The Dolon Institute aspires to contribute to the policy conversations around patient access and promoting innovation in rare and severe diseases. Our work is grounded in the fundamentals of orphan medicine economics and experience engaging with stakeholders around the launch of orphan medicines. We seek to develop analyses and evidence-led insights disseminated through peer-reviewed publications, white papers and external events.
What do the new Medicare negotiations mean for pipeline orphan therapies?
Rob Wood, Ema Slapikaite, Katarina Kitarovic, Sarah Forbes
March 2025
Our latest infographic breaks down a critical, but under-discussed, implication of the Medicare drug price negotiations: the risk to orphan medicines. While the IRA aims to lower the cost of some therapies, its structure may unintentionally discourage biopharma companies from…
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ISPOR Europe panel report: Pricing and reimbursement of multiple indication medicines: Can a balance be found between different stakeholder perspectives to optimise value and access for patients, while ensuring sustainable and affordable innovation?
March 2025
This ISPOR Europe 2024 panel report explores pricing and reimbursement (P&R) of multiple medicines in Europe. Work has culminated from the panellists and audience of ~800 participants, where they discussed key challenges and forward-looking solutions for multi-indication products. The insights…
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How might JCA impact orphan sustainability in Europe?
Isabelle Laurence, Emilie Neez, Adam Hutchings
February 2025
In recognition of Rare Disease Day 2025, we turn our attention to EU Joint Clinical Assessment (JCA), to explore the potential impact of JCA across three key dimensions important to sustaining investment and launch of new rare disease treatments in…
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2025 themes for pricing and access success for rare disease and oncology medicines
Richard Sear, Emilie Neez, Richard Dutton
January 2025
In this report, explore six themes Dolon believes will impact pricing and access success for rare disease and oncology medicines in 2025. Theme 1: Rising pricing tension Theme 2: Protectionist Policies Theme 3: GLP-1 Wave Theme 4: Oncology Crowding Theme…
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Joint Clinical Assessment: The finger on the scale for orphan sustainability in Europe?
Emilie Neez, Elisa Garau, Adam Hutchings
January 2025
EU Joint Clinical Assessment (JCA) has the potential to transform the evaluation of medicines in Europe, including orphan drugs. While there’s hope that JCAs will streamline fragmented processes and improve patient access, the road ahead is far from simple. Concerns…
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Evolution of willingness to pay in cost-effectiveness countries United Kingdom, Australia, Canada and Sweden
Lindsay Kreill, Emilie Neez
May 2024
Willingness to pay for new medicines has steadily declined in real terms in countries that incorporate cost-effectiveness thresholds in their pricing and reimbursement (P&R) processes. Nations such as the UK, Australia, Canada, and Sweden have not adjusted their thresholds for…
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Mini-series – Realizing the potential of gene therapy: how do we pave the path to patient delivery and access?
Pfizer, ARM, Dolon
October 2024
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant…
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Promoting multi-stakeholder education to support access to gene therapy
Pfizer, ARM, Dolon
October 2024
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant…
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Enhancing the delivery of gene therapy through specific care models: examining centers of excellence
Pfizer, ARM, Dolon
October 2024
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant…
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Facilitating effective health technology assessment for gene therapy
Pfizer, ARM, Dolon
October 2024
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant…
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Novel payment models to facilitate patient access to gene therapy
Pfizer, ARM, Dolon
October 2024
While gene therapies offer the potentially groundbreaking opportunity to improve outcomes for individuals with rare and severe diseases, their adoption introduces distinct challenges for health systems that are currently tailored for more conventional medicines. As such, there is a significant…
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ISPOR Europe panel: How do we attain sustainability, affordability and availability of CAR T therapies?
Amanda Whittal
November 2023
This ISPOR Europe panel explored the evolving landscape of CAR T therapies, their high potential for lifelong benefits, and the economic and logistical hurdles they face. From complex manufacturing to unique pricing and reimbursement challenges, CAR T therapies require novel…
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Exploring alternative pricing approaches for orphan medicines
Richard Sear, Emilie Neez
March 2024
On March 12th 2024, Richard Sear and Emilie Neez explored alternative pricing approaches for orphan medicines in a presentation at the World Evidence Pricing and Access Congress in Amsterdam. Supporting slides are available here; this companion piece provides our (the…
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Revision of the General Pharmaceutical Legislation: Impact Assessment of Key Orphan Proposals on Member States
Isabelle Laurence, Emilie Neez, Elena Nicod
May 2024
While the revision of the General Pharmaceutical Legislation (GPL) targets EU-level frameworks and processes, proposed changes are sure to have immediate as well as downstream consequences at the country level. This paper explores these potential consequences, focusing on a selection…
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What does it take to see continued rare disease innovation in Europe?
Isabelle Laurence, Lindsay Kreill, Adam Hutchings, Gisela Rovira Tomas, Emilie Neez
February 2024
In recognition of Rare Disease Day 2024, we explore four critical elements to sustaining rare disease innovation in Europe. These elements are outlined below, building on published work by Dolon team members.
