Dolon Publications

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…

Read more…
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…

Read more…
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…

Read more…
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…

Read more…

External Publications Co-authored by Dolon Staff

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 J Rare Dis 14, 287 (2019).

https://doi.org/10.1186/s13023-019-1246-7 Download
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. Appl Health Econ Health Policy 16, 803–817 (2018).

https://doi.org/10.1007/s40258-018-0419-1 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 J Rare Dis 12, 50 (2017)

https://doi.org/10.1186/s13023-017-0601-9 Download
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 J Rare Dis 10, 53 (2015).

https://doi.org/10.1186/s13023-015-0269-y Download
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 J Rare Dis 9, 22 (2014).

https://doi.org/10.1186/1750-1172-9-22 Download
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 Health 16, 1163-1169 (2013).

https://doi.org/10.1016/j.jval.2013.10.002 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 J Rare Dis 6, 62 (2011).

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