International Precision Medicine Conference

April 19-21, 2021 | Orlando, USA

Scopus Indexed Conference
Holiday Inn Orlando SW Celebration Area, 5711 W. Irlo Bronson Memorial Highway, Kissimmee, FL 34746, USA
Phone : 1 (702) 988 2320
Toll Free: 1800–883-8082
Whatsapp: +1 (540) 709-1879
April 19-21, 2021 | Orlando, USA

Safiyya Gassman

International Precision Medicine Conference- Safiyya Gassman
Safiyya Gassman
Pfizer, New York, USA
Title : The evolution of rare disease policy& national plans


Rare diseases collectively exert a global public health burden in the severity of their manifestations and the total number of people they afflict. For many patients, considerable barriers exist in terms of access to appropriate care, delayed diagnosis and limited or non-existing treatment options. Motivated by these challenges, the rare disease.patient community has played a critical role, elevating the patient voice and mobilizing legislation to support the development of programs that address the needs of patients with rare diseases.  Despite these legislative effortsand the growing contributions of patient advocacy groups in moving forward implementation and adoption of rare disease programs, gaps still exist across the policy landscape for several countries.  This presentation will provide an overview of the ongoing policy developments for rare disease patients, provide perspectives on current status and/or challenges with implementation and recommendations for future developments.

Audience Take Away:

  • With small patient populations – not much is known about how health systems are looking to address the needs of rare disease patients.  Thus, this presentation will expose the audience to the health policy developments that are currently being debated to support rare disease treatments
  • This will offer innovative thinking as to potential “solutions” as to how to address rare diseases
  • As we think about the evolution of more targeted/precision therapies – most of these new treatments will be for a small set of patients – thus the developments that many health systems are looking to implement for rare disease patients may be the new way of healthcare solutions for all sub-sets of populations that will benefit from targeted treatments


Safiyya is currently a Director of Public Affairs at Pfizer, focused on advancing patient access to treatments in rare disease. In this role, her responsibilities span health policy issues, working closely with the global stakeholder community. Prior to this role, Safiyya was a part of PhRMA International Policy, where she led the research-based biopharmaceutical industry efforts around healthcare reform and access issues. Her work involved engagement with the WHO, OECD and national regulatory organizations.   Safiyya holds an MSPH in Health Policy from the Johns Hopkins School of Public Health and a Bachelor of Arts & Science focusing from McGill University.