Rare Diseases and Orphan Drugs

Bring new drugs to market with the confidence of our insights

A decline in blockbuster drugs has seen pharmaceutical companies keen to investigate new areas of R&D and the introduction of the Orphan Drug Act has incentivised manufacturers to research and develop new drugs for rare diseases, bringing many new treatments to market.  

Given that there are over 7,000 classified rare diseases and 70% of them have no form of treatment, there are considerable unmet needs in this area. Regulatory benefits such as longer market exclusivity, breakthrough designations, reduced fees and tax incentives are all encouraging investment. However, the marketing process and lifecycle for a rare disease drug are very different to a mass market product and require particular skills and knowledge.

We understand the challenges and constraints in undertaking effective market research in this area and have considerable experience in conducting research programmes for both existing and new drugs for treating a wide range of diseases including haemophilia, cystic fibrosis, lupus, motor neurone disease, acromegaly, Fragile X syndrome, Gaucher’s disease and Gorlin’s syndrome, as well as many others.  We’re using new techniques afforded by technology to reach out to hard-to-find patients and new methodologies

We can help you with research programmes designed to guide:

  • Market landscaping and opportunity assessment

  • Product development and portfolio management

  • Stakeholder engagement

  • Physician and patient journey understanding

  • Advocacy group and funding agency liaison

Rare Diseases and Orphan Drugs resources:

Rare disease- Rapport


Discover the key steps required to implement an effective market research programme.


How we evaluated the potential for an existing AI product to treat a rare disease

Case study

How we evaluated the potential for an existing AI product to treat a rare disease.

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rare disease infographic-small-


Find out more about our experience and expertise in rare diseases and orphan drugs.