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Revision of the General Pharmaceutical Legislation: Impact Assessment of European Commission and EFPIA proposals
Emilie Neez, Lindsay Kreill, Ai Xi Souw, Adam Hutchings
November 2023
The European Commission has proposed revisions to the Pharmaceutical Legislation, with the view to tackle unmet medical needs, competitiveness, access and affordability. This update represents a once-in-a-generation opportunity to strengthen the European biopharmaceutical ecosystem, if fit-for-purpose policy options are implemented…
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Rare innovation: How it happens, when it doesn’t, and what can be done to sustain it
Isabelle Laurence, Adam Hutchings, Elena Nicod
October 2023
Addressing remaining unmet needs in rare diseases hinges on a positive and enduring policy environment which helps to achieve the necessary clinical and economic conditions for drug development in small, hard-to-treat populations. This white paper examines through historic case analysis…
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Revision of the Orphan Regulation: Estimated impact on incentives for innovation of changes proposed by the European Commission
Emilie Neez, Adam Hutchings
September 2023
Despite the tremendous therapeutic advances delivered in rare diseases since the adoption of the Orphan Regulation in 2000, the European Commission has proposed a set of legislative revisions aimed at bolstering innovation whilst enhancing access and affordability. This study, based…
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Amyotrophic Lateral Sclerosis, a rare neurodegenerative disease: European landscape assessment and policy recommendations for improved diagnosis, care, and treatment.
Kate Quigley, Gisela Rovira Tomas, Elena Nicod
September 2023
The European Amyotrophic Lateral Sclerosis (ALS) Coalition has published a paper that sets forth 10 policy recommendations aiming to improve the lives of people living with ALS and their caregivers. The paper sheds light on what it means to receive…
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Innovative contracting for ATMPs in Europe: Recent learnings from the manufacturer experience
Adam Hutchings
August 2023
The past 5 years have been a period of great innovation for cell and gene therapy with a wave of very exciting and effective therapies approved in Europe. While the clinical potential of these products are great, there are recognised…
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Paid early access in Europe – Is it needed, and what should it look like?
Tom Kelly, Joceleyn Triest
June 2023
Dolon sponsored World EPA Congress 2023 and had the opportunity to present at the conference. In this infographic, we provide a summary of key discussion points from the roundtable ‘Paid early access in Europe – Is it needed, and what…
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Why do rare diseases warrant special treatment?
Adam Hutchings, Emilie Neez, Amanda Whittal, Tom Kelly, Lindsay Kreill
February 2023
In recognition of Rare Disease Day 2023, we explore four key reasons why rare diseases warrant special treatment. These reasons are summarised below, building on published work by Dolon team members.
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Introducing the Dolon Institute
Adam Hutchings, Emilie Neez, Elena Nicod, Amanda Whittal
February 2023
Dolon is pleased to announce the launch of the Dolon Institute, which aspires to contribute to the policy discussion that surrounds the development and availability of innovative medicines for rare and severe diseases globally. We hope to do our part…
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Absence of comparative survival data in health technology assessment of oncology therapies
Emily Groves, Adam Hutchings
January 2023
This paper brings together a multi-disciplinary panel of expert co-authors who share perspectives on the current challenges, implications and potential solutions for absence of comparative survival data in health technology assessment (HTA) of oncology therapies.
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Exploring the economics of gene therapy innovation and price
Amanda Whittal, Esther Idicula, Adam Hutchings
January 2022
Gene therapies are revolutionising the treatment of severe conditions, offering previously unattainable advances in health gains. Simultaneously, this therapeutic potential is matched by concerns around price and affordability. Are these fears reflective of reality? To address this question requires an…
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Addressing unmet needs in extremely rare and paediatric-onset diseases: how the biopharmaceutical innovation model can help identify current issues and find potential solutions
Emilie Neez, Arianna Gentillini, Adam Hutchings
July 2021
Although the Orphan and Paediatric Regulations have been successful at stimulating innovation in these previously underserved areas, significant unmet medical needs remain. EFPIA held in May 2021 a workshop, which gathered Senior staff from member company R&D, Clinical, Strategy, and…
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How did we get here and where do we go now? An economist’s perspective on EU orphan drug policy
Adam Hutchings
February 2021
The divergence between the rapid pace of scientific progress in rare diseases and declining sentiment towards orphan medicines reflects a paradox that is at the heart of the European orphan drug debate. While the last twenty years have witnessed a…
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Estimated impact of EU Orphan Regulation on incentives for innovation
Emilie Neez, Arianna Gentillini, Richard Dutton, Adam Hutchings
October 2020
The European Commission is currently reviewing the 2000 Regulation on orphan medicinal products (OMP). It is critical that any consideration of reform of the Regulation should be informed by a robust understanding of the relationship between incentives, investment, innovation and…
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A review of the challenges and proposals for improving patient access to advanced therapeutic medicinal products in 10 EU countries
RARE IMPACT Working Group
January 2020
Gene and cell therapies represent some of the most advanced treatments being employed in biomedical research to improve the length and quality of life of patients with rare and complex diseases. Patients’ access to these therapies has been hampered by…
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