Dolon Publications

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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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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External Publications Co-authored by Dolon Staff

Faciliating More Efficient Negotiations for Innovative Therapies: A Value-Based Negotiation Framework

March 2022
Whittal A, Jommi C, De Pouvourville G, Taylor D, Annemans L, Schoonaert L, Vermeersch S, Hutchings A, Patris J.

Facilitating More Efficient Negotiations for Innovative Therapies: A Value-Based Negotiation Framework. International Journal of Technology Assessment in Health Care. 2022;38(1).

https://doi.org/10.1017/S0266462322000095 View
Consideration of quality of life in the health technology assessments of rare disease treatments

September 2021
Nicod E, Meregaglia M, Whittal A, Upadhyaya S, Facey K, Drummond M.

Consideration of quality of life in the health technology assessments of rare disease treatments. The European Journal of Health Economics. 2021 Oct 29:1-25.

https://doi.org/10.1007/s10198-021-01387-w View
Implementing Outcomes‐Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and Tisagenlecleucel

July 2021
Facey KM, Espin J, Kent E, Link A, Nicod E, O'Leary A, Xoxi E, van de Vijver I, Zaremba A, Benisheva T, Vagoras A, Upadhyaya S.

Implementing outcomes-based managed entry agreements for rare disease treatments: nusinersen and tisagenlecleucel. Pharmacoeconomics. 2021 Sep;39(9):1021-44.

https://doi.org/10.1007/s40273-021-01050-5 View
Examining the impact of different country processes for appraising rare disease treatments: a case study analysis

March 2021
Whittal A, Nicod E, Drummond M, Facey K.

Examining the impact of different country processes for appraising rare disease treatments: a case study analysis. International journal of technology assessment in health care. 2021;37(1).

https://doi.org/10.1017/S0266462321000337 View
The Use of Patient‐Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment

January 2021
Whittal A, Meregaglia M, Nicod E.

The use of patient-reported outcome measures in rare diseases and implications for health technology assessment. The Patient-Patient-Centered Outcomes Research. 2021 Sep;14(5):485-503.

https://doi.org/10.1007/s40271-020-00493-w View
The estimation of health state utility values in rare diseases: overview of existing techniques

August 2020
Meregaglia M, Nicod E, Drummond M.

The estimation of health state utility values in rare diseases: overview of existing techniques. International Journal of Technology Assessment in Health Care. 2020 Oct;36(5):469-73.

https://doi.org/10.1017/S0266462320000665 View
Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

July 2020
Nicod E, Whittal A, Drummond M, Facey K.

Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches. Orphanet journal of rare diseases. 2020 Dec;15(1):1-4.

https://doi.org/10.1186/s13023-020-01462-0 View
An analysis of orphan medicine expenditure in Europe: is it sustainable?

January 2020
Mestre-Ferrandiz J, Palaska C, Kelly T, Hutchings A, Parnaby A.

An analysis of orphan medicine expenditure in Europe: is it sustainable? Orphanet journal of rare diseases. 2019 Dec;14(1):1-5.

https://doi.org/10.1186/s13023-019-1246-7 View
Expenditure in EU5 to 2021: Adjusting for the Impact of Discounts and Rebates

August 2018
Espin J, Schlander M, Godman B, Anderson P, Mestre-Ferrandiz J, Borget I, Hutchings A, Flostrand S, Parnaby A, Jommi C.

Projecting pharmaceutical expenditure in EU5 to 2021: adjusting for the impact of discounts and rebates. Applied health economics and health policy. 2018 Dec;16(6):803-17.

https://doi.org/10.1007/s40258-018-0419-1 View
Dealing with Uncertainty and Accounting for Social Value Judgments in Assessments of Orphan Drugs: Evidence from Four European Countries

May 2017
Nicod E, Berg Brigham K, Durand-Zaleski I, Kanavos P.

Dealing with uncertainty and accounting for social value judgments in assessments of orphan drugs: evidence from four European countries. Value in Health. 2017 Jul 1;20(7):919-26.

https://doi.org/10.1016/j.jval.2017.03.005 View
HTA programme response to the challenges of dealing with orphan medicinal products: process evaluation in selected European countries

April 2017
Nicod E, Annemans L, Bucsics A, Lee A, Upadhyaya S, Facey K.

HTA programme response to the challenges of dealing with orphan medicinal products: process evaluation in selected European countries. Health Policy. 2019 Feb 1;123(2):140-51.

https://doi.org/10.1016/j.healthpol.2017.03.009 View
Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)

March 2017
Annemans L, Aymé S, Le Cam Y, Facey K, Gunther P, Nicod E, Reni M, Roux JL, Schlander M, Taylor D, Tomino C, Torrent-Farnell J, Upadhyaya S, Hutchings A, Le Dez L.

Recommendations from the European working Group for Value Assessment and Funding Processes in rare diseases (ORPH-VAL). Orphanet journal of rare diseases, 12(1), pp.1-15.

https://doi.org/10.1186/s13023-017-0601-9 View
Scientific and Social Value Judgments for Orphan Drugs in Health Technology Assessment

September 2016
Nicod E, Kanavos P.

Scientific and social value judgments for orphan drugs in health technology assessment. International Journal of Technology Assessment in Health Care. 2016;32(4):218-32.

https://doi.org/10.1017/S0266462316000416 View
Principles for consistent value assessment and sustainable funding of orphan drugs in Europe

May 2015
Gutierrez L, Patris J, Hutchings A, Cowell W.

Principles for consistent value assessment and sustainable funding of orphan drugs in Europe. Orphanet journal of rare diseases. 2015 Dec;10(1):1-9.

https://doi.org/10.1186/s13023-015-0269-y View
Estimating the budget impact of orphan drugs in Sweden and France 2013–2020

February 2014
Hutchings A, Schey C, Dutton R, Antonov K.

Estimating the budget impact of orphan drugs in Sweden and France 2013–2020. Orphanet Journal of Rare Diseases. 2014 Dec;9(1):1-9.

https://doi.org/10.1186/1750-1172-9-22 View
A Pilot Study of Multicriteria Decision Analysis for Valuing Orphan Medicines

November 2013
Sussex J, Rollet P, Garau M, Hutchings A.

A pilot study of multicriteria decision analysis for valuing orphan medicines. Value in Health. 2013 Dec 1;16(8):1163-9.

https://doi.org/10.1016/j.jval.2013.10.002 View
What Is Wrong with Orphan Drug Policies? Suggestions for Ways Forward

October 2012
Kanavos P, Nicod E.

What is wrong with orphan drug policies? Suggestions for ways forward. Value in health. 2012 Dec 1;15(8):1182-4.

https://doi.org/10.1016/j.jval.2012.08.2202 View
Estimating the budget impact of orphan medicines in Europe: 2010–2020

September 2011
Schey C, Milanova T, Hutchings A.

Estimating the budget impact of orphan medicines in Europe: 2010-2020. Orphanet journal of rare diseases. 2011 Dec;6(1):1-0.

https://doi.org/10.1186/1750-1172-6-62 View