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Rare Diseases and Orphan Drugs

Delivering global market research insights for rare and orphan diseases

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: The market opportunity and market research considerations

Drawing upon our considerable experience of managing both small and large scale studies across a number of different diseases, we have created a guide outlining the key steps required to implement an effective market research programme. The guide covers: 

- Sampling and recruitment
- Ethical considerations and constraints
- Suitable methodologies
- Impactful analysis and outputs 

This guide is free to marketing and market research professionals of pharmaceutical manufacturers. 

Click here to request your complimentary copy »