Orphan Drug Commercial Models: Strategies for Success


Ensuring payer support, delivering stakeholder benefit and generating commercial returns: Pharma's growing orphan drug challenge

Rising competition and closer scrutiny by hard-pressed payers are just two of the trends that are challenging pharma's orphan drug ambitions. Success hinges on ever closer relationships with patients, HCPs and KOLs to create therapy awareness, conduct research and build evidence of value. But what are the patient engagement, market access and pricing strategies that will deliver success in this changing landscape?

In Orphan Drug Commercial Models: Strategies for Success, orphan drug experts from leading pharma companies speak out on the current commercial imperatives for companies working in the orphan drug space and the strategic refinements necessary to meet the challenges of today.

Reasons to Purchase

Orphan drug experts answer tough questions

  • Do you have a framework to establish and demonstrate a link between an orphan drug's price and the value it brings to patients and society?
  • What more could medical affairs and MSLs be doing for your orphan drug program?
  • What are the evidence requirements of HTAs and formulary decision makers, and when should you engage with them?
  • What role do risk-sharing, outcomes-based, and conditional reimbursement agreements play in the orphan drug sector?
  • Are you integrating real-world evidence into your stakeholder education resources?
  • When should you engage with payers to inform them of your evidence generation strategy?
What orphan drug experts say…

Aligning with your market access team is crucial. Building a pharma economic model together with the reimbursement and pricing process is key for small population drugs like orphan drugs. The support of the medical affairs team is also crucial when you want to make a strong value story of what the treatment means for patients. As orphan drugs are not commonly seen by HCPs, the medical affairs team can help you raise awareness regarding the disease. Richard Van LeeuwenMerck

With regards to the price, the starting point is understanding the value and the innovation of the product; the real benefit that this new drug is bringing to the patients. Beside this, we usually consider many different factors, which we believe are equally important. However, it varies from product to product and country to country. The factors and methods that we take into account can usually be divided in two different groups: a) external factors like benchmarks set by similar products (particularly those that target the same indication or are in the same drug class), health system budget impact and payer willingness; and b) internal factors like our assessment of the drug's potential and its running and maintenance costs or the expected return on investment to take into due consideration the costs for development.

Luigi Longinotti


We have learned that to keep patients at the centre of drug development, and to determine how best to support them when the product enters the market, you need to actually talk to patients, their caregivers, and the advocacy societies that work with them every day. In addition, we need to talk to providers who have a genuine interest in helping patients and listen to their feedback. It's all about spending time listening to those stakeholders and acting on their feedback.

Parth R Shah


What to expect
  • A detailed report exploring the latest thinking on orphan drug commercial models, pricing and contracting strategies, stakeholder engagement, the role of medical affairs, evidence generation and proving orphan drug value.
  • An examination of 10 key issues that pharma needs to understand and adopt in their planning
  • 20 targeted questions put to orphan drug experts
  • Their perceptive responses that provided 86 current insights supported by 155 directly quoted comments
Expert Contributors

