Isn’t it Strange?
by Joe Pugliese, President and CEO

Last year we developed a five-year plan for the Alliance activities to meet the needs of our members and the patients they serve. We recently met to review our progress to date and reaffirm that our plans for 2023 targeted the key issues. There was general agreement that we have the right programs in place or under development to address the challenges facing the members of the Hemophilia Alliance and the larger bleeding disorders community.

Regular readers of our newsletter know that the Alliance has steadily grown as a go-to organization for all parts of our community. What started in 1999 as a cooperative effort took root in 2006 and arguably took off in 2010. In 2022 and 2023, we have expanded our support for the community once again. We are adding more resources by adding two new Directors of Member and Community Relations, bringing our community facing number to 8. This is in addition to our administrative staff and our best-in-class consultants for Legal, Advocacy and Payer issues.

As I look towards the near future, there are three interrelated, difficult, and intractable issues that are clearly not in our control but desperately need to be addressed. All three have a direct and obvious impact on access to care.

1. The first of the year has long been a traditional time to raise prices and again this year, we saw price increases basically across the board. While pricing calculations are complicated, it is possible to show the effects of what are individually modest price increases. The Alliance recently modeled what a hypothetical HTC that purchases a representative cross-section of all types of products. This mythical but representative HTC purchased 17,639,354 i.u., mg or mcg in 2022. When you apply the increase in prices on all these purchases, it will cost an additional $2,327,936 to purchase the same products in 2023.
2. As we all remember from high school physics classes, every action has an equal reaction. I believe that ever-increasing prices are responsible in part for the ever-more common payer/PBM program like alternative funding programs, co-pay accumulator adjustor and co-pay maximizer programs, narrow preferred drug lists and formularies, and burdensome prior authorization processes. All of these raise the out-of-pocket costs of individual patients and families, make it harder for people to access the medication that their physician thinks will be most effective for them, and consume countless hours of wasted time for providers and patients.
3. Against this backdrop, last November, the FDA approved the first of many long-awaited gene therapy products for the bleeding disorders community. The community was and is very excited for this new option that has been 25+ years in the making. But the approval was also widely reported by virtually all media outlets. This isn’t because it was the first gene therapy product approved, but because it the most expensive drug ever brought to market . . . to date.

Today, we are already blessed with a broad array of excellent therapies and there are three more gene therapy products and at least three “novel” therapies likely to be approved in the next 12 months. While none of these products represent a cure, all continue a now decades-long trend of continuous improvement.

The challenge going forward will be to ensure that patients have access to these products and to federally-recognized HTCs. We will need vigilant and consistent enforcement of HRSA grant rules that require program income to be reinvested in maintaining and expanding the care for the bleeding disorders community, as well as policies to ensure that HTCs can maintain control of their patients, who are at risk of being poached by other covered entities.

We need to address these issues in the very near term if we want to preserve the clinical services the community needs today and for the foreseeable future.