Prioritize patients over price controls

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Maryland is a national leader in medical innovation and is at the forefront of discovering new treatments and cures for many chronic and rare illnesses, including HIV, cancer, cystic fibrosis, and more. Patients with rare and chronic diseases like my daughter, Grace, are depending on our nation’s longstanding commitment to meet unmet patient needs and advanced medical innovation has given countless patients, families, and caregivers hope that a new treatment or cure is on the horizon.

However, federal and state policymakers are continuing to prioritize imposing price controls on critical medications that will bring this much-needed innovation to a screeching halt and abandon patients in need.

One in 10 Americans is living with a rare disease or a condition that affects fewer than 200,000 people in the United States. There are more than 7,000 rare diseases affecting about 30 million Americans and more than half of all rare disease patients are children.

At 8 years old, Grace was diagnosed with Pediatric Sjogren’s, a chronic autoimmune disease where the body’s own immune system mistakenly attacks its own cells and tissues. While more common in adults, Sjogren’s is extremely rare in children and Grace was left with little options for care.

A decade later, Grace’s condition is manageable today, yet there still remains no treatment or cure for Sjogren’s that are geared towards children. Some children are able to take the medications intended for adults, but not every patient has that option.

In addition, there are few clinical trials in this space, and providers who specialize in this disease vertical are few and far in between. This reality is not unique to just Grace’s illness – nearly 95% of all rare diseases have no treatment.

We’ve already seen groundbreaking advancements in treatment development for patients living with rare diseases. Prior to the passage of the Orphan Drug Act, legislation that incentivized in the development of treatments for rare conditions, there were only 38 treatments approved by the Food and Drug Administration for all rare diseases.

Today, there are more than 550 medications that treat more than 1,000 rare disorders – and thousands of Americans, including my daughter, who are holding out hope that a treatment for their illness will be developed next. There are nearly 800 new medicines currently in development for rare diseases, including 51 for autoimmune diseases.

However, despite the vast unmet patient need that persists, federal and state legislators continue to move forward with policies that will make it more challenging for patients with rare and chronic conditions to access much-needed and, in many cases, life-saving treatments and services, as well as disincentive future medical innovation that many rare disease patients have been waiting their whole lives for.

In 2019, Maryland established the nation’s first Prescription Drug Affordability Board, or a board made up of independent bureaucrats tasked with identifying and examining prescription drugs that pose affordability challenges for patients. Although well-intentioned, this board can actually do more harm than good for patients by blocking access to treatments that are already working for patients and disrupting the very ecosystem that enables patients to live longer, healthier lives.

Nine other states, such as Maine, New Hampshire, and Colorado have established PDABs, with Colorado’s board already selecting five medications to conduct affordability reviews on, including a rare disease medication.

In addition, as part of the Inflation Reduction Act signed into law in 2022, 10 drugs, including cancer medications, were selected for price negotiations by Medicare. Patients across the country are continuing to raise their voice on the impact that these policies will have on access to crucial medications and future investments in drug development, yet our lawmakers are continuing to prioritize arbitrary price controls on life-saving medications over the very patients who rely on such innovations.

I hold out hope that my daughter, and countless other children and adults living with a rare illness, will see treatments for the thousands of conditions that don’t yet have a solution. But this dream will not be fully realized until our federal and state policymakers choose to stand with patients and protect the pipeline of future medications. We can’t fall back on rare disease innovation – too many lives are depending on it.

Sharon Tiger is a Maryland mother of a child living with a rare disease and member of The Bonnell Foundation.