What are the essential factors for orphan drug success?
Several orphan drugs have proven to be commercial winners, so what are the key ingredients for success in 2020? For example, as the sector has evolved the power of the patient voice has grown in significance and has led to the emergence of "Patient KOLs". The value of rare disease patient input, from the setting of enabling research to influencing regulators, HTAs and payers, has become a central feature in orphan drug launches. The market dynamics of taking an orphan drug to market differ significantly from commercializing mainstream drugs—so what lessons do experts have for research, stakeholder, evidence and pricing strategies that will deliver success?
To explore the organizational structures and commercial models that deliver both ROI and patient benefit we interviewed, in July 2020, rare disease experts from companies such as UCB, Takeda and Recordati. In Orphan Drugs: Benchmarking Commercial Models 2020 they examine in-depth the models, practice and strategies that are critical for successful orphan drug programs.
Rare disease experts explore key questions such as...
- How are the most important success factors determined, and how do they vary across orphan drugs/therapeutic areas?
- What are the different internal teams that are essential for successful orphan drug commercialization, and what unique roles do they perform?
- What pricing models are being adopted to ensure orphan drugs are affordable and yet achieve a return on investment?
- What is the role of patients in the design of clinical trials for orphan drugs, and how does such involvement impact the overall commercial success of a drug?
- What are the most important action points for creating awareness of a rare disease, and how can these be integrated into a successful commercial model?
- What innovative reimbursement contracts are proving to be most successful in the orphan drug space, and why?
What Experts say...
"If there is too late an approach to the market, physicians, payers, and policymakers, you'll have to start from scratch with your commercial model – otherwise, you'll have a drug that patients have no access to, which impairs the commercial success of the drug launch. Externally, a lack of awareness, understanding, and strategy contributes to the failure of orphan drug models. Internally, success is about coordinating the activity of medical, economic, epidemiological, and clinical aspects."
Claudio Marelli Takeda Pharmaceuticals
"Engagement with patients and patient advocacy groups is very important in many aspects, and without any doubt, must start from involving patients in the design of the main pivotal trials. Nowadays, patients are a strong stakeholder sitting at the decision-making tables of regulators, payers and HTAs. They also voice their needs, feelings, and what matters to them in terms of improvements; these can then be measured as endpoints in trials. Also, patients are being listened to by regulators, payers and HTAs, to help this community to learn about these rare and minimally-described conditions."
Paula Albuquerque UCB
"Presenting a cohort of patients and case studies from real life is going to demonstrate orphan drug value. Not everybody can publish a cohort of several patients for a rare disease because not everybody can have that many patients. In this case, even one patient of a unique, rare disease is something that can demonstrate orphan drug value."
Gaël Le Rouzo EUSA Pharma
Case studies included in the report
- International cooperation is critical for developing new treatments for neuroblastoma: The role of SIOPEN
- Exubera's poor R&D doomed the orphan drug's commercial success
- LEO Pharma's 'Nothing about You without You' campaign in rare diseases
- The contribution of the Cystic Fibrosis Foundation brought successful commercialization of a drug candidate for the disease
- European Reference Network on Rare Endocrine Conditions—Raising awareness of rare diseases
- Innovative reimbursement contracts—Novartis & Gilead
What to expect
A detailed report exploring, through expert insights, current thinking on commercial models for orphan drugs and the essential "must haves" for success:
- An examination of 9 key issues that are impacting commercial strategies for orphan drugs
- 15 targeted questions put to industry experts
- Their responses which provided 34 insights supported by 103 directly quoted comments
Deliverables: Includes PDF report and PowerPoint slide deck
Orphan Drugs: Benchmarking Commercial Models 2020 is delivered as:
- A detailed PDF report including all insights, quotes and intelligence exhibits
- A useful PowerPoint slide deck providing a summary of the report's key findings to include in your presentations and for sharing with colleagues
Experts contributing to this report have been screened to ensure they:
- Are pharma professionals with at least 10 years' experience specifically in orphan drugs
- For at least five years have had direct involvement in the commercialization of orphan drugs
- Are experienced in successful orphan drug commercialization
Experts interviewed included:
- Paula Albuquerque is the Director, Global Payer Value Strategy Lead for rare diseases at UCB, Belgium. She has worked at UCB for over 2 years, focusing on immunology and neurology. Paula has over 16 years' worth of experience within the pharmaceutical industry, at both a local and global level, and is passionate about improving access to novel transformative science for patients with severe or rare diseases. Working for 12 of the top 25 pharmaceutical companies, Paula is highly knowledgeable about HEOR methods, pricing control mechanisms and international health policies. She has negotiated price and reimbursement for 13 global brands, including 2 orphan drugs.
