Emil D. Kakkis, M.D., Ph.D., President and Founder joined us at the World Orphan Drug Congress USA 2016, to give a detailed keynote presentation on Creating Medicines with Real Value for Orphan Diseases.
How?
Investment is needed in R&D for Orphan Drugs, which is clinically a challenging area due to smaller patient populations. This challenge breeds less certainty about a drug's potential marketability. Drug costs also always end up very high, as Rare Diseases require specialized therapies - hence why working with Payers and other stakeholders is so vital. Without financial incentive, offered by legislation, it is save to say that orphan drug development would be hindered, if not halted. Can we produce lower cost drugs for rare diseases? Two possible solutions include:
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Reduce the cost/time of development: e.g. Biomarkers
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Leverage the investment we have made in existing drugs
Download Emil's presentation to find out more about these options and other legislative updates >>