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Nov. 18, 2021

TOPIC: Advocacy, Get Involved

With the Build Back Better Act, Congress Threatens Progress for Americans with Rare Diseases

Posted by Rohan Narayanan

As Congress continues to debate the policies to include in the final version of the Build Back Better Act, the House of Representatives has inexplicably chosen to take aim at one of the most vulnerable populations in our nation: the rare disease community. The Build Back Better Act, H.R. 5673, currently includes a harmful provision that threaten the decades-long success of the Orphan Drug Tax Credit (ODTC) and drastically reduce the therapeutic options available for the 25-30 million rare disease patients in America. 

Rare diseases have small patient populationswhich makes developing drugs to treat these conditions inherently more complicated and costly than for common medical conditions. Prior to 1983, there were only 30 drugs approved by the FDA for rare diseases. Simply put, the pharmaceutical industry was just not interested in making the investment necessary to treat rare diseases, only to find a small market once a drug was FDA approved 

Recognizing this market failure, Congress passed the landmark Orphan Drug Act, which established a clever mix of policies designed to make small patient populations a more attractive prospect for the pharmaceutical industry. One of the key incentives was the ODTC, which allows drug developers to write off significant portions of the clinical trial costs associated with rare disease drug development. The ODTC has been instrumental in encouraging drug companies to pursue clinical trials to prove that a drug is safe and effective for a rare disease, or sometimes multiple rare diseases. By most accounts, both the Orphan Drug Act and ODTC are working as intended. In stark contrast to before the passage of the Orphan Drug Act, today there are 652 drugs approved for 1,006 rare disease conditions.  

Still, the need for the package of incentives that became law in 1983 is as unmistakable today as it was 38 years ago1-in-10 people in the United States are living with one of the approximately 7,000 known rare diseases, many of which are life-limiting or fatal. More than 90% of these diseases have no treatment or cure that has been approved by the FDA. 

Yet, the latest version of the Build Back Better Act includes a provision that would severely diminish the ODTC by limiting its availability to only the first orphan use of a drug. It is not unusual for a single drug to be determined to be safe and effective for the treatment of multiple rare diseases. For rare disease patients, every time a treatment for a rare disease (or “orphan indication”) is approved by FDA, it is often life-saving progress. It helps patients and their health care providers have confidence that the drug they are utilizing is safe and effective for their condition. FDA approval can also make it easier to get coverage for the treatment and to lower patient out of pocket costs.  History has proven that there must be appropriate incentives, like the ODTC, to see the continued development drugs to treat rare diseases.  

While negotiations to include drug pricing provisions in the Build Back Better Act continue, tying the ODTC to the on-going drug pricing conversation is misguided and limited. To be clear, NORD shares concerns over the high cost of drugs in America, but attacking rare disease drug development, particularly an incentive that helps offset the high costs associated with clinical testing in rare disease patients, isn’t drug pricing reformIn its efforts to alleviate the burden of high prescription drug costs, Congress should focus on the true drivers of high drug costs, and not attack the incentives necessary for developing products for those who need them most: rare disease patients.  

On behalf of the nearly 30 million Americans with rare diseases and their families, NORD urges Congress to recognize the success of the Orphan Drug Tax Credit and preserve it in the name of protecting these individuals and their hope for a better tomorrowThere is still more work to be done, and rare disease patients cannot wait.  

Peter L. Saltonstall is president and CEO of the National Organization for Rare Disorders (NORD).