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Principles for consistent value assessment and sustainable funding of orphan drugs in Europe


, : Principles for consistent value assessment and sustainable funding of orphan drugs in Europe. Orphanet Journal of Rare Diseases 10: 53-53

The European Orphan Medicinal Products (OMP) Regulation has successfully encouraged research to develop treatments for rare diseases resulting in the authorisation of new OMPs in Europe. While decisions on OMP designation and marketing authorisation are made at the European Union level, reimbursement decisions are made at the national level. OMP value and affordability are high priority issues for policymakers and decisions regarding their pricing and funding are highly complex. There is currently no European consensus on how OMP value should be assessed and inequalities of access to OMPs have previously been observed. Against this background, policy makers in many countries are considering reforms to improve access to OMPs. This paper proposes ten principles to be considered when undertaking such reforms, from the perspective of an OMP manufacturer. We recommend the continued prioritisation of rare diseases by policymakers, an increased alignment between payer and regulatory frameworks, pricing centred on OMP value, and mechanisms to ensure long-term financial sustainability allowing a continuous and virtuous development of OMPs. Our recommendations support the development of more consistent frameworks and encourage collaboration between all stakeholders, including research-based industry, payers, clinicians, and patients.

US$19.90

PMID: 25935555

DOI: 10.1186/s13023-015-0269-y


Other references

Hoffmann, KH.; Neuhauser, T.; Lorenz, MW.; Stay, B., 1994: Structure and immunological properties of allatoregulating peptides from a cricket, Gryllus