Advocacy in Action
*We are dedicated to assisting advocates on their advocacy journey. Find helpful advocacy links on our Resources page!
Our Watch List: Wisconsin Legislative and Regulatory
Pharmacy Benefit Manager Regulation and Copays
“Cole’s Act”
This bill reduces drug costs by limiting the money Pharmacy Benefit Managers (PBMs) get from the drug supply chain, including pharmacists, patient groups, doctors, clinics, and drug companies. This bill also requires health insurance to apply amounts paid by or on behalf of an individual for brand name prescription drugs to apply to the patient’s out-of-pocket maximum or any cost-sharing requirement. One-Page Summary
Impact to Rare
On average, people with rare diseases spend 3-5x more on healthcare. Copay assistance was created to help vulnerable patient families afford medicines prescribed by their doctors. Some examples include pharmaceutical copay cards, and nonprofit financial assistance.
Pharmacy benefit managers (PBMs) and insurers increasingly create programs to prevent such assistance from counting towards patient cost-sharing. As a result, patients may find it challenging to afford and adhere to their medications.
Newborn Screening
This bill adds federal newborn screening recommendations to the state-required newborn screenings, granting rule-making authority, and providing an exemption from emergency rule procedures. One-Page Summary
Newborn Screening One-Pager
Governor’s NBS Proclamation
Impact to Rare
Newborn screening is crucial for rare diseases as it allows for early detection and intervention, potentially preventing or mitigating long-term problems and improving outcomes for affected children.
Prior Authorization
This bill increases transparency and regulation of prior authorization requirements for health insurance plans. It requires insurers to disclose and streamline prior authorization processes. The goal is to make it easier for patients and providers to understand and navigate insurance approvals for medical services.
Impact to Rare
Rare disease patients often face complex and frequent prior authorization requirements for specialized treatments, therapies, and medications. This bill could mean faster approvals for critical, often high-cost therapies, fewer interruptions in treatment, and clearer communication between providers and insurers, helping to improve overall health outcomes.
Prior Authorization (Therapies)
Bill regulates health insurance plans in WI by establishing specific rules for prior authorization of services such as physical therapy, occupational therapy, speech therapy, chiropractic services, and other similar therapies.
Impact to Rare
This may translate into more timely access to critical therapies, fewer disruptions in care, and clearer communication between providers and insurers, helping to support better health outcomes and continuity of care.
Rare Disease Day
This day will be recognized February 28, 2025, as Rare Disease Day in Wisconsin.
Impact to Rare
Rare Disease Day is important for raising awareness, advocating for research, and building community for the 300 million people worldwide living with rare diseases, their families, and caregivers, and for pushing for better access to diagnosis, treatment, and care.
Pain Parity
This bill requires insurance plans in WI to cover non‑opioid drug alternatives for the treatment and management of pain. Specifically, it mandates coverage of clinically appropriate, FDA‑approved non‑opioid treatments and prohibit certain utilization controls that limit access to them.
Impact to Rare
This could improve quality of life for rare disease patients by providing timely access to appropriate therapies, reducing reliance on opioids, and supporting more effective, tailored care strategies.
Redeterminations of Eligibility for Medicaid
This bill prohibits DHS from automatically renewing the eligibility of Medicaid members.
This bill requires people to provide eligibility twice a year (currently once). It removes coverage for 6 months if reporting is not completed on time and requires the state to review all Medicaid cases (over 1.2 million people) by January 1, 2026.
Impact to Rare Disease
Many rare disease families rely on Medicaid, Family Care, IRIS, CLTS, BadgerCare, MAPP, or ForwardHealth. This bill adds a new burden to rare disease families who are already managing medical needs, ongoing paperwork, and complicated systems. Additional steps, forms, and deadlines may put health coverage at risk.
Rare Disease Advisory Council (RDAC)
While there is not a current bill, establishing an RDAC is regularly discussed. More than 25 states have already enacted RDACs in their states.
For more on RDACs, please see video provided by our friends in Michigan.
Impact to Rare Disease
Having a state RDAC allows for a dedicated group of subject experts to be at the ready to provide important rare disease knowledge to state government bodies as needed.
