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PPMD Continues to Lead Work on Critical Duchenne Access Issues

With the promise of drug approvals on the horizon, access to these therapies will be critical to our Duchenne community. PPMD has been leading work related to Duchenne and rare disease community access issues on multiple fronts. 

The Institute for Clinical and Economic Review (ICER) is an independent organization that conducts publicly available analyses of the ‘value' of the new drugs at the time of their introduction. Insurers, provider groups, patients, and policy makers often utilize this information to anchor discussions regarding appropriate use, pricing, and payment for new drugs.

 

The basic approach of ICER’s Value Assessment Framework (or the algorithm used to calculate value) is to serve as a way to categorize and integrate various conceptual elements into judgments of two different aspects of value, called “care value” and “provisional health system value.” ICER led an initiative to develop a conceptual framework intended for insurers to apply to guide their assessment of the value of medical services, including drugs, medical devices, and procedures.

The specific aim of the project was to develop a conceptual framework that identifies the relevant domains of value and describes options for measuring these domains and for integrating them into an overall assessment of value. ICER stated that the overall goal of the project is "to improve the reliability and transparency of value determinations made by insurers in the US, an outcome that will produce greater consistency across insurers, provide greater certainty for manufacturers, and enhance the legitimacy of medical policy decisions with patients and the public”. 

To inform a planned 2017 update to the methods that underpin its evidence reports on new drugs and other health care interventions, ICER recently conducted a national call for suggestions on how to improve its value assessment framework. PPMD’s ICER Assessment Framework comment was submitted on Monday September 12th and can be downloaded here. 

PPMD urges ICER to allow for expansion of their Value Assessment Framework such that the algorithm is inclusive of the expertise, experience, and evidence around patient-preference – and the trade-offs and considerations patients make when seeking treating options.




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