Project Scope

To evaluate the diagnostic, treatment, public funding landscape, pricing and cross border referrals for Duchenne Muscular Dystrophy (DMD)

Challenge

Our client is a leading provider of pharmaceutical products for ultra-rare disorders. Their objective was to understand the treatment landscape, competitive pharmacotherapies in use, public and third-party funding opportunities, and pricing rules for ultra-rare neuromuscular disorders

Solution

Conducted extensive primary and secondary research to get detailed insights on:

• Diagnosis and treatment landscape for ultra-rare neuromuscular diseases
• Strengths and weaknesses of therapies available for DMD management
• Availability and sources of public funding for ultra-orphan medications
• Official price-setting process, impact of IRP pricing and value assessment process for ultra-orphan drugs
• Procedure of cross border treatment and referrals

Results

• Successfully facilitated client understanding of the diagnostic and treatment landscape for ultra-rare neuromuscular disorders
• Identified the funding potential for DMD drugs through public and third party routes
• Enabled the client to gain insights on pricing process and formulary inclusion process of DMD treatments
• Enabled the client to understand treatment referral patterns for ultra-rare diseases