The U.S. Senate has a plan to improve drug development for rare disease patients. The exit of controversial CBER chief Vinay Prasad will help clear the path.

Original title: “Rare Disease Patients Can't Wait for Regulatory Process”

Rare disease biotech stocks pop on the news that Vinay Prasad, the FDA’s chief biologics regulator, will depart the FDA at the end of April; Sen. Ron Johnson launches an investigation into recent rare disease drug rejections; and Roche and Zealand’s amylin analog fails to match investor expectations—and Eli Lilly’s rival candidate—in a mid-stage trial.

Original title: “Prasad Out at FDA, Lawmaker Takes Action on Rare Disease Rejections and a Spate of Obesity Data”

Referred to the Committee on Energy and Commerce, and in addition to the Committee on Ways and Means, for a period to be subsequently determined by the Speaker, in each case for consideration of such provisions as fall within the jurisdiction of the committee concerned.

Original title: “HR 7666: SOS: Sustaining Outpatient Services Act”

Cell and gene therapies may provide life-extending treatments for patients. However, paying for these therapies using a single upfront payment will be challenging because of uncertainty about long-term clinical effectiveness and affordability. Developers, recognizing the challenges of paying ... Cell and gene therapies may provide life-extending treatments for patients. However, paying for these therapies using a single upfront payment will be challenging because of uncertainty about long-term clinical effectiveness and affordability. Developers, recognizing the challenges of paying for these therapies, have offered payers 5-year outcomes-based installment plans. Gene Therapy - Securitization as a means to pay for cell and gene therapies for orphan diseases: a simulation study Long-term PBAs, therefore, are primarily useful as a payment model for genetically driven orphan diseases, for which durable cell and gene therapies are likely to emerge. While the patient populations for individual orphan diseases are comparably small, the cumulative number of patients affected by the thousands of single-gene disorders is large, necessitating this proposed financing mechanism. FiercePharma. 2019. Retrieved 2025. from https://www.fiercepharma.com/pharma/insurer-response-to-novartis-2-1m-sma-gene-therapy-reflects-material-resistance-report. WHO. Genes and human diseases. Geneva: WHO;2019. Online Mendelian Inheritance in Man (OMIM®) OMIM Gene Map Statistics.

Original title: “Securitization as a means to pay for cell and gene therapies for orphan diseases: a simulation study | Gene Therapy”

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled "Flavored Electronic Nicotine Delivery Systems (ENDS) Premarket Applications--Considerations Related to Youth Risk; Draft Guidance for Industry." The draft document describes FDA's current thinking and provides recommendations regarding evidentiary considerations for premarket tobacco product applications (PMTAs) for flavored ENDS submitted under section 910 of the Federal Food, Drug, and

Original title: “Flavored Electronic Nicotine Delivery Systems (ENDS) Premarket Applications-Considerations Related to Youth Risk; Draft Guidance for Industry; Availability”

The Office of Child Support Enforcement (OCSE), Administration for Children and Families (ACF), is requesting the Office of Management and Budget (OMB) to extend approval of the Income Withholding for Support (IWO), with changes, for an additional three years. The current OMB approval expires August 31, 2026.

Original title: “Proposed Information Collection Activity; Income Withholding for Support”

The senator, who has long advocated for expanding access to experimental therapies, reportedly called the FDA’s request for a sham surgery–controlled Phase 3 trial for uniQure’s Huntington’s disease gene therapy “bureaucratic idiocy.”

Original title: “Senator Ron Johnson Launches Investigation Into FDA Rare Disease Rejections”

Industry and FDA representatives have reached a general agreement on planned pre-submission facility meetings but have expressed different views about the specifics.

Original title: “FDA Sets Scope for Attempt To Reduce Manufacturing-Related Approval Rejections”

Read twice and referred to the Committee on Finance.

Original title: “S 927: Protecting Pharmacies in Medicaid Act”

Read twice and referred to the Committee on Finance.

Original title: “S 1058: Preserving Patient Access to Home Infusion Act”

Referred to the Committee on Energy and Commerce, and in addition to the Committees on Ways and Means, and Education and Workforce, for a period to be subsequently determined by the Speaker, in each case for consideration of such provisions as fall within the jurisdiction of the committee concerned.

Original title: “HR 7496: Health Investment Zones Act of 2026”

Referred to the House Committee on Energy and Commerce.

Original title: “HR 6977: Better Care for PFAS Patients Act of 2026”

Learn how to improve referral, enrollment, and participation rates in cardiac rehabilitation programs.

Original title: “Cardiac Rehabilitation”

Original title: “Determination of Regulatory Review Period for Purposes of Patent Extension; AURORA EV-ICD”

This notice announces that the Centers for Medicare & Medicaid Services is considering granting approval of the National Committee for Quality Assurance's renewal application for Medicare Advantage "deeming authority" of Health Maintenance Organizations and Preferred Provider Organizations to continue participation in the Medicare program.

Original title: “Medicare Program; Request for Renewal of Deeming Authority of the National Committee for Quality Assurance (NCQA) for Medicare Advantage Health Maintenance Organizations and Preferred Provider Organizations”

The Food and Drug Administration (FDA, Agency, or we) is announcing the availability of a revised draft guidance for industry entitled "New and Revised Draft Q&As on Biosimilar Development and the BPCI Act (Revision 4)." The question and answer (Q&A) format is intended to inform prospective applicants and facilitate the development of proposed biosimilar products and proposed interchangeable biosimilar products, as well as describe FDA's proposed interpretation of certain statutory requirements

Original title: “New and Revised Draft Q&As on Biosimilar Development and the Biologics Price Competition and Innovation Act; Draft Guidance for Industry; Availability”

This notice announces our decision to approve the American Association for Accreditation of Ambulatory Surgery Facilities, DBA QUAD A, for continued recognition as a national accrediting organization for rural health clinics that wish to participate in the Medicare or Medicaid programs.

Original title: “Medicare and Medicaid Programs: Application From the American Association for Accreditation of Ambulatory Surgery Facilities for Continued CMS-Approval of its Rural Health Clinic (RHC) Accreditation Program”

The Office of Child Support Enforcement (OCSE), Administration of Children and Families (ACF), U.S. Department of Health and Human Services, is requesting the Office of Management and Budget to approve a new information collection, Central Authority Payment Service (CAP). Participating state child support agencies (CSAs)and foreign authorities will use CAP to send or receive child support payments electronically.

Original title: “Submission for Office of Management and Budget Review; Title Central Authority Payment Service (New Collection)”

Original title: “Center for Scientific Review; Cancellation of Meeting”

The Department of Health and Human Services, National Institutes of Health published a Notice in the Federal Register on February 25, 2026. That notice requires a correction in the SUPPLEMENTARY INFORMATION section.

Original title: “Proposed Collection: 30-Day Comment Request; The Clinical Trials Reporting Program (CTRP) Database (NCI); Correction”