Throughout my health policy career, I’ve focused on advancing the frontiers of medical innovation through science and policy — in precision medicine, women’s health, and biopharmaceutical research. Since 2014, I’ve also been a rare disease patient with an illness called eosinophilic esophagitis (EoE), a chronic condition where an allergic reaction to foods causes the esophagus to narrow, making swallowing difficult and painful.
For me, EoE symptoms like the inability to swallow and food impaction in the esophagus are triggered by the milk protein (milk, cream, butter, cheese) and tree nuts, so I avoid them. When I poorly manage the disease, I have pain when eating that is similar to when you accidentally swallow a small bone and it scrapes down your throat. The pain can last for a few hours or reoccur when eating for a few days.
I’ve tried many treatments, but until May 20, 2022, when the Food and Drug Administration (FDA) approved Dupixent as the first-ever on-label medication for EoE, there were no medicines indicated for this condition. This is an exciting milestone, but the challenges in getting to this moment and the hurdles in accessing this new medicine are emblematic of the scientific, regulatory, and policy barriers facing patient communities with diseases that lack on-label treatments.
I haven’t felt the need to publicly share my struggle with EoE until now. Seeing provisions in the Inflation Reduction Act that could make it less likely for drugs like mine to reach patients motivated me to speak about the real-world implications of this (and other health policies) for patients.
Over the coming months, I will share a series of blog posts exploring public policy considerations from the perspective of a rare disease patient. This first post will focus on my health struggles and how the Inflation Reduction Act may curb industry investment in diseases like mine. I hope my story helps illustrate the implications of policy decisions on patients.
Few Options, Until Now
Being a rare disease patient can be extremely frustrating.
One incident stands out as emblematic of this frustration. After five years of poorly managed disease due to limited treatment options, I found myself with a pill stuck in my esophagus. When I could not get it out on my own, I went to the emergency room for medically assisted removal. After the pill was removed, I got a lecture about disease management. The same speech was repeated the next day at my doctor’s office. It felt like an endless, unfair refrain. How was I to manage a disease with few treatment options, all off-label? I can’t just stop eating.
With the first FDA-approved treatment for EoE, I now feel hope for the first time since my diagnosis. When I asked my doctor to prescribe the medicine, I told her that I tried to get into clinical trials — and failed — but that I understood the risks and benefits of the medication. She investigated the treatment and said she couldn’t imagine a better candidate for the drug. With great optimism, I took my first dose of Dupixent in September of 2022.
Policy Challenges Loom
Dupixent was first approved to treat atopic dermatitis in 2017. The drug has subsequently received approvals for asthma, chronic rhinosinusitis with nasal polyposis, EoE, and prurigo nodularis. It’s also received follow-on approvals that extend the population of patients eligible to receive treatment for these conditions to include, in some cases, children. To date, the FDA has approved Dupixent eight different times. Each of these approvals required the drugmaker to conduct one or more clinical trials, submit an application with all of their clinical data, and go through a lengthy review process. This is time consuming and expensive.
Each of these follow-on approvals conferred the drug company with additional exclusivity. This exclusivity helped the company justify the investment in conducting additional clinical trials for new indications and going through the FDA regulatory process because they could expect to recoup their investments in this critical development work.
However, the Inflation Reduction Act may curb industry investment in continued research on therapies already on the market by rendering that additional exclusivity meaningless through government price setting. The IRA will allow Medicare to “negotiate” the price of select prescription drugs that are more than nine years (for small-molecule drugs) or 13 years (for biological products) from their initial FDA-approval date. But it’s not uncommon for drugs to have important indications approved many years after the initial approval. Dupixent was approved for EoE five years after its initial FDA approval.
In addition, medicines are only exempted from price setting if they have a single orphan indication, meaning they are FDA approved to treat one rare disease. This lessens the incentive to do post-market research for patients with other rare diseases. As a result, time consuming and costly clinical development work in new indications, like EoE, may never be completed and rare disease patients like me, who could benefit from an existing drug, will miss out.
While a lack of investment in post-market research would not preclude a drug from theoretically being prescribed off-label, the absence of an on-label approval creates a myriad of access challenges for patients, including finding a clinician willing to prescribe a drug in the absence of clinical data and lack of insurance coverage for off-label uses. In my next blog, I will explore the many ways even “good” health insurance plans impose numerous barriers to patient access and the high costs of health care for patients with no treatment options.