Dawn Rotellini, COO of the National Hemophilia Foundation, discusses how recent policy changes seeking to improve access to and create new therapies for patients with rare diseases.
The impact of changes in policy, programs, and attitudes towards rare diseases was on full display at the 2019 National Organization for Rare Diseases (NORD) and Orphan Products Breakthrough Summit.
With presentations and studies highlighting the impact of the US Food and Drug Administration’s efforts, including those of the Center for Drug Evaluation and Research, the ongoing progress was a hot topic of discussion throughout the meeting.
Yet even with talk of clinical trials and new data, the impact of these advances on patients is not lost in the shuffle. For patients and their families, the advancements in recent decades have resulted in life-changing therapies and altered the state of many rare diseases.
Patients with hemophilia and their care providers are a prime example. Decades ago, the outlook was bleak and the treatments that were available were often inaccessible and, sometimes, dangerous. For Dawn Rotellini, chief operating officer of the National Hemophilia Foundation, advances and pitfalls in the treatment of hemophilia is something that impacts her life in a professional and also a personal way.
For more on how the FDA’s recent push on for new rare disease therapies has impacted the lives of patients with rare diseases, MD Magazine® sat down with Rotellini between sessions at NORD Summit 2019.
MD Mag: How has the FDA’s recent push for new rare disease therapies impacted the lives of patients with rare diseases?
Rotellini: I guess from my perspective—so, I have a son with hemophilia and I had a dad with hemophilia and so looking at a lifespan going back decades of what does access to treatment and care look like in a very expensive disease— with the onset of federal changes for example the ACA, the Affordable Care Act, while people may dislike or like certain parts of that, what it did for our population was made sure that we had access to expensive therapies.
So, for example, prior to the ACA you would reach your lifetime cap in hemophilia by the time your severe son with hemophilia A was 5 or 6 years old. Lifetime cap—what does that mean for the family? That means you would have to change jobs. If that wasn't a possibility, you might have to get divorced, and those were things that people were recommending for these rare disease families with super high-cost medicines.
Once the federal government started looking at the gaps in coverage of these expensive diseases, it really opened up the possibility of coverage for every family—and certainly nothing is perfect but—it really did allow for no discrimination against pre-existing conditions, keeping your children on your policy until they're 26 years old, which helped in that college and then first job gap that you wouldn't drop off an insurance and, especially, if you have a rare disease that's life-threatening if you don't have your medicine. That allowed you to be able to go from young adult to prosperous young adults.
So, I think that some of those advancements and acknowledgments by the federal healthcare system have been crucial for keeping our population healthy and able to be productive citizens. That's really a big change in the last several years.