Genomics-Enabled Drug Repurposing and Repositioning: A Workshop

When: June 24, 2013 (8:30 AM Eastern)
Where: Keck Center (Keck 100) • 500 Fifth St. NW, Washington, DC 20001

Topics Biomedical and Health Research, Public Health
Activity: Roundtable on Genomics and Precision Health
Board: Board on Health Sciences Policy

The path of drug development can be expensive and time-consuming. Recent estimates predict, on average, an expenditure of at least one billion USD and a time frame of ten years to bring a drug to the market. While spending on drug research has increased, this has not led to an increase in the number of drugs approved by the Food and Drug Administration. It is therefore no surprise that pharmaceutical companies are examining options to either give drugs not commercially available a new chance at approval (drug repositioning) or to search for new indications for existing drugs (drug repurposing).

Drug repositioning or repurposing holds many appeals. Typically these drugs have been extensively profiled and studied in terms of safety, efficacy and toxicity, thereby decreasing the amount of preclinical research required. This streamlined process saves time and money, with repurposed drugs generally being approved in much shorter timelines (3-12 years) and at about 60% of the cost of typical development. While approximately 10% of new drug applications gain market approval, repurposed drugs approach approval rates near 30%, presenting a significant market-driven incentive for companies that also aligns with patient desires for new therapeutics.

Historically drug repurposing was an unintentional, serendipitous process where off-target effects were noted to occur, occasionally in such a way that they could be used for a new indication. Perhaps the most recognizable example of a successful reposition effort is sildenafil. Originally developed as an anti-hypertensive, sildenafil has been repositioned for treatment of erectile dysfunction with revenues peaking at $1.88 billion USD in 2003. Another drug, thalidomide, was essentially removed from the market after its connection to serious fetal limb defects was discovered. However, recent research has shown it to be an effective treatment in leprosy and multiple myeloma. These success stories, and an increasing number of other examples, have prompted pharmaceutical companies to consider repurposing or repositioning drugs in their own arsenals.

Technological advances and the increasing availability of genomic data and computational systems have resulted in new methods to systematically identify both drug targets and pathways for detecting drugs with a secondary, often times seemingly unrelated, indication. The Roundtable on Translating Genomic-Based Research for Health will host a workshop to assess the current landscape of drug repurposing activities in industry, academia, and government, examine enabling tools and technology, and evaluate the business models and economic incentives for drug repurposing programs. Stakeholders, including government officials, pharmaceutical company representatives, academic researchers, regulators, funders, and patients will be invited to present their perspectives and participate in discussions during the workshop.

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