Accelerate patient access to quality medicines, treatments,
and services for rare diseases by harnessing the collective expertise of partners with shared values
Every patient living with a rare disorder has equitable access to quality healthcare.
Harpreet Ram, President, EVR Consulting
Harpreet is the founder and the president of EVR Consulting, a small consulting firm providing services to biopharmaceutical companies. She has nearly 20 years
of experience helping both large and small companies optimize Medical Affairs operations. In addition to her expertise in Medical Affairs Operations, she has spent the last 10 years focusing specifically on “access to medicines” for rare disease patients. Harpreet has been instrumental in the design, development, and implementation of several Global Managed Access programs, providing lifesaving therapies to rare disease patients in five continents.
Dr. Patti Tracey
Dr. Patti Tracey completed her PhD in Nursing, University of Toronto in 2015, with a Collaborative Program PhD in Global Health and an International Nursing PhD Collaborative from the University of the Balearic Islands, Majorca, Spain.
She currently, holds a full-time faculty position with the Trent Fleming School of Nursing, Trent University, Ontario Canada. Dr. Tracey's area of research focuses on the role and impact of North American NGO (non-governmental organizations) led Short- Term Medical Missions in LMICs (low and middle income countries) with a focus on improving quality and safe medication practices. She completed a 4-year PQMD/Americares sponsored initiative in Malawi, Honduras and the Philippines to promote the development and implementation of best practice guidelines by host countries. During the final year of the project the feasibility of guidelines were evaluated (study results below). Biannually, she provides clinical instruction to undergraduate nursing students who wish to complete an International Community Health placement in Central America. Dr. Tracey participated in the first Canadian Knowledge Exchange Delegation to Guatemala as part of the Horizon's of Friendship (a Canadian NGO)'s 4-year Maternal, Newborn and Child Health Project in Totonicapán, Guatemala. She continues to volunteer as an active executive board at Horizon’s of Friendship https://www.horizons.ca/.
Dr. Tracey led the GARDaccess Guidelines workstream and delivered the development of Guidelines for Rare Diseases.
Access to life-saving medicine programs in low- and middle-income countries (LMIC) presents a significant unmet need for patients living with rare disorders. Rare genetic disorders collectively impact a significant portion of the world’s population. Although individually rare, collectively the 7,000+ rare disorders impact 400 million individuals worldwide. Approximately 80% of these rare disorders are genetic in origin and half of these disorders affect children.
There were virtually no treatments available for rare disorders until the 1980s and consequently, these often life-threatening conditions took parents from children and children from parents at a very early age. The Orphan Drug Act of 1983 was designed to provide financial incentives to U.S. based companies and encourage the development of therapies to treat rare disorders. Many rare disease therapies have emerged over the decades; these have been priced to justify the cost of research, manufacturing, distribution and serving small patient populations. Rare diseases and orphan drugs are now at the forefront of novel development and groundbreaking research.
The vision of the Global Alliance for Rare Disorders Access (GARDaccess)can bring hope and promise to many by providing seamless access to those individuals residing in nations where access to these life-saving therapies is limited. The proposed goal of GARDaccess is a patient-centric vision whereby everyone contributes in a meaningful way, shares a united voice, and cross-collaborates. GARDaccess will overcome the challenges and obstacles inherent in single-focused access programs to create a strategic, integrated operating model for global access to rare disease therapies, evolve guidelines to ensure safe and quality products and services for patients with rare disorders globally and raise rare disease awareness and education in under-resourced countries.
GARDaccess’ Advisory Committee is responsible for the development and implementation of the organization's strategic direction, and is composed of an Advisory Board and ad hoc members. Sitting and ad hoc membership of the board will be composed of representatives from alliance partner organizations representing the pharmaceutical/ biotechnology industry, patient advocacy, private sector, global foundations, as well as a medical ethicist. Sitting Advisory Board members are responsible for advising on GARDaccess strategy, alliance memberships, and guidelines on program implementation. The Advisory Board will work in tandem with the Secretariat to help guide strategic vision.