Each February 28th, the world recognizes the needs of patients living with rare diseases on Rare Disease Day. From meetings at our nation’s capitol to podcasts highlighting work being done here in Arizona, an upcoming events at the State Capitol, and at Voice of the Patient during AZ Tech Week that amplifies the voices of patients, our AZBio community is working to make a difference for patients.
Rare Disease Day is a globally coordinated movement dedicated to rare diseases, striving for equity in social opportunities, healthcare, and access to diagnosis and therapies for people living with a rare disease. Since its creation in 2008, Rare Disease Day has played a crucial role in building an international rare disease community—multi-disease, global, and diverse, yet united in purpose. (Source)
The Critical Path Institute Podcast and the evolving rare disease landscape
In this episode of The Critical Path Institute Podcast, CEO Klaus Romero is joined by Vice President of Rare/Orphan and Pediatric Disease Programs Collin Hovinga for an in-depth discussion on the evolving rare disease landscape and the unique role C-Path plays in advancing drug development. With more than 10,000 rare diseases identified, each with distinct characteristics but often shared underlying mechanisms, the conversation highlights both the complexity and opportunity within this space, emphasizing the importance of collaboration and cross-disease learning to accelerate progress.
The episode also explores how C-Path is leveraging data and partnerships to drive innovation, particularly through C-Path’s Rare Disease Cures Accelerator–Data and Analytics Platform, which aggregates and standardizes data from multiple sources to inform smarter, more efficient clinical trials. By enabling tools such as trial simulations and alternative study designs, and by fostering collaboration among regulators, industry, researchers, and patient communities, C-Path is helping to reduce duplication, improve decision-making, and ultimately accelerate the development of therapies for people living with rare diseases.
AZBio at the Nation’s Capitol
U.S. Capitol (AZBio Image)
AZBio President & CEO Joan Koerber-Walker travelled to Washington, D.C. to meet with members of Arizona’s Congressional Delegation. In each meeting she provided information on how public policy why it matters to Arizonans.
REAUTHORIZING THE SBIR PROGRAM IS OUR #1 PRIORITY
Uncertainty & Perceived Investment Risk is making it more challenging than ever for smaller, early-stage companies to raise funds. When the SBIR Reauthorization was delayed, it created a huge gap in the early-stage funding base. This is creating significant challenges for innovators working on all conditions including rare disease therapies that are often developed by small businesses.
Good News This Week: Sens. Joni Ernst, chairwoman of the Senate Small Business and Entrepreneurship Committee, and Ed Markey, its top Democrat, intend to introduce new legislation known as the “Small Business Innovation and Economic Security Act,” which would reauthorize SBIR for the next five years. After the Senate votes, the legislation will return to the House for review and action.
H.R. 6423/S. 864: HELP Copays Act
The HELP Copays Act is a bipartisan federal bill designed to lower prescription drug costs by requiring health insurers and pharmacy benefit managers (PBMs) to count third-party financial assistance—such as manufacturer copay cards or non-profit help—toward a patient’s annual deductible and out-of-pocket maximum. WHY it matters for Arizonans. Arizona passed its law in 2019 to protect patients from co-pay accumulators for Arizonans covered by state regulated health plans, but it does not apply to Medicare or ERISA plans.
H.R. 5509/S. 2903: Safe Step Act
The Safe Step Act is bipartisan federal legislation designed to reform “step therapy” or “fail first” protocols in employer-sponsored health plans. It requires insurers to establish a clear, transparent, and quick exception process—within 72 hours (or 24 hours for urgent cases)—if a prescribed medication is contraindicated, ineffective, or harmful to the patient. WHY it matters for Arizonans. Arizona passed its law in 2021 to address issues created by step therapy policies for Arizonans covered by state regulated health plans, but it does not apply to Medicare or ERISA plans.
Reintroduced: the PASTEUR Act
The bipartisan Pioneering Antimicrobial Subscriptions to End Upsurging Resistance (PASTEUR) Act would encourage innovative drug development targeting the most threatening infections, improve the appropriate use of antibiotics and antifungals (including treatments for Valley Fever), and ensure domestic availability of critical antimicrobials when needed. WHY it matters for Arizonans. Arizona researchers are actively working on better solutions for people living with Valley Fever and how to address a wide range of other pathogens. Due to the nature of these diseases, and their populations, commercialization funding can be extremely challenging without government support.
Reintroduced – (H.R. 1492): the EPIC Act
This Ensuring Pathways to Innovative Cures (EPIC) Act lengthens the amount of time for which drug products must have market approval in order for the products to qualify for negotiation under the Medicare Drug Price Negotiation Program. The Medicare Drug Price Negotiation Program requires the Centers for Medicare & Medicaid Services to negotiate the prices of certain prescription drugs under Medicare beginning in 2026. Among other requirements, drugs must have had market approval for at least 7 years (for drug products) or 11 years (for biologics) to qualify for negotiation. The bill modifies these provisions so as to require drug products to also have had at least 11 years of market approval to qualify for negotiation. The bill’s changes apply retroactively. WHY it matters for Arizonans. The “pill penalty” is having a significant impact on Health innovator’s ability to attract investment. Pills are often less expensive to manufacturer and present an opportunity for lower cost treatments than injectable alternatives.
