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 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 the Global Alliance for Rare Disorders Access (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.