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It Starts with One (Rare) Patient

Theresa Brady
Theresa Brady
April 15, 2019

In 1983, patient advocates – now known as the National Organization for Rare Disease, NORD – ignited a movement and rallied support  for the Orphan Drug Act to facilitate development of treatments for conditions affecting fewer than 200,00 Americans.  It started with this one idea and served as the catalyst to ignite a new generation of enhanced support, advocacy and treatments for rare disease patients.

Today, there are hundreds of patient advocacy groups, more than 770 new treatments approved since the Orphan Drug Act, and over 560 treatments in development for rare diseases. The rising popularity of precision medicine and the availability of gene therapy are enabling researchers and clinicians to address rare disease in ways unlike ever before. Yet, with only 5% of the 7,000 classified rare diseases having an available treatment, how can we continue to up our game to win the war on rare disease?

This June in Philadelphia at the 2019 BIO International Convention, we will again put the patient at the center of all we do. In rare disease, this means tackling contemporary and oftentimes complex challenges and sharing strategies to ensure patients get safe and effective treatments as quickly as possible. Panels at Convention will explore such top priorities as innovative trial design, access, and clinical development. We are proud that patient advocates will join these discussions, as they continue to enhance progress.

In clinical trial design, even as the FDA and other government agencies have incentivized drug development for rare diseases, significant hurdles remain. At BIO 2019, leaders will discuss innovative strategies for producing trials in rare disease that yield rich and compelling data needed for drug approval. These include designing trials for small populations, the use of combined registries to identify enough patients for research, and identifying new biomarkers to serve as clinical endpoints for when there is no regulatory precedent or established endpoints.

Given the relatively high cost of rare disease treatments, access is at the top of everyone’s agenda. Experts will examine innovative payment models that are breaking new ground, and share perspectives and solutions for what data should be captured within clinical trials to satisfy both the payer and patient.

We are seeing more and more ways where the biotechnology industry is thinking creatively and acting flexibly to combat the unique challenges associated with treating rare diseases. BIO 2019 will serve as the platform to engage, learn and progress these latest initiatives and thinking; initiatives of today that we believe will have a significant impact tomorrow on the rare disease community.