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Orphan Drug Commercial Models: Sustaining the rare disease business

Orphan Drug Commercial Models: Sustaining the rare disease business

Choosing the right commercial model for orphan drug success

The societal and commercial ambitions of orphan drug developers determine which commercial model will best meet their aims. Profit, potential and patient benefit all play a part, so which model is best and what are the considerations which underpin that choice?

This report analyses current orphan drug commercial models, assesses their benefits and challenges, assesses how the market is developing and, with the help of expert input, examines how they benefit “pure play” orphan drug developers and big pharma.

Answering key questions:

  • Meeting expectations: What factors are vital for orphan drugs commercial teams to ensure that their business model works for all stakeholders?
  • Key components: What are the key components of current commercial models and how are these evolving?
  • Hybrid best? What can be learnt from the hybrid (or partnership) commercial strategy as exemplified by Sanofi and Genzyme? Is this the way of the future?
  • In house: Roche and Novartis are pursuing an integrated strategy - what is the challenge for big pharma in taking this path?
  • Small developer challenges: What research and market access challenges exist for “pure play” orphan drugs companies and how are they overcoming them?
  • Assessing success? Profit or patient benefit - what factors are critical for assessing orphan drug success?
Key Topics Explored
  • Big pharma could learn lessons from smaller orphan drug players in their consultative and inclusive attitude to patients
  • Size doesn’t matter. The dynamics of the orphan drugs market mean that company size is not a critical factor for success. The market is characterised by a high number of smaller orphan drug companies who are focussing on a range of rare diseases with high unmet needs.
  • Competition in the rare disease space is now a fact and the market dynamic is changing dramatically. What are the potential negative impacts on the wider orphan drug sector?
  • The fully integrated commercial model allows access to the resources and reach of big pharma but competing priorities can dilute the attention given to rare diseases.
Expert Contributors
  • Tony Hall, Co-founder of FindaCure
  • Luigi Longinotti, Portfolio Manager – Orphan Drugs at Recordati
  • Pedro Lendinez Ortega, Products Affairs Manager, Orphan Drugs Especialist en Orphan Europe
  • Wendy White, SVP Rare Disease Dohmen Life Science Services
  • Chairman and CEO, Rare Disease Company
  • Chairman and CEO, Rare Disease Company
  • VP, Rare Diseases, Biopharmaceutical Company.
  • CEO and Rare Disease Expert, Bioscience and Biopharmaceutical Company.
  • Rare Disease Expert, Healthcare regulatory policy and government relations consulting firm
About FirstWord

FirstWord is an innovative industry intelligence leader serving over 240,000 Pharma and MedTech professionals worldwide. FirstWord offers a range of products and services designed to help your company gain a competitive edge by making key business decisions with speed and confidence.
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Executive summary
Research objectives and methodology
Contributors
Current structures
Key findings
Orphan drugs market overview
What are orphan drugs?
The orphan drugs market is increasing in value
There are a large number of orphan drugs being approved each year
Some orphan drugs can generate significant revenue
New products will continue to drive growth
Companies involved in orphan drugs
Orphan drugs commercial models and leading companies
Key findings
There are different structures for orphan drugs companies
A fully integrated model is favoured by larger pharmaceutical companies
Partnership or partially integrated models are being increasingly used
Independent and focused on orphan drugs
Shire: increasing its focus on rare diseases
BioMarin: a blueprint for orphan drug success
Strategies for success
Key findings
Past orphan drugs success has been largely down to the unmet need
Product Acquisition removes the risk but can deliver returns
A focused R&D strategy is essential for success
Education and awareness is an important part of the commercialisation process
Stakeholder engagement is fundamental for orphan drugs
Patients are seen as the most important stakeholder by many
Companies have a role to play in educating and supporting physicians
Determining the value proposition is an important element for commercial success
A successful model is not necessarily the one that delivers the highest revenues
Traditional commercial measures of success are used for orphan drugs
Patient outcomes are increasingly important for orphan drugs
Key challenges for current orphan drugs models
Key findings
Pricing of orphan drugs is expected to become more challenging
The orphan drugs market place is becoming more competitive
There are emerging challenges in the approval processes
The definition of an orphan drug is being scrutinised
Evolution of commercial models
Key findings
A more focused model is perceived as being most effective
Patient engagement will continue to be critical for the orphan drugs model
Innovation will be key for future orphan drug success
Out-sourcing commercial activities can maintain focus and competitiveness
Contributors
Tony Hall, Co-founder of FindaCure
Luigi Longinotti, Portfolio Manager – Orphan Drugs at Recordati
Pedro Lendinez Ortega, Products Affairs Manager, Orphan Drugs Especialist en Orphan Europe
Wendy White, SVP Rare Disease Dohmen Life Science Services
Chairman and CEO, Rare Disease Company
Chris Garabedian, Former CEO of Sarepta
VP, Rare Diseases, Biopharmaceutical Company.
CEO and Rare Disease Expert, Bioscience and Biopharmaceutical Company.
Rare Disease Expert, Healthcare regulatory policy and government relations consulting firm

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