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Examining the Expanding Role of Patient Advocacy Groups


Patient Advocacy groups are playing a larger role in development of drugs to treat cancer and rare diseases.

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Tweet: There are more than 7,000 known rare diseases, afflicting an estimated 30 million Americans http://ctt.ec/x3U52+There are more than 7,000 known rare diseases, afflicting an estimated 30 million Americans

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September 9, 2016 | by Megan Sims

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.

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.

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 development of drugs to treat cancer and rare diseases, even directly funding clinical trials, recruiting patients, and contributing to study design.

Join Premier Research and rare disease advocacy pioneer Global Genes for a live webinar examining this important trend. Juliet Moritz, Executive Director of Rare Diseases at Premier Research, and Susan Stein, a member of the Global Genes board of directors, will present The Powerful and Evolving Role of Patient Advocacy Groups in Orphan Drug Development on Wednesday, September 14, at 11 a.m. Eastern time.

Register to attend this presentation, which will examine 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.

The presenters will trace the expanding role of advocacy to 1988, when the FDA began working with HIV/AIDS patient advocates. They’ll discuss how the relationship between drug companies and patients is becoming a genuine partnership that will strengthen as regulatory and legislative changes — including the pending 21st Century Cures Act — further promote cooperation between patients and the organizations that champion their cause.

Keywords: Patient Advocacy, Premier Research, Rare Diseases  



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