Strategic Patient Advocacy vs. Evidence Package: The “Rare” Challenge for Orphan Drug Manufacturers

On September 19, 2016, the USA FDA granted accelerated approval to $SRPT (SAREPTA THERAPEUTICS) lead product, EXONDYS 51™(eteplirsen), the first targeted treatment for (DMD), a devastating rare disease. What makes the case unusual is that in approving EXONDYS 51™, the FDA went against the recommendation of its Advisory Committee, a…

WHITE PAPER: The CEESP Economic Evaluation: Can Clinical Efficacy and Cost-effectiveness Co-exist in France?

The emergence of economic appraisals conducted by the Commission Évaluation Économique et de Santé Publique (CEESP) represents an important development in attaining market access to France. As of October 2013, manufacturers of certain innovative products are required to submit to an economic evaluation from the (CEESP) in addition to the…

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