An overview of the Evaluation Fellowship Program for potential applicants.

Original title: “About the Evaluation Fellowship Program”

The 21 studies involved 120 countries ... cross-border access in clinical trials in the field of pediatric rare diseases can, in some cases, be the only therapeutic opportunity available for patients with a rare disease condition that lacks approved treatment options.... The 21 studies involved 120 countries worldwide, with an average of 5.71 countries per trial and a standard deviation of 4.99 (Max: 15; Min: 1). Considering these figures, the likelihood of a patient in Europe or globally being able to participate in a clinical trial in their country of residence is quite low. Facilitating cross-border access in clinical trials in the field of pediatric rare diseases can, in some cases, be the only therapeutic opportunity available for patients with a rare disease condition that lacks approved treatment options. Cross-border access to clinical trials in Europe lacks specific regulation. Language diversity in Europe, with 24 official languages, is a key factor that must be considered when including international pediatric patients in multi-country studies. Providing translation enables patients to participate in clinical trials across borders. Providing translation enables patients to participate in clinical trials across borders. Specific consideration requires pediatric rare disease clinical trials for conditions with no approved treatment, where only a few countries participate, making cross-border access even more critical. Innovative and advanced treatments, such as gene and cell therapies, will soon require highly specialized clinical trial sites with expertise in managing these studies.26,27 This scientific trend also points to a reduction in the number of sites conducting such trials, emphasizing the need for the inclusion of international patients. Additionally, these studies often require long-term follow-up, sometimes up to 15 years. Establishing regulations or guidelines for cross-border access to these complex and long-term experimental treatments would be essential to ensure equal opportunities for all patients across Europe. The number of countries and sites involved in conducting pediatric rare disease clinical studies in Europe is limited.

Original title: “Cross-border access to clinical trials: participation of pediatric patients and language inclusion | Pediatric Research”

Richard Pazdur had spent 26 years at the FDA before stepping down last year due to disagreements with Commissioner Marty Makary about lowering the number of clinical trials required for a new drug application, Pazdur told the media.

Original title: “‘Just Agree to It:’ Pazdur Said He Was Told To Cosign FDA’s Reduced Trial Requirements”

This newsletter is a reader-supported publication. If you find value in this work, please consider becoming a paid subscriber. For the cost of a latte, paid subscriptions are the fuel that keeps me doing this work. Thank you for your support · Happy Rare Disease Month, lovely RDG readers an easy tool to find out. I started writing this when I was on dialysis. It’s intended to be both memoir and a practical tool to help folks who might be going through something similar or those caregivers and family supporting someone with a challenging diagnosis. NOTE: This is not intended to replace actual medical guidance. Some people ask me how they can support Rare Disease patients and helping us advocate is a HUGE way. But many of these issues aren’t just affecting folks like me, they also impact all patients.

Original title: “How does your state rate on Rare Disease Policy?”

The current state of political affairs in the U.S. does not bode well for the direction of that turn. The country is at real risk of losing its long-held lead in biotech innovation.

Original title: “2026 Could Mark a Turning Point for American Innovation”

In accordance with the Privacy Act of 1974, as amended, the Department of Health and Human Services (HHS) is partially modifying an existing system of records maintained by the Office for Civil Rights (OCR), "Program Information Management System (PIMS)," System No. 09- 90-0052. The modifications include changing the system of records name to "HHS Civil Rights and Health Information Privacy Program Records" and affect only certain sections of the System of Records Notice (SORN), so HHS is not re

Original title: “Privacy Act of 1974; System of Records”

Original title: “National Institute on Minority Health and Health Disparities; Cancellation of Meeting”

In compliance with the Paperwork Reduction Act of 1995, the National Institutes of Health (NIH) has submitted to the Office of Management and Budget (OMB) a request for review and approval of the information collection listed below.

Original title: “Submission for OMB review; 30-Day Comment Request; The National Institute of Mental Health Data Archive (NDA) Data Access Closeout Report, NIMH”

The Health Resources and Services Administration (HRSA) administers section 340B of the Public Health Service Act (PHS Act), referred to as the "340B Drug Pricing Program" or the "340B Program." HRSA is issuing this Request for Information (RFI) to gather input from interested parties regarding the potential use of rebates to effectuate the ceiling price under the 340B Program, including the standards and procedures that should govern the approval of manufacturer rebate plans and the impacts on

Original title: “Request for Information: 340B Rebate Model Pilot Program”

Referred to the House Committee on Ways and Means.

Original title: “HR 2085: Mental Health Research Accelerator Act of 2025”

Referred to the Committee on Education and Workforce, and in addition to the Committees on House Administration, and Oversight and Government Reform, 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 7531: To allow Americans to earn paid sick time so that they can address their own health needs and the health needs of their families.”

Referred to the Committee on the Judiciary, and in addition to the Committees on Energy and Commerce, and 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 7569: To increase the penalties for health care fraud, and for other purposes.”

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 1867: To amend title XVIII of the Social Security Act to remove in-person requirements under Medicare for mental health services furnished through telehealth and telecommunications technology.”

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 1922: Ensuring Access to Essential Drugs Act”

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 1899: Audio-Only Telehealth Access Act of 2025”

Ordered to be Reported by Voice Vote.