The report harnesses critical insights from front-line experts who completely understand the commercial and market access challenges of bringing orphan drugs to market. To allow for candid responses, we have agreed to keep some of their names anonymous.
  • Júlio Cesar Avella was the Business Unit Director at Alexion Pharmaceuticals (until 2017), Brazil. He boasts an illustrious career in the biopharma industry with over 21 years of sales and marketing experience.
  • Richard Van Leeuwen is the Business Unit Director at Merck, Netherlands, where he has been responsible for directing a team of professionals (sales, marketing, and medical) in various therapeutic areas, including orphan drug segments.
  • Luigi Longinotti is the Global Head of portfolio and project management for orphan drugs at Recordati, Italy.
  • Jerry Martell is the Regional Business Manager of Rare Diseases at Shire, US
  • Hector de la Riva is the Senior Product Manager of Liver Disease for Gilead Sciences, Spain. In the orphan drugs field, de la Riva has participated in the launch of numerous orphan and ultra-orphan drugs from various perspectives as from Medical Affairs Specialist, to Commercial Strategy Manager.
  • Parth R Shah is the Marketing Director of Haematology/Oncology for Amgen, US, where he is responsible for overseeing the US Oncology Business Unit and leading the Nplate (haematology, rare disease) brand leadership team.
  • Dr Andreas Trinkl is the Business Unit Director Haemophilia DACH, at Swedish Orphan Biovitrum AB (Sobi), Munich, Germany. Andreas is focused on using real-world evidence and facilitating fast communication with selected stakeholders to reduce the time between commercial availability and access for patients.
  • Anonymous is the Senior Director and Global Business Lead for a large US-based pharmaceutical company where he is responsible for the early commercial strategy development of orphan disease compounds.
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1.Subject synopsis
2.Research methodology and objectives
2.2.Which experts were interviewed and why?
3.Key issue summary
4.Key benchmarking insights
4.1.Intelligence exhibit
5.Issues and insights
5.1.Current orphan drug commercial models
5.1.1.Issue summary
5.1.3.Key insights
5.1.4.Supporting quotes
5.1.5.Intelligence exhibits
5.2.Commercial teams within orphan drugs commercial models
5.2.1.Issue summary
5.2.3.Key insights
5.2.4.Supporting quotes
5.2.5.Intelligence exhibits
5.3.Pricing strategies within orphan drug commercial models
5.3.1.Issue summary
5.3.3.Key insights
5.3.4.Supporting quotes
5.3.5.Intelligence exhibits
5.4.Innovative reimbursement contracts within orphan drug commercial models
5.4.1.Issue summary
5.4.3.Key insights
5.4.4.Supporting quotes
5.4.5.Intelligence exhibits
5.5.Regulatory challenges for orphan drug commercial models
5.5.1.Issue summary
5.5.3.Key insights
5.5.4.Supporting quotes
5.5.5.Intelligence exhibits
5.6.Stakeholder engagement within orphan drug commercial models
5.6.1.Patient engagement within orphan drug commercial models
5.6.2.Issue summary
5.6.4.Key insights
5.6.5.Supporting quotes
5.6.6.Intelligence exhibits
5.7.HCP engagement within orphan drugs commercial models
5.7.1.Issue summary
5.7.3.Key insights
5.7.4.Supporting quotes
5.7.5.Intelligence exhibits
5.8.Payer engagement within orphan drug commercial models
5.8.1.Issue summary
5.8.3.Key insights
5.8.4.Supporting quotes
5.8.5.Intelligence exhibits
Value demonstration within orphan drug commercial models
5.2.1.Issue summary
5.2.3.Key insights
5.2.4.Supporting quotes
5.2.5.Intelligence exhibits
Evidence generation within orphan drug commercial models
5.2.1.Issue summary
5.2.3.Key insights
5.2.4.Supporting quotes
5.2.5.Intelligence exhibits
5.9.HCP engagement within orphan drugs commercial models
5.9.1.Issue summary
5.9.3.Key insights
5.9.4.Supporting quotes
5.9.5.Intelligence exhibits
5.10.Payer engagement within orphan drug commercial models
5.10.1.Issue summary
5.10.3.Key insights
5.10.4.Supporting quotes
5.10.5.Intelligence exhibits
5.11.Value demonstration within orphan drug commercial models
5.11.1.Issue summary
5.11.3.Key insights
5.11.4.Supporting quotes
5.11.5.Intelligence exhibits
5.12.Evidence generation within orphan drug commercial models
5.12.1.Issue summary
5.12.3.Key insights
5.12.4.Supporting quotes
5.12.5.Intelligence exhibits
5.13.Overcoming challenges within orphan drug commercial models
5.13.1.Issue summary
5.13.3.Key insights
5.13.4.Supporting quotes
5.13.5.Intelligence exhibits
5.14.The future orphan drug commercial model
5.14.1.Issue summary
5.14.3.Key insights
5.14.4.Supporting quotes
5.14.5.Intelligence exhibits

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