The new California Rare Disease Access Coalition aims to
improve access to diagnoses and treatments for orphan diseases.
This is an exciting time for the rare disease community in California. Scientific discovery is advancing at an extremely rapid pace and more life sciences companies are working to develop therapies for previously untreatable diseases.
The bleeding disorders community has long been on the forefront of these advancements, as demonstrated by the recent FDA approval of two gene therapy treatments for hemophilia. The advancement of new gene and cell therapies is exciting news across rare disease, but this accelerating innovation will likely require new payment models.
At the same time, health care costs generally are on the rise. Payers and patients are both looking for ways to reduce and minimize costs. Unfortunately, many of the measures used to reduce costs can have the impact of impeding access to treatment. There is also an increasing awareness of health inequity and the need to ensure equitable access to life saving and sustaining treatments for all. Rare diseases are particularly vulnerable to inequities – treatments are costly, provider expertise and workforce is limited, and knowledge of standards of care is lacking.
California is in the unique position to lead on issues impacting rare diseases given that it is the birthplace of the biotechnology industry and home to significant life sciences research and innovation.
Over 460,000 Californians are directly employed by the biotechnologies industry, by over 16,000 companies, generating over $413 billion dollars in economic output. California researchers alone have developed 366 cancer drugs, 151 treatments for infectious disease, 77 immune system therapies, 57 treatments for blood disorders and 44 gastrointestinal treatments (to name just some of the innovation).
Communication and collaboration between patients, advocates, providers and other stakeholders can easily become siloed and sometimes disjointed, potentially impeding access and increasing costs while negatively impacting patient outcomes.
The purpose of the coalition is to bring together patient advocates, rare disease providers, innovators and other stakeholders in order to enhance communication and positively impact outcomes, costs and patient experience in the diagnosis and treatment of rare disease through innovative policy collaborations.
Who Should Join?
• Rare Disease Patient Advocacy Groups
• Rare Disease Patients and Caregivers
• Providers caring for Rare Disease Patients (i.e., Hemophilia Treatment Centers, Sickle Cell Centers, etc.)
• Other health care providers (i.e. Kaiser, physician groups, etc.)
• Life Science and Biotechnology Companies
• Specialty Pharmacies
• Policy Makers
Benefits Of Participation
- Network and build relationships with other patients and patient groups who have similar experiences, needs and priorities
- Network with providers and innovators working in your therapeutic area
- Collaborate on policy issues
- Coordinate advocacy efforts for greater effectiveness
- Learn how to overcome access barriers
- Share and learn from best practices in advocacy, health care access and public policy
2026 Agenda
In 2026, the collaborative will host three half-day meetings. They will be in-person in Sacramento with a hybrid option for virtual participation.
The format of the meetings will allow for robust participation and discussion. This is a multi-way conversation. We want to know what issues are facing all sides and work together to find solutions. Agendas for the meetings will be created by the collaborative steering committee with input from all members. Monthly updates and meeting minutes will also be provided to Members.
Potential Meeting Topics
- California All Copays Count Coalition – protection of patient assistance programs
- Rare Disease Advisory Council
- Access to Diagnostics and Genomic Testing
- Value Based Contracts/Agreements
- Prior Authorization Reform
- Alternative Funding Model
Calendar
Select an event to register
Steering Committee
- Bleeding Disorders Council of California
- Cystic Fibrosis Research Institute
- Axis Advocacy – Sickle Cell Disease Education & Support
- California Biotechnologies Foundation
- California Life Sciences Association
Join the Coalition
TO BECOME A MEMBER, PLEASE COMPLETE THE APPLICATION BELOW.
Patient advocacy groups may join at no cost. Ticket fees will apply for industry stakeholders.
Thank You To Our 2026 Coalition Sponsors