*Provided with permission by The Bonnell Foundation in Michigan
Our Watch List: Federal Legislative and Regulatory
The Access to Genetic Counselor Services Act
This bill provides for coverage under Medicare of genetic counseling services that are furnished by genetic counselors. Covered services include those services, as well as incidental services and supplies, that would otherwise be covered under Medicare if provided by a physician.
Impact the Rare Disease
Most rare diseases are genetic diseases and access to genetic counselors is vital in helping a family understand their disease.
Accelerating Kids’ Access to Care Act (AKACA)
This bill provides for Medicaid physicians to enroll in out-of-state Medicaid programs for a 5 year period in order to accelerate care to children needing services from other states.
Impact the Rare Disease
Many children with rare diseases do not have specialists available in their state. In order to access expert care, they need to travel out of state. Children on Medicaid are limited to accessing care in only their home state. Exceptions can be made, but the process for obtaining care is a complicated, long process. When a child is in a crisis due to their rare disease, they don’t always have the time to wait for approval before receiving treatment. This bill would allow physicians to opt in to multi-state Medicaid programs thus eliminating the wait period a child would have to wait in order to access out-of-state care.
Give Kids a Chance Act
The Rare Pediatric Disease Priority Review Voucher (PRV) Program is a U.S. FDA initiative designed to encourage development of treatments for rare childhood diseases. Under the program, a company that gains approval for a qualifying rare pediatric drug receives a transferable voucher for a priority review of another drug—significantly speeding up FDA review time.
The Rare Pediatric Disease Priority Review Voucher (PRV) program’s previous authorization lapsed on December 20, 2024. The current reauthorization bill seeks to extend the program’s deadlines through the late 2020s, ensuring continued incentives for rare disease innovation.
Impact to Rare Disease
This extension encourages pharmaceutical companies to invest in developing treatments for rare childhood conditions that might otherwise lack commercial incentives. As a result, the Act promotes innovation, supports the development of new therapies, and helps improve access to critical treatments for children with rare diseases.
Health Equity and Rare Disease Act of 2025 (HEARD Act)
This would direct the National Institutes of Health (NIH) and other federal agencies to boost research, data collection, outreach, and support for rare diseases, especially those that disproportionately affect minorities and underserved populations.
Impact to Rare Disease
It aims to address long-standing inequities in health care and research for rare diseases – many of which are understudied, underdiagnosed, and often hit minority or marginalized communities harder.
Safe Step Act
This bill will require group health plans to establish an exception process to medication step therapy protocol.
Impact to Rare Disease
Many times the drug that a doctor prescribes to the patient is denied by insurance until similar, cheaper drugs already on the market have been tried. By forcing a patient to use a drug choice not recommended by the physician, the patient may experience major side effects that cause adverse health effects and could lead to costly hospitalizations.
This bill would make it so that if the treatment on the market was expected to be ineffective or likely cause an adverse reaction, or was expected to decrease the patient’s ability to handle daily functions, that the Step protocol would be suspended and the physician prescribed drug would be covered by insurance within 72 hours after receipt of the request, or 24 hours when the protocol jeopardizes the life or health of the individual.
ORPHAN Cures Act
This bill aims to change how “orphan drugs” (drugs developed for rare diseases) are treated under the federal government’s drug-price negotiation rules.
Impact to Rare Disease
Many rare-disease drugs are first approved to treat one condition, then — after further research — are later found useful for other rare conditions. Under current policy, that second (and third, etc.) indication can trigger loss of pricing-exemption, discouraging further research.
By preserving the incentive structure even when additional indications are found, the ORPHAN Cures Act seeks to encourage pharmaceutical companies to conduct “follow-on” research. This could lead to more treatments for more rare diseases — an important issue for the roughly 30 million Americans living with rare diseases.
Ask Your Members to Join the Rare Disease Congressional Caucus
The Rare Disease Congressional Caucus is a bipartisan bicameral caucus that works to raise awareness of rare diseases. It is open to ALL legislators. If your legislator is not a member, please reach out and ask them to join!
Impact to Rare Disease
This Caucus helps bring public and Congressional awareness to the unique needs of the rare disease community (including patients, physicians, scientists, and industry), and creates opportunities to address barriers to the development of and access to life-altering treatments. It gives a permanent voice to the rare disease community.
