Federal Policies Must Incentivize Rare Disease Drug Development, Not Hinder It
For most of my daughters’ lives, I lived with the reality that any day could be their last. After they were both diagnosed with cystic fibrosis (CF), our lives centered around constant check-ups, testing, and a revolving door of pills, inhalers, and other medications to manage their symptoms. Many of the treatments my daughters relied on to go about their lives and help mitigate their symptoms were small molecule drugs. Small molecule drugs account for the majority of drugs approved by the U.S. Food & Drug Administration and treat a variety of diseases, including many cancers, HIV, psychiatric disorders, and other chronic conditions like CF that require consistent treatment.
Trikafta, a small molecule treatment regimen for CF, drastically improved my daughters’ quality of life when it came to market. Before beginning treatment, my daughter Emily couldn’t run more than a quarter mile due to her CF symptoms, including chronic coughing, wheezing and shortness of breath. The first time she went for a run after starting Trikafta, she went out hoping to run one mile. She returned home after running five miles with ease, wondering why everyone didn’t run like that every day.
Small molecule drugs are often oral medications taken in pill form, which provide greater flexibility to patients as they are easy to access, administer, and store. Biologic medications (or large molecule drugs), on the other hand, must be infused or injected at a hospital, doctor’s office, or clinic, increasing barriers for patients who may be unable to travel to receive care due to physical, medical, or financial restraints.
Recognizing the challenges that American families face to access medications following complex diagnoses, federal lawmakers are advancing policies that seek to lower drug costs for patients. In 2022, the Inflation Reduction Act (IRA) was signed into law, empowering Medicare to negotiate drug prices directly with drug manufacturers. The law includes a specific provision to determine the timeline for these negotiations based on whether the treatment is a small or large molecule drug. While it may be well-intentioned, this timeline fails to consider the impact on drug discovery and threatens to devastate patient access to life-saving treatment options.
Within the broader price negotiation policy under the IRA, small molecule drugs will become eligible for price negotiation 9 years after approval from the FDA, while large molecule drugs will become eligible for price negotiation 13 years following FDA approval. This arbitrary distinction raises significant alarms for the future of small molecule drugs.
By providing biologics an additional four years on the market before becoming eligible for negotiation, the IRA disincentivizes critical investment into small molecule drugs, which are vital to supporting the health of vulnerable patients, including patients living with chronic and rare diseases. Consider my daughters. I wonder how the IRA could have upended research efforts on Trikafta if it were law just a few years ago.
Now consider the countless patients and their families who are still waiting for a treatment or cure. Over 7,000 rare diseases impact up to 30 million Americans. Of those diseases, only 500 have an approved treatment.
In order to recoup their original investment and re-invest in the development of new breakthroughs, drug manufacturers expect their products to have a certain amount of time on the market without negotiation. By granting large molecules a longer period on the market before negotiation, the IRA will likely stifle innovation of new small molecule medications. If investment into small molecule drugs decreases, existing barriers to care will be greatly exacerbated and patients will suffer.
Without access to innovative small molecule drugs like Trikafta, my daughters and thousands of other patients living with CF and other chronic diseases would be unable to lead healthy lives. As federal policymakers consider solutions to alleviate the burden of high out-of-pocket costs, they must hear the voices and concerns of patients and their loved ones around policies that would negatively impact the development of lifesaving treatments. Because we should be increasing opportunities to bring lifesaving drugs to market, not limiting them.
Laura Bonnell is the CEO of the Bonnell Foundation, a non-profit organization dedicated to supporting families affected by cystic fibrosis, and the mother of two daughters living with cystic fibrosis.
