 3 communities whose treatments would not be possible if the currently proposed changes to the Orphan Drug Tax Credit were enacted.
- Prader-Willi Syndrome: PW Syndrome is a pediatric-onset, genetic condition that affects many parts of the body and causes varied symptoms such as low muscle tone, developmental delays, cognitive impairment and others. PW patients have only one treatment option that targets just one symptom out of many. Pitolisant was approved for Narcolepsy in 2019. The drug addresses excessive wakefulness by targeting misfunction in the hypothalamus, something that also occurs in many with PW Syndrome. Following approval for narcolepsy, and at the urging of the PW Syndrome patient community, Pitolisant is now being studied in a PW Syndrome phase 2 clinical trial program, offering some hope to thousands of children and their families.
- Sickle Cell Disease: Between 70,000-100,000 people in the U.S. are affected by Sickle Cell Disease and left with limited treatment options for debilitating pain and other serious complications caused by the disease. Mitapivat is a drug first studied for the treatment of a rare hemolytic anemia, pyruvate kinase (PK) deficiency estimated to effect between 3,000 and 8,000 in the U.S. and the E.U. While still under review for the first indication, further clinical programs are underway for multiple types of thalassemia, one of which has no other treatment options. Studies are now being planned to evaluate the treatment for Sickle Cell Disease pain and anemia, possibly offering a novel, oral medication option for two of the most debilitating symptoms experienced by this underserved population.
- Neuromyelitis optica spectrum disorder (AQP4+ NMO): This rare, autoimmune condition leads to vision loss and paralysis, with each attack leading to further disability. Until 2019, there were no approved treatment options. Eculizumab is an antibody treatment first approved in 2007 for the treatment of another rare condition, Paroxysmal nocturnal hemoglobinuria (PNH). After the first approval, Eculizumab has been studied and approved for Atypical hemolytic uremic syndrome (aHUS) in 2011 and investment in another clinical program resulted in a third approval in 2019, offering AQP4+ NMO patients their first treatment option.
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. We urge Congress to ensure this dangerous provision is removed from the final reconciliation package.
