Deb Jennings, Head of North America Patient Services Operations at Kyowa Kirin, discusses evolving policy landscapes for rare disease access.
In a panel discussion at the 2025 World Orphan Drug Congress, Ms. Jennings explained the volatility of the current environment in policy. Factors contributing to the current state include artificial intelligence (AI), tariffs, and uncertainty within the government, specifically the Department of Health and Human Services. With so many unknowns, she stressed the importance of controlling the things that you can, such as work effort, planning for change, and reactions to changes.
In Ms. Jenning’s opinion, one of the more pressing policy issues is accumulators and maximizers. Accumulators are benefit design plans within insurance companies, however these plans do not allow patients to utilize manufacturer copay cards and apply them to out-of-pocket costs. This creates access barriers to medication that is oftentimes life saving in the rare disease community. To combat this issue, Ms. Jennings describes Kyowa Kirin’s development of campaigns that educate patients and caregivers on this topic, therefore reducing barriers to access.
Another primary concern in the rare disease space is the use of artificial intelligence (AI). While digital and technological advancements are inevitable and necessary, Ms. Jennings discusses the importance of maintaining a human element. To truly understand patient needs and pain points and address such concerns, conversations with patients and caregivers is essential. However, AI can be useful in the aggregation of data which can help with matching patients to clinical trials, building information platforms for physicians and patients, and making information more easily understood. It is important for companies to be able to differentiate when to use a digital solution and when to use a human solution.
Finally, Ms. Jennings wants policy makers to understand why the rare disease community is so important. While individual diseases are rare, the rare disease community is composed of over 10,000 diseases and around 95% of these diseases do not have a targeted treatment. The development of treatments is important to the current population, as well as generations to come, since many rare diseases are genetic. She also believes it to be important to get rare disease patients in front of policy makers so that they can listen to and understand their stories to develop better solutions and see past the disease to the person.
For more information about Kyowa Kirin’s work in rare diseases, click here.
To learn more about rare disease advocacy, visit https://checkrare.com/rare-disease-advocacy-2/
