Haystack's comments on the small biotech exception to the drug negotiation provisions of the Inflation Reduction Act
/See our comments here
See our full comments here
If CMS determines to develop the NDH, we ask that it do so in consultation with patient groups, including rare and ultra-rare disease advocacy organizations, so that the end product is useful, accessible, and efficient for patients and their caregivers. Haystack expects that the types of information that will be most helpful to patients includes, at a minimum:
Provider name and contact information, including all practice locations
Complete licensure information o This should include all states in which a provider is licensed regardless of physical location, as recent advances in telehealth allow providers to treat across state lines
All networks and plans to which a provider is enrolled (and their status, i.e., participating in a plan, “accepting” reimbursement as payment in full, out-of-network provider willing to submit claims, etc.) o This should include Medicare, Medicare Advantage, Medicaid fee for service (and states for which Medicaid is accepted), Medicaid managed care (including Medicaid plans in other states) and all private insurance
Provider specialties and sub-specialties
Years in practice
Hospital affiliation • Whether new patients are being accepted
Average wait time for both new and existing patient appointments
The availability of telehealth services
Languages spoken
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
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The Haystack Project is a 501(c)3 nonprofit that brings together patients organizations representing patients suffering from or caring for patients with extremely rare diseases. Our mission is to educate policymakers and other stakeholders about the need for policies that recognize the unique circumstances of extremely rare conditions and treatments and expand incentives critical to ensuring they can reach patients.