H.R. 1672: The MINI Act
The bipartisan Maintaining Investments in New Innovation (MINI) Act, legislation ensuring continued access to medical therapies developed with genetically targeted technology (GTT). Drugs made with GTT are often designed to treat life-threatening rare diseases, and the MINI Act will incentivize investment in the next generation of cures for patients in desperate need. By removing an artificial bias against GTT cures, the legislation is creating a brighter future for those suffering from serious medical conditions.
H.R. 4299: Protecting Patient Access to Cancer and Complex Therapies Act
The Protecting Patient Access to Cancer and Complex Therapies Act would revert physician reimbursement for administering drugs under Medicare Part B to Average Sales Price (ASP) plus 6 percent, create an additional rebate paid by manufacturers, and hold patients harmless by basing coinsurance rates off the Maximum Fair Price (MFP). WHY it matters for Arizonans. This is a priority for patient access, especially in communities where sites will close if they cannot cover their costs.
H.R. 5256: 340B Access Act
The 340B Affording Care for Communities and Ensuring a Strong Safety-Net Act (340B ACCESS Act) establishes critical oversight and transparency of the 340B program while providing clear, practical, and achievable solutions to help ensure the 340B program can be a force for good in the nation’s health care safety net. WHY it matters for Arizonans. This is a critical program for our healthcare safety net hospitals. Challenges with implementation of 340B are well known. The result is multiple lawsuits and a patchwork of state laws that are also being passed/challenged or proposed. An update to the 340B program at the federal level is needed to fix this.
H.R. 5269/S. 2761: Reforming and Enhancing Sustainable Updates to Laboratory Testing Services (RESULTS) Act
Preserves patient access to timely, essential clinical laboratory tests by reforming the Medicare rate-setting process for clinical laboratory tests. WHY it matters for Arizonans. Structural flaws of the Protecting Access to Medicare Act (PAMA) have resulted in clinical laboratory payment cuts of more than $4 billion since 2018, negatively impacting laboratories, patients, and providers. The Medicare Clinical Laboratory Fee Schedule (CLFS) accounts for less than 1 percent of total Medicare spending, yet clinical laboratories have an outsized impact as laboratory results inform 70 percent of clinical decisions.
H.R. 5343/S. 1717: Ensuring Patient Access to Critical Breakthrough Products Act WITH diagnostic tests included.
The legislation would provide four years of transitional coverage for breakthrough-designated devices approved or cleared by FDA appropriate for the Medicare population. It creates a roadmap for additional evidence collection by the Centers for Medicare and Medicaid Services (CMS) to make a permanent coverage decision after that period. This would ensure beneficiaries have quicker access to these life-changing technologies, while providing the necessary autonomy for CMS to evaluate the technologies. In 2024, CMS finalized the Transitional Coverage for Emerging Technologies (TCET) policy which attempted to address that gap. WHY it matters for Arizonans. While the TCET program made some improvements to the coverage with evidence development (CED) process, the policy did not go far enough in ensuring seamless coverage after Breakthrough devices achieve FDA authorization and the current version does not include diagnostics.
H.R. 6197/S. 1399: Health Tech Investment Act
Artificial intelligence (AI) and machine learning (ML) play a growing role in healthcare overall, Algorithm-Based Health Services (ABHS), which includes Software as a Service (SaaS), are a distinct subset of AI/ML services and occupy a unique role in the broader discussion of healthcare services. This legislation would
- Define ABHS in the Social Security Act under the various hospital outpatient programs.
- Include criteria specific to ABHS in their new technology payment programs.
- Revise the New Technology Ambulatory Payment Classification (APC) policies.
- Provide ABHS with at least 5 years of consistent payments while assigned to a New Technology APC.
WHY it matters for Arizonans. Current payment pathways provide little incentive to develop and adopt these tools. The FDA has approved or cleared many ABHS, but as of January 1, 2024, fewer than ten ABHS have received hospital outpatient payment assignments through Medicare. Without appropriate reimbursement for providers, patient access to these transformational technologies, especially for patients in rural and underserved communities, will remain limited.
AZBio Events in March and April Amplify Patient Voices
Rare Skeletal Conditions Advocacy Day at the Arizona Capitol (March 16, 2026 – Phoenix)
Rare skeletal conditions have been left out of the rare disease narrative in recent years. The status quo to “wait and see” what happens should not be the norm for the children born each year with rare skeletal conditions. AZBio, the Skeletal Conditions Advocacy Coalition, the the Little Legs Big Heart Foundation, and the Noonan Syndrome Foundation invite you to join a day of advocacy and action on behalf of all Arizonans impacted by rare skeletal conditions. Join us at the Arizona State Capitol to meet your state legislators, share your patient story, and help advance key policy priorities. We’ll highlight the critical need for early diagnosis and intervention, dignity in care, and support through multiple life stages.
Voice of the Patient – AZ TECH WEEK (Apr 9, 2026 – TUCSON)
In our health innovation community, patients are the reason we do what we do. Join us fin Tucson at the University of Arizona for a conversation on life science innovation from the patient perspective.