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Pfizer's Rare Disease Commitment: Transforming Patients' Lives

At Pfizer, we are dedicated to delivering life-changing therapies that improve the lives of individuals affected by rare diseases and envision a day where many more diseases have treatment options. To that end, we have more than 20 approved medications as well as additional drugs currently in clinical trials.

Pfizer is uniquely positioned to lead advances in rare disease treatment. We bring deep scientific expertise to our mission, and years of exploring emerging scientific frontiers with some of the world's leading research organizations as our partners. We work closely with patients, advocacy organizations, research centers, academic institutions, and other experts to share innovative practices from diverse perspectives. This approach has provided advancements and ensures that we continue to add meaningful value to patients and society.

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Pfizer Rare Disease Resources

Clinical Trials and Pipeline
Pfizer is working tirelessly to expand its Rare Disease pipeline, focused on treatments for hematological, neuromuscular, pulmonary, and inherited metabolic diseases. Clinical trials are underway, some in recruiting stages.
Areas of Focus
With unprecedented experience in research and treatment of rare disease, Pfizer is strategically focused on key areas and new frontiers in which we can apply our deep scientific expertise and resources. See where we're concentrating our strengths to catalyze change.
At Pfizer Rare Disease, we believe there's only one way to succeed - together. Collaboration is the catalyst for progress. Pfizer Rare Disease works with a diverse range of stakeholders, including patients and their advocacy organizations.
Patient Assistance Program Pfizer RxPathways®
Pfizer RXPathways is Pfizer's patient assistance program that helps eligible patients get access to their Pfizer medicines by offering a range of support services.
Compassionate Access
Because we realize that some patients are without options and are running out of time, Pfizer is making some of its investigational rare disease treatments available to patients in great need. A treating physician must apply.
Grants and Contributions
Pfizer supports work that improves patients' lives and advances breakthroughs in rare disease. Grants are available for medical researchers, patient groups, and other organizations.
For Healthcare Professionals
PfizerPro provides access to Clinical and Prescribing Information on Pfizer Medicines. Support and services for your practice are also available including, Pfizer product samples for eligible US Healthcare Professionals, clinical trial listings, and medical information inquiries.
Rare Disease Research
A deeper understanding of the genetics of rare diseases gives us an opportunity to explore breakthrough science, new approaches, and pioneering collaborations to potentially deliver next-generation therapies for people living with rare diseases.
Pfizer Rare Disease Consortium
Pfizer is pioneering a collaborative approach to develop future rare disease therapies. Its Rare Disease Consortium works with academic centers of excellence in the United Kingdom. Future expansion is planned across Europe and the United States.
Business Partnering Opportunities
Our innovation is fueled by collaborations with centers of research excellence and innovative businesses. Have an idea or platform that could improve rare disease outcomes? Let us know.
Meet Our Rare Disease Leaders
Based on our vast experience, we've created a model to serve the rare disease community, which includes dedicated research, medical, and commercial teams. Our leaders are a driving force behind our commitment to advance rare disease therapies and to serve the needs of patients and families.
Pfizer advocates for rare disease patients year round. There's a lot to celebrate in September! We are joining patients around the world to mark these important diseases awareness observances:
  • Sickle Cell Awareness Month
  • World Duchenne Awareness Day on September 7
  • Worldwide Cystic Fibrosis Day on September 8
  • Growth Awareness Week starting September 18