Patient and Stakeholder Engagement

Register Now: Patient Advocacy Groups and Their Role in Orphan Drug Development

There are more than 7,000 known rare diseases, and these conditions afflict an estimated 30 million Americans (almost one in 10). Worldwide, some 350 million are living with a rare disease. But for 95 percent of these conditions, there are no FDA-approved treatments — not one.

Conventional wisdom holds that innovation follows need: that if enough people seek a new or better answer to a problem, enterprising companies will fill the void. While countless thousands of new and refined products bear this out, treatments for rare diseases are a persistently stubborn exception to the rule.

In the entire decade that preceded enactment of the Food and Drug Administration’s Orphan Drug Act in 1983, the pharmaceutical industry introduced just 34 orphan drugs. These compounds are incredibly expensive to develop and most don’t get approved, making pharma companies understandably reticent to commit massive resources for the prospect of little reward.

But things are changing fast. The Orphan Drug Act and similar legislation in Europe, Asia, and Australia are stepping up incentives to address this large unmet need, creating a fertile environment to expand the strength and influence patient advocacy groups. These groups play an ever larger role in the development of drugs to treat cancer and rare diseases, even directly funding clinical trials, recruiting patients, and contributing to study design.

Register for today’s webinar, co-presented by our own Juliet Moritz, Executive Director of Rare Diseases at Premier Research, and Susan Stein, a member of the Global Genes board of directors, as they discuss how advocacy organizations — once largely relegated to fundraising and lobbying roles — are greatly influencing orphan drug development from discovery to approval to market entry. These organizations are growing in sophistication, occupying more seats on the advisory boards and committees of prominent research groups and helping patients themselves become more involved in the design and execution of clinical trials.

*Header image: Our RTP office honors Rare Disease Day 2016 in their jeans and their Global Genes Blue Denim Genes Ribbons.™