Published April 19, 2021
On April 8, 2021, Marc Yale, IPPF Advocacy & Research Coordinator, issued a statement during the Medicaid and CHIP Payment and Access Commission (MACPAC) public meeting. MACPAC is a non-partisan legislative branch agency that provides policy and data analysis and makes recommendations to Congress, the Secretary of the U.S. Department of Health and Human Services, and the states on a wide array of issues affecting Medicaid and the State Children’s Health Insurance Program (CHIP). Thank you to Haystack Project for working with the IPPF on the implications for our community and helping with our remarks. We asked the commission to reconsider their recommendations to increase rebates for manufacturers who use the FDA Accelerated Approval Pathway. MACPAC’s recommendations have the unintended consequence of disproportionately harming ultra-rare patients like us.

The following is the prepared statement Marc Yale presented at the MACPAC public meeting.

Public Statement

My name is Marc Yale. I’m the Advocacy & Research Coordinator for the International Pemphigus & Pemphigoid Foundation. I also serve on the Board of Directors for Haystack Project.

Through my own personal experience as a rare disease patient living with pemphigus, as well as my involvement in advocacy for pemphigus patients and others living with extremely rare conditions through Haystack Project, I can tell you that accessing treatment can be an immense undertaking. Pemphigus and pemphigoid have been treated “off-label” with a variety of different therapies that have varying degrees of success in helping control our disease, and payers can make getting access to off-label treatments extremely challenging. We are now one of the handful of ultra-rare conditions with an FDA approved treatment- Rituxan, which is now considered a first-line therapy for pemphigus vulgaris.

  • There is no question that manufacturer incentives – like FDA Priority Review, Breakthrough Therapy Designation, and Orphan Drug Designation were vital to getting this therapy as quickly as possible to people with pemphigus who struggle every day to control their symptoms.  
  • We were fortunate that Rituxan has been on the market since its approval in 1997 and that, due to the nature of our condition, clinical trials could be completed without surrogate endpoints.  
  • The proposal to create an additional rebate for accelerated approval, though, could have been a significant deterrent to developing a new product in treating pemphigus.  
  • The proposal also raises questions that do not have good answers from a policy perspective. 
    • For example, if Rituxan’s pemphigus indication were achieved through accelerated approval, would an additional rebate apply? 
    • And would it only apply to the indication?  
    • It would seem that the proposal would decrease research rather than encourage post-market studies, especially when medically accepted off-label uses would be covered without the additional rebate.

Placing a disincentive on a pathway that is designed to encourage early access to promising treatments could have the greatest impact on ultra-rare disease patients. And your proposal today is basically a vote against investment in patients like us.