CBPartners Runs ISPOR Workshop Evaluating Orphan and Ultra-Orphan HTA Processes Determining New Product Pricing and Market Access

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The workshop evaluated orphan disease new drug pricing and market access policies across Europe, Latin America, and Asia.

CBPartners, a global leader in orphan and ultra-orphan disease drug pricing, market access, and health economics / value demonstration, led a workshop assessing the advantages and disadvantages of different countries’ evaluation processes. The workshop used a case-based approach focusing on rare diseases including Hodgkin’s Lymphoma, Myelofibrosis, Cystic Fibrosis, Chronic Lymphocytic Leukaemia, and Madoteaux-Lamy Syndrome.

The session was led by Ms. Monica Martin de Bustamante, Managing Director at CBPartners, and Dr. Rachel Beckerman, Principal of Value Demonstration at CBPartners. Ms. Meghan Gallagher, Director of Evidence Value Development at Sanofi contributed specific perspectives on the challenges involved with modeling cost-effectiveness of therapies for rare disease, particularly when limited by countries’ existing new drug review policies.

“Orphan disease drugs are critical to patient care, but due to the disease’s dynamics, carry many limitations for evidence and modeling ability,” states Ms. Bustamante. “Although there is some recognition by policy-makers in established markets, many still do not apply a different lens for the review of these unique drugs.”

“When no adjustments in the pricing and reimbursement process are made for orphan disease drugs, the reviews rarely result in positive reimbursement decisions,” adds Dr. Beckerman. “Traditional cost per QALY methods are unlikely to properly demonstrate the value of orphan disease drugs.”

Principals from CBPartners continue to speak around the world about orphan disease drug policy development; the next public session will be at VisionGain’s 11th Pricing and Reimbursement Conference in London on July 17, 2013.

A copy of the ISPOR workshop presentation may be downloaded from CBPartners’ website.

About CBPartners

CBPartners is a leading global strategy consulting firm focusing on healthcare issues for a variety of clients, including biopharmaceutical / pharmaceutical companies, medical device companies and government health authorities. Headquartered in New York City, CBPartners maintains a continuous throughput of local market intelligence updates from advisors throughout over 70 countries. CBPartners provides expertise through five practice areas including Pricing and Market Access, Value Demonstration, Portfolio Optimisation, Commercial Planning, and Government Policy Advisory.

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Cyrus Chowdhury
CB Partners
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