FDA needs to be more flexible assessing rare

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The FDA needs to be more flexible in assessing treatments forrare diseases, like the one that seemed to help my son

By Karen Quandt Sept. 7, 2022 Maria Fabrizio for STAT

Every time I read about clinical trials testing possible treatments for rare diseases, I think of my son, Ty, whose brief but successful foray into such atrial highlights their value and their devastating limitations. Ty was born in Pocatello, Idaho, in October 1996 with green eyes, a big personality, a great sense of humor, and — as we finally learned eight yearslater — a rare and fatal disease called Niemann-Pick type C. He appeared to be healthy at birth, and hit all of his first developmental milestones of walking and talking and growing. Yet we realized that he hadtrouble moving his eyes upward, and had to move his whole head to look up. Then his fine motor skills and balance began to lag, and he had difficulty learning. An enlarged spleen complicated the picture.Ty was eventually diagnosed with Niemann-Pick type C.

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The Access to Rare Indications Act could be a game changer for millions of Americans

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By Darcy Krueger and Emanual Maverakis

June 1, 2022

The term “medically necessary” is the yardstick by which insurance companies, including Medicare and Medicaid, decide if they will pay for a particular treatment. For the millions of Americans living with rare diseases, most of which do not have FDA-approved treatments, identifying a treatment as medically necessary can be a lifeline — or the end of the line.

A white paper released in April by the influential Institute for Clinical and Economic Review offers several misguided proposals that attempt to balance public interest in incentivizing innovation with payer cost containment goals in rare diseases. The proposals are unlikely to benefit people living with rare diseases, clinicians like us who care for them, or those trying to bring rare treatments to market.

A far more thoughtful approach is embodied in the Access to Rare Indications Act (H.R. 6160), introduced in the House of Representatives by Reps. Doris Matsui (D-Calif.), Mike Thompson (D-Calif.), Mike Kelly (R-Penn.) and Markwayne Mullin (R-Okla.). The bill recognizes that the vast majority of people with rare diseases don’t have an FDA-approved treatment, meaning their disease is not listed on the treatment’s label, and their clinicians shouldn’t have to struggle to convince payers that medically accepted off-label treatments are medically necessary. Specifically, this bill seeks to give people with rare diseases the same level of insurance coverage for off-label treatments as patients with “common” conditions such as cancer and insomnia.

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Haystack Project's sign-on letter commenting to CMS' proposed NCA for monoclonal antibodies

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While the proposed NCA addresses new treatments for an all-too-common condition in Medicare beneficiaries, Haystack Project has serious concerns that it will, if finalized,

  • Represent an unprecedented challenge to the validity of the accelerated approval process that is integral to advancing new therapies in rare diseases

  • Perpetuate and exacerbate health inequities associated with race, ethnicity, and socioeconomic status - Impermissibly substitute CMS’ analysis of clinical evidence and conclusions for decisions delegated to and made by FDA, and

  • Raise significant ethical concerns by conditioning coverage for on-label use of the first FDA-approved treatment with potential to mitigate disease progression on participation in CMS-directed, randomized clinical trials.

The proposed NCA represents an unprecedented challenge to the validity of the accelerated approval process.

See the full comment letter here