Rare Disease

Seeking orphan drug approval for rare diseases is challenging. With over 7,000 identified rare diseases and approximately 300 million patients—the need for rare disease treatments and the consequences on healthcare spending has increased globally. 

Crucial differences exist between traditional and orphan commercial markets, such as:

  • Limited data from small patient groups
  • Insufficient knowledge base for the rare condition 
  • Higher prices due to specialized technology and longer research and development 
  • The lack of dedicated approaches to evaluating rare disease drugs

These differences translate to challenges in developing economic models and HTA submissions that meet conventional standards for health technology submissions. Additionally, as new programs are implemented to formalize the assessment of rare disease therapies, it is important to align with a team that has a deep understanding and years of experience with market access for rare diseases.

Effective regional strategy for rare diseases requires a high level of adaptation. Developing a comprehensive market access strategy will ensure the long-term launch plan is positioned for success. Country-specific strategies are needed for pricing (including strategic rebates and discounts), reimbursement thresholds, health technology assessments (HTA), public vs private payer dynamics, and the acceptance of real-world evidence (RWE)-based modeling. 

IMAC has considerable expertise in market access strategy for rare diseases. We understand the nuances in approach from pre- and post-launch evidence generation, to developing a rigorous and innovative economic model structure for rare disease and implementing the best strategy for the support and successful reimbursement of these critically important treatments. IMAC has an unparalleled track record of success assisting clients with global launches for rare disease treatments. 

Some examples:

  • One of the first submissions to the NICE (UK) Highly Specialised Technology programme for a genetic therapy in an ultra-rare disease—this was a challenging and highly successful reimbursement win. IMAC not only developed the submission but was heavily involved in the post submission negotiation strategy with NICE. IMAC subsequently supported the client with submissions in the EU and US.

  • Global launch of a breakthrough orphan oncology product with successful reimbursement secured in the UK, European authorities, US (AMCP), and in Canada (CADTH and INESSS).

How can we help you?

Contact us at one of our International offices or submit a business inquiry online.

Yulia specializes in systematic literature reviews, pharmacoeconomic analyses, and outcomes research.

Yulia Vadziuk
Yulia Vadziuk
Senior Consultant, Health Economics

Looking for a first-class market access partner?