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Rare Disease Research and Clinical Trials

An Unwavering Commitment to Patients and Ethical Research

Pfizer's mission is to deliver medicines that can make a real difference for people living with a rare disease. We pledge to maintain the safety of patients who take part in our rare disease clinical trials and to uphold the highest ethical standards in all of our research initiatives. Our Research and Development (R&D) Scientists are committed to delivering new treatments.

On this page, you will find more information about our rare disease pipeline, which investigational therapies are under development, and in which stages. Search here to learn more about clinical trials in your area and for the investigational therapies in which you are most interested.

Rare Disease Pipeline as of May 1, 2024

Rare Disease Pipeline & Trials

Our Focus in Rare Disease   -  

  Learn more about Pfizer Rare Disease