May 3, 2017

Real Endpoints' White Paper

Preferences in MS Therapy: White Paper and Literature Review

By Beth Nash, MD, Stacy Mowry, MA, R. Brett McQueen, PhD, Roger Longman, Chief Medical Officer, REAL ENDPOINTS LLC

When it comes to the practical decisions of using and paying for specialty drugs, the dominant voices belong to physicians and payers, whose preferences around specific drugs are reflected in their prescribing and formulary choices. Pharmaceutical companies certainly make themselves heard about the value of their own products. And patient advocacy organizations may participate in discussions about opening access to all drugs, but less often to influence a decision about a specific one. In most prescribing and coverage decisions, the patient’s voice is the quietest. And for good reason: faced with a complex, serious disease, most patients take their doctors’ advice, which is itself based on that physician’s experience with other patients and his or her reading of the clinical literature – and often filtered by the coverage rules of the patient’s insurer. But for a variety of reasons, getting the patient involved in the decision is increasingly important. In the first place, the patient is paying an increasingly large part of the bill – and should therefore have a seat at the decision-makers table. In the second, patients have different points of view on their diseases and how they should be treated – different from each other and different from their physicians. And regulators increasingly want to hear from them. The 21st Century Cures bill specifically adds patient-reported outcomes to a regulatory approval agenda which is otherwise dominated by clinical endpoints important to physicians and researchers. The challenge: what do patients want – and does what patients want differ meaningfully from the preferences of payers and physicians? To begin to answer these questions, Real Endpoints (RE) undertook both a broad literature review and a survey of patient, physician and payer preferences. To keep the project manageable, we limited its scope to multiple sclerosis therapy. The project was funded by the Pharmaceutical Research Manufacturers Association.

Read full insight paper > Key Findings, Preferences in MS Therapy >