Policy strategist Kristi Martin discusses how the Orphan Drug Act reshapes rare disease treatment and drug-pricing challenges. In this episode, Kristi Martin, a longtime Washington, DC–based policy strategist with deep experience implementing major legislation like the Affordable Care Act and the Inflation Reduction Act, explores how the Orphan Drug Act, passed in 1983 as a bipartisan, patient-driven effort, transformed · rare disease treatment by aligning scientific advances, government investment, and market incentives. equity challenges as the Orphan Drug Act intersects with modern drug pricing policy, including the shielding of high-spending orphan oncology drugs from negotiation. While acknowledging the law’s life-saving impact, Martin argues that reform may now be necessary—rethinking incentives for blockbuster drugs, refining orphan drug eligibility, and shifting toward more targeted, evidence-based negotiation—while emphasizing the federal government’s continued role in sustaining innovation through agencies such as the NIH, FDA, and CMS.

Original title: “The Orphan Drug Act in the Modern Drug Pricing Era | AJMC”

Today, the U.S. Food and Drug Administration took additional steps to support the transition of our nation’s food supply from the use of artificial petroleum-based colors to alternatives derived from natural sources.

Original title: “FDA Takes New Approach to "No Artificial Colors" Claims”

Regulatory challenges have been even more top of mind than usual given recent upheaval at the FDA. BioSpace spoke to three industry experts about key issues, which include applying new artificial intelligence guidance. The experts also shared advice for working with regulators.

Original title: “3 Top Challenges Facing Regulatory Professionals Right Now”

Bill S-5 would set common standards for tech companies transferring health data The federal government is introducing legislation to make it easier for physicians and patients to share medical records and other types of health data. Bill S-5, the Connected Care for Canadians Act, tabled in the Senate Wednesday, is similar to Bill C-72, which was introduced by the previous Liberal government in 2024 and subsequently died when Parliament was prorogued ahead of last year’s election. Margot Burnell, president of the Canadian Medical Association, the federal lobby group for physicians, said the inability to easily share records between health care providers could lead to poor care for patients. Patients can struggle to access their own medical records, which may reside in doctor’s offices, hospitals, pharmacies and other locations. Sharing records with each other can be difficult. She cited the example of a patient who goes to an emergency room with pain, is referred to a cardiologist, and then is sent back to their family physician for ongoing care.

Original title: “Canada introduces legislation to create a more connected health care system - The Globe and Mail”

View comprehensive data on unintentional and undetermined intent drug overdose deaths.

Original title: “SUDORS Dashboard: Fatal Drug Overdose Data”

Doxycycline is one of the main antibiotics used to help prevent anthrax after people are exposed.

Original title: “Emergency Use of Doxycycline”

Learn to prepare an antibiotic mixture during an anthrax emergency.

Original title: “Preparing Doxycycline Hyclate for Non-Pill Swallowers”

CDC Disease Detective Camp: Frequently Asked Questions - David J. Sencer CDC Museum

Original title: “CDC Museum Disease Detective Camp: Frequently Asked Questions”

The McDermott+ team dives into recent pharmacy benefit manager (PBM) reform legislative and regulatory activity. PBMs function as intermediaries between group health plans and insurance providers, on one hand, and pharmaceutical manufacturers and pharmacies, on the other. They are one of many players in the complicated drug supply chain that have received attention from policymakers. To help me dive into recent PBM reform legislative and regulatory activity, I’m bringing in my colleagues Amy Kelbick and Lynn Nonnemaker. Finally, Section 6702 requires commercial PBMs to remit 100% of all rebates, discounts, fees, and other remuneration received from any applicable entity related to utilization of drugs or drug spending to the group plan sponsor on a quarterly basis. This section holds harmless any responsible plan fiduciary that did not know that the covered service provider acting as a PBM failed to make required remittances and directs that the service provider take steps to remit the required amounts. While the law was enacted on February 3, 2026, DOL issued a proposed rule on January 29, 2026, to require commercial PBMs to report on several transparency measures to plan fiduciaries of self-insured group health plans subject to the Employee Retirement Income Security Act (ERISA). The rule would implement section 12 of President Trump’s Executive Order 14273, “Lowering Drug Prices by Once Again Putting Americans First,” which instructs DOL to propose regulations to “improve employer health plan fiduciary transparency into the direct and indirect compensation received by [PBMs].” Instead, PDPs are allowed to pay PBMS a “bona fide service fee” that reflects fair market value for an itemized service performed on behalf of the PDP that the PDP would otherwise perform, and that is not passed on to a client or customer. The fee must be a flat dollar amount and cannot be based on or contingent upon drug prices, discounts, rebates, fees for covered drugs, or coverage or formulary placement decisions.

Original title: “PBM reform: The intersection of legislation and regulations - McDermott+”

By signing a government funding bill that ended a partial shutdown, President Donald Trump has also reauthorized a beloved program meant to speed the development of new drugs for rare childhood dis | By signing a government funding bill that ended a partial shutdown, President Donald Trump ... By signing a government funding bill that ended a partial shutdown, President Donald Trump has also reauthorized a beloved program meant to speed the development of new drugs for rare childhood dis | By signing a government funding bill that ended a partial shutdown, President Donald Trump has also reauthorized a beloved program meant to speed the development of new drugs for rare childhood diseases. Congress has struggled over the last year to bring back the beloved program, which initially expired at the end of 2024. “The passage of the Mikaela Naylon Give Kids a Chance Act is a bipartisan victory for children and families living with rare diseases,” John Crowley, president and CEO of the Biotechnology Innovation Organization, said in a Feb. The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon Give Kids a Chance Act and reviving the rare pediatric disease (RPD) priority review voucher program. The program will now be funded through September 2029.