- Júlio Cesar Avella is the Commercial and Marketing Director at Ultragenyx Pharmaceuticals, Brazil. His career in the biopharma industry spans more than 23 years of sales and marketing experience. For 13 of those years, Júlio has worked on the commercial side, including within the commercialization of drugs for rare and ultra-rare diseases such as PNH (Paroxysmal nocturnal haemoglobinuria - Haematology) at Alexion Pharmaceuticals. Successful launches of blockbuster brands within the rare disease space include Crysvita (burosumab), Soliris (eculizumab), Humira (adalimumab) and Avonex (interferon beta-1a).
- Olivier Delannoy is the Vice President of Pierre Fabre Pharmaceuticals USA, where he is responsible for the management of all pharma business units. His expertise in the field of sales management includes new product launches, product life cycle management and commercial leadership across various therapeutic areas. As VP at Pierre Fabre, Olivier oversaw a 10% growth in Rx volume of an orphan drug indicated for infantile haemangioma. With his experience in the commercial management of orphan drugs and other products, Olivier provides valuable insights into the issue of commercial models for orphan drugs.
- Camilla Krough Lauritzen joined LEO Pharma, Denmark, as their Chief Patient Officer and Head of Patient Engagement for Rare Diseases in May 2018. Prior to this, she founded and led the Patient Relation function at Abbott, AbbVie and Novo Nordisk. Before joining the pharmaceutical industry, she held roles in patient organizations and patient/science alliances. Camilla believes that 'patients' themselves are disease experience experts, establishing a pioneering 'Nothing About You Without You' concept in the rare disease unit at LEO Pharma in August 2019. This concept commits to getting insight into rare diseases from patients and their immediate caregivers. Camilla and her team are passionate about developing interventions against autoimmune and genetic diseases that currently have no available therapies.
- Claudio Marelli is the Global Medical Lead for Rare Metabolic Diseases at Takeda Pharmaceuticals, UK. For this report, Claudio draws on his expertise in orphan drug research to provide crucial insights on the key components of successful commercial models. He has over three decades of experience across various pharmaceutical companies, including Shire, ViroPharma, and most recently, Takeda, specializing in orphan drugs. Other areas of expertise include Cardiology and Endocrinology, Drug Research and Development, and Global Disease Strategy. In the last three years, Claudio has led the European Medical Affairs Endocrinology division in orphan drug research for a rare disease.
- Massimo Mineo is the Managing Director and General Manager for Europe and Middle at Recordati Rare Diseases, France. Massimo has accumulated over two decades of experience in corporate strategy planning and commercial pharmaceutical product and/or development compound licensing. He has applied his expertise to the orphan drug market, where Recordati Rare Diseases serves as the Marketing Authorization Holder of Osilodrostat, a drug for adults with Cushing's Syndrome.
- Gaël Le Rouzo is the Regional Business Director of Emerging Markets at EUSA Pharma, where he is currently responsible for the sales development of the orphan drug siltuximab (Sylvant) in the Latin American and Asia-Pacific markets. His sales experience in the pharma industry spans over a decade, with a track record of sales growth and pricing and reimbursement strategies across multiple regions. Gaël has also been involved with patient access programs and disease awareness campaigns for pediatric cancers and other rare diseases.
- Anonymous is a Market Access Expert with over 9 years of experience in various market access roles across several leading pharma and biotech companies. A seasoned MBA pharma professional with expertise in speciality care, vaccines and orphan drugs, Anonymous is currently the Lead of Regional Market Access for a multinational pharmaceutical company in the US. In his current role, he leads and designs the HEOR, pricing and value proposition for orphan drugs and speciality care. In previous roles, he has also functioned as a rare disease expert responsible for market access tactics, including strategies to increase penetration of speciality care high-cost molecules and take a competitive advantage over competitors.
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