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HFA and NHF co-hosted a Safety Summit in Washington, D.C., over three days at the end of January. The summit was convened in recognition and response to community concerns arising from a series of drug safety reports in 2019. Those safety incidents rocked the hemophilia community ‚Äď and revived memories of the community‚Äôs tragic experiences with HIV and HCV infection through tainted clotting factor products.

The Safety Summit‚Äôs mission was to ‚Äúarticulate expectations for monitoring, informing, educating, and communicating issues related to product safety,‚ÄĚ with attention to the role of each stakeholder in the process. Eighty-five stakeholders participated in the summit, including patients, patient organizations (national, international, and state-level), clinicians, manufacturers, specialty pharmacy, federal health agencies, and more.

The summit kicked off on Wednesday, Jan. 29, with a keynote address by Dr. Joseph Schwarcz of McGill University, titled ‚ÄúSense, Nonsense, and Science.‚ÄĚ It continued with two days of presentations, panel discussions and workshops, addressing safety notification standards and processes (what they are now, where gaps exist, and how safety communication practices could improve); rights, roles and responsibilities of different stakeholder groups with respect to product safety and safety reporting; and new considerations that come into play as novel and gene therapies come onto the market.

Follow up to the safety summit will include a preliminary report out to the bleeding disorders community followed by an open comment period for community input. After the completion of a written report development of educational resources to address identified gaps will commence.

Quick Hits:

  • HFA joined with 19 other patient and health advocacy groups to file an amicus curiae brief with the U.S. Supreme Court, urging the court to grant expedited review in the case of Texas v. United States. The case is the latest court challenge to the Affordable Care Act. The patient advocates were disappointed to learn that the Supreme Court turned down the ACA defenders‚Äô request for expedited review on Jan. 21. Three days later, though, the Supreme Court also turned down the opposing parties‚Äô request to delay submitting their briefs in the case ‚Äď meaning that it is still possible that the Supreme Court will consider the case through its regular process. In the meantime, the ACA continues in effect while the litigation is pending, and patient advocates remain committed to protecting Americans‚Äô access to affordable, quality health care.
  • HFA, together with NHF, filed comments opposing the Social Security Administration‚Äôs proposal to subject certain disabled individuals to more frequent continuing disability reviews (CDRs), as a condition for remaining eligible for disability benefits. The comments noted that the agency had offered no evidentiary justification for the more frequent CDRs, and that this action would potentially terminate critical benefits for millions of disabled Americans.
  • On Jan. 27, the Supreme Court ruled that the U.S. Department of Homeland Security can begin enforcement of the Administration‚Äôs new ‚Äúpublic charge‚ÄĚ rule. This rule says that noncitizens can be penalized when they apply for green cards if the government believes they are likely to become a ‚Äúpublic charge‚ÄĚ by virtue of their use of certain safety net programs, including Medicaid. HFA reminds community members that many people are exempt from the rule; not all benefits count as negative factors; and every situation is different, so if you have questions about your own case, you may want to consult with an immigration lawyer. You can find additional information about the public charge rule (in English and in Spanish) here.
  • On Jan. 28, the U.S. Food and Drug Administration published Guidance for Industry on Human Gene Therapy for Hemophilia. This document lays out the FDA‚Äôs recommendations for manufacturers that are developing human gene therapy products for the treatment of hemophilia, including recommendations for clinical trial design and for the development of appropriate factor activity assays. HFA and NHF had provided comment to FDA on the draft version of this guidance in December 2018.
  • The U.S. Centers for Medicare and Medicaid Services (CMS) published guidance on Jan. 30 laying out a pathway for state Medicaid programs to apply for block grants or per capita caps through Section 1115 demonstration waivers. CMS portrayed this as ‚Äúa new opportunity for states to potentially achieve new levels of flexibility in the administration and design of their Medicaid programs,‚ÄĚ but HFA and other patient and consumer groups issued a public statement warning that implementation of the CMS proposal would reduce access to quality and affordable health care for vulnerable individuals served by the Medicaid program.
  • On Jan. 21, the New Jersey legislature enacted A2431, a bill requiring state-regulated health insurance plans to offer at least one plan with a capped copay (rather than coinsurance) for a 30-day supply of any single drug. The enactment of the bill represents the culmination of a five-year long effort.
  • Grassroots advocates in Oklahoma met the requirements to put Medicaid expansion on the ballot for 2020. Following the release of the CMS block grant guidance (above), Oklahoma‚Äôs governor announced that he would seek to expand Medicaid via the new block grant pathway that could allow states to limit benefits. Also in January, new Medicaid expansions went into effect for Idaho and Utah, and Kansas’s governor reached agreement with Republican leaders and state hospital associations on plan to expand Medicaid under the ACA for 2021.


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