Original title: “FDA’s rare pediatric disease voucher program revived by 2026 government funding bill”

The Orphan Drug Act revolutionized rare disease treatment, yet rising costs and access issues challenge equitable health care solutions. Policy debates now center on whether the Orphan Drug Act’s incentives remain aligned with unmet need, leaving patients, their caregivers, and their care teams to navigate a patchwork of prior authorization requirements, benefit exclusions, and inconsistent definitions of medical necessity. Diagnostic innovations, such as the ability to now identify BRCA-positive cancers, permit more distinct disease classifications, making oncology fertile ground for orphan drug designations with improved mortality trends and marking a sharp contrast to the decades before the Orphan Drug Act’s passage in which effective cancer treatments were limited, especially for pediatric patients. The ORPHAN Cures Act provision of the new bill included 2 significant changes regarding this: Drugs can have more than 1 indication for a rare disease or condition, and a rare drug will only become eligible for price negotiation starting with the date of the first nonorphan indication. Drilling down to Medicare Part B and Part D for patients with blood cancers, Waters noted, the updated provision under the ORPHAN Cures Act means that drugs for these patients that may have become eligible for negotiation no longer would be. More than 40 years after its passage, the Orphan Drug Act has fundamentally reshaped the rare disease landscape—fueling scientific innovation, expanding therapeutic pipelines, and transforming once-neglected conditions into active areas of research and development. But its success has also exposed fault lines: rising prices, uneven access, and policy frameworks that struggle to accommodate small, heterogeneous patient populations.

Original title: “Is Rare Disease Policy a Stress Test of US Health Care? | AJMC”

Read twice and referred to the Committee on Health, Education, Labor, and Pensions. (text: CR S443)

Original title: “S 3757: A bill to amend the Higher Education Act of 1965 to establish fair and consistent eligibility requirements for graduate medical schools operating outside the United States and Canada.”

Rep. Jake Auchincloss of Massachusetts said the Commissioner’s National Priority Voucher program did not receive congressional backing. The FDA has also not yet made disclosures for eight senior reviewers, according to Auchincloss.

Original title: “FDA's Commissioner’s Priority Voucher Program ‘Shrouded in Secrecy,’ Democratic Rep Says”

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 7324: To amend title XVIII of the Social Security Act to expand eligibility for incentives under the Medicare health professional shortage area bonus program to practitioners furnishing mental health and substance use disorder services.”

Ordered to be Reported in the Nature of a Substitute (Amended) by the Yeas and Nays: 37 - 3.

Original title: “HR 5343: Ensuring Patient Access to Critical Breakthrough Products Act”

Preventing anthrax before and after exposure to the disease.

Original title: “Prevention”

Some jobs and activities put people at risk for anthrax, but there are ways to lower risk.

Original title: “People at Increased Risk for Anthrax”

Take steps to be ready if there's ever an anthrax emergency.

Original title: “Bioterrorism and Anthrax: The Threat”

Editorial and production staff for CDC’s Morbidity and Mortality Weekly Report (MMWR).

Original title: “<em>MMWR</em>  Staff”

Washington, DC, Feb. 03, 2026 (GLOBE NEWSWIRE) -- National Association of Specialty Pharmacy (NASP) President and CEO Sheila Arquette, R.Ph. and Chairman... Specialty pharmacies serve communities of patients who have complex health conditions like cancer, rheumatoid arthritis, multiple sclerosis, cystic fibrosis, hemophilia, organ transplantation and rare diseases. Specialty pharmacies are accredited by an independent, third party nationally recognized accreditation organization ensuring consistent quality of extensive drug management and clinical patient care services. NASP has long advocated for this law, which we believe will stabilize and strengthen the specialty pharmacy infrastructure that complex therapies depend on—supporting innovation, adherence, and improved long-term outcomes for patients with cancer, autoimmune diseases, rare disorders, and other serious conditions,” said Scott Guisinger. For too long, specialty pharmacies have faced underwater reimbursement, opaque practices, and restrictive networks that have forced many qualified providers out of network and disrupted continuity of care for some of the sickest patients in America. This landmark federal action represents the most significant step yet toward ending those practices,” said Sheila Arquette. 22, 2026 (GLOBE NEWSWIRE) -- The National Association of Specialty Pharmacy (NASP) applauds the House for taking decided action today to enact reforms to support Medicare... Read More · January 21, 2026 15:31 ET | Source: National Association of Specialty Pharmacy (NASP) Pharmacist and Pharmacy Organizations Including NASP Commend 2026 Spending Bill with Important PBM Provisions, Urge Passage · Washington, DC, Jan. 21, 2026 (GLOBE NEWSWIRE) -- The National Community Pharmacists Association, the National Association of Chain Drug Stores, FMI – The Food Industry Association, the National...

Original title: “NASP Applauds Congressional Passage of Landmark Bill to Protect Patient Access to Specialty Pharmacies and Support Specialty Pharmacy Businesses”