Our second newsletter for the month of November, 2021, features a breakdown of federal health policy developments from columnist James Capretta, our recent “5 Slides We’re Watching” conversation on gene therapy and rare diseases, and insight on addressing the youth behavioral health crisis. As always, Thanks for reading our stuff!   Eli Kirshbaum State of … Read more

Groundbreaking gene therapy treatments have tremendous potential to provide treatment to the around one in ten Americans living with rare diseases. But these game-changing therapies also bring with them new challenges, including how the individuals who need them most will get access to and be able to pay for these expensive treatments. To tackle this … Read more

Gene therapy has proven to be a promising treatment for rare diseases like muscular dystrophy. As these life-saving treatments enter the market, however, their high costs pose affordability challenges for individuals living with rare diseases.     In our upcoming virtual conversation, “5 Slides: Gene therapy and the promise for rare disease,” we will host … Read more

Rare disease treatment and gene therapy were the topics of a State of Reform 5 Slides We’re Discussing panel.  Gene therapy is a form of treatment where genes are inserted into the cells of a patient, instead of relying on drugs or surgery. The therapy may be especially promising for rare diseases.      Rare … Read more

Bhash Parasuraman, vice president of value and access for rare disease and internal medicine at Pfizer, joined State of Reform on Thursday for a conversation on the future of gene therapy.  As part of our virtual “Leadership Series,” Parasuraman discussed the outlook for gene therapy, the policies she’d like to see to support these treatments, … Read more

A new study suggests that people with rare diseases face much higher health care costs than those without a rare disease.  The study was led by the National Institute of Health and published on Oct. 21. It found that over a 15 year period, the cost per patient per year for a patient with a … Read more