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.


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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