Original title: “HR 6808: To designate the facility of the United States Postal Service located at 417 West 7th Street in Columbia, Tennessee, as the "Pharmacist's Mate First Class John Harlan Willis Post Office Building".”

In their continued fight against major health care corporations, Senators Elizabeth Warren and Josh Hawley are aiming to delink ownership among PBMs, insurers, and wholesalers. Under these new rules, parent companies of prescription drug wholesalers would also be barred from owning medical providers or management services organizations. The impact on independent pharmacy operations has been a central theme in the push for this legislation. Since 2019, nearly 4000 independent pharmacies have shuttered their doors as massive conglomerates steer patients toward their own affiliated pharmacies and subsidiaries. These Big Medicine giants—including major companies like UnitedHealth Group, CVS Health, and Cigna—have created layers of complexity that allow them to squeeze independent practices out of business while increasing prices for patients, according to KFF Health News.4 · Mark Cuban, founder of Cost Plus Drugs, has also lauded the bill as a “no-brainer,” urging President Trump to put his weight behind the measure to lower costs for the public.3 Pharmacy practice is always changing. Stay ahead of the curve with the Drug Topics newsletter and get the latest drug information, industry trends, and patient care tips. This legislative push arrives as health care affordability reaches a crisis point for many Americans, with insurance costs consuming more than 10% of median family income in 19 states.4 The tension in the current system is further evidenced by ongoing contract disputes, such as the fight between Mount Sinai Health System and Anthem Blue Cross Blue Shield over hundreds of millions in unpaid claims, which has left patients uncertain about their access to critical cancer therapy. Beyond the legislation, other lawmakers are also exploring systemic changes, including potential overhauls to the Medicare physician payment system to address the concerns of medical societies seeking a successor to current incentive programs. The Break Up Big Medicine Act includes rigorous enforcement mechanisms to ensure compliance.

Original title: “Bipartisan Legislation Aims to Address Consolidation and Vertical Integration of PBMs | Drug Topics”

Ways and Means Committee legislation led by Congressman David Schweikert (AZ-01) to modernize the tax treatment of less-than-lethal public-safety tools... Ways and Means Committee legislation led by Congressman David Schweikert (AZ-01) to modernize the tax treatment of less-than-lethal public-safety tools passed the House of Representatives yesterday. The Innovate Less Lethal to De-Escalate Tax Modernization Act – which was included in H.R. Certain less-than-lethal devices, such as tasers, are currently defined under federal regulation as “firearms.” As such, they are subject to the Firearms and Ammunition Excise Tax (FAET) and the National Firearms Act of 1934 (NFA). The Innovate Less Lethal to De-Escalate Tax Modernization Act ensures critical less-than-lethal devices such as tasers and other innovative technologies are not subject to the Firearms and Ammunition Excise Tax, both promoting growth in the sector and keeping our communities safe. 2189, the Law-Enforcement Innovate to De-Escalate Act – updates outdated tax policy that currently treats certain less-than-lethal weapons, such as tasers, similarly to traditional firearms, a mismatch that can raise costs and slow adoption of life-saving technology.

Original title: “House Passes Ways and Means Legislation to Provide Better Access to Life-Saving Tools for Law Enforcement - Ways and Means”

This Week's Dose Senate committees continue to examine fraud in federal and state programs. Hearings this week focused on fraud affecting federally funded programs and taxpayer dollars in Minnesota and across the country. House Energy and Commerce Health Subcommittee holds hearing on healthcare ... This Week's Dose Senate committees continue to examine fraud in federal and state programs. Hearings this week focused on fraud affecting federally funded programs and taxpayer dollars in Minnesota and across the country. House Energy and Commerce Health Subcommittee holds hearing on healthcare affordability, prescription drug supply chain. Another weekly check-up on the healthcare industry, health legislation and regulation for the week ending on Feb 13: fraud investigations, affordability hearing, sen The bill would prohibit parent companies from simultaneously owning medical providers or management services organizations and pharmacy benefit managers or insurers, and would bar prescription drug or medical device wholesalers from owning provider entities. The legislation would require companies to divest prohibited ownership interests within one year of enactment and would impose automatic penalties for noncompliance. This bill builds upon on Sens. Warren and Hawley’s bipartisan Patients Before Monopolies Act by extending similar structural separation requirements to additional segments of the healthcare industry. The information collected through this RFI will assist HRSA in evaluating the operational, financial, and access to drugs for patients of a rebate model on covered entities, manufacturers, and other stakeholders across the drug supply chain.” It further states that, “With the information collected from this RFI, HRSA will evaluate if a potential 340B Rebate Model Pilot Program is in the public’s interest and, if so, determine a viable implementation strategy, consistent with the 340B statute.” Comments on the RFI are due March 19, 2026. Sens. Warren (D-MA) and Hawley (R-MO) introduced S. 3822, the Break Up Big Medicine Act to address vertical integration in the healthcare system.

Original title: “This Week in Health and Insurance Legislation and Regulation”

The FDA has lifted longstanding and the most severe warnings off of hormone replacement therapies for menopausal women.

Original title: “FDA removes box warnings for 6 menopausal therapies”