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. 2020 Sep 3;15(1):235.
doi: 10.1186/s13023-020-01519-0.

Registries for orphan drugs: generating evidence or marketing tools?

Carla E M Hollak  1   2 , Sandra Sirrs  3 , Sibren van den Berg  4 , Vincent van der Wel  4 , Mirjam Langeveld  5 , Hanka Dekker  6 , Robin Lachmann  7 , Saco J de Visser  4
Affiliations

Registries for orphan drugs: generating evidence or marketing tools?

Carla E M Hollak et al. Orphanet J Rare Dis. .

Abstract

Independent disease registries for pre-and post-approval of novel treatments for rare diseases are increasingly important for healthcare professionals, patients, regulators and the pharmaceutical industry. Current registries for rare diseases to evaluate orphan drugs are mainly set up and owned by the pharmaceutical industry which leads to unacceptable conflicts of interest. To ensure independence from commercial interests, disease registries should be set up and maintained by healthcare professionals and patients. Public funding should be directed towards an early establishment of international registries for orphan diseases, ideally well before novel treatments are introduced. Regulatory bodies should insist on the use of data from independent disease registries rather than company driven, drug-oriented registries.

Keywords: Disease registries; Orphan drugs; Real world data; Registries.

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Conflict of interest statement

CH receives payments from the National Healthcare Institute (The Netherlands) as member of the “Horizonscan” for new drugs for metabolic and endocrine disorders and the Dutch Advisory Committee to the insured Package (ACP). She is an unpaid member of Takeda’s Humanitarian Aid Program.

CH and ML are involved in pre-marketing studies with orphan drugs for which the Amsterdam University Medical Center receives financial compensation for study costs from Sanofi, Protalix and Idorsia.

SS participated in research projects funded by Sanofi, Takeda, Idorsia, Actelion, and Amicus and received support for travel to attend educational meetings from Amicus and Sanofi. She participated in an advisory board for Amicus. The Adult Metabolic Diseases Clinic at Vancouver General Hospital receives unrestricted grant funding from Sanofi, Takeda, and Amicus to support the services of a genetic counselor for patients of the clinic. She is an unpaid member of the multidiscliplinary committee that provides input to our provincial ministry of health on the use of drugs for rare diseases.

VvdW is director and shareholder of the company “Patient One”, which develops and orphan drug according to Fair Medicine principles. He is employed by the Fair Medicine Foundation, that is financed by the Dutch government to develop novel pharmaceutical business models.

HD receives payments from the National Healthcare Institute as member of the “Horizonscan” for new drugs for metabolic and endocrine disorders. VKS has received financial compensation for work on patient friendly texts from Lysogene and Chiesi. She receives travel support but no fees for patient advisory boards of Sanofi, Takeda, Ultragenyx and Orchard Therapies.

RL has received consulting fees from Sanofi/Genzyme, Biomarin and Kyowa Kirin International and honoraria and travel support from Sanofi/Genzyme and Takeda His department receives staff funding from Kyowa Kirin International and research grants from Nutricia. He is unpaid Chair of the Scientific Committee of the Recordati Rare Diseases Foundation.

SvdB and SdV declare no conflicts of interest.

Figures

Fig. 1
Fig. 1
Origin of registries (data from the Encepp PAS database, risk management plan, European public assessment report (EPAR) or publicly available information) [–15]
See this image and copyright information in PMC

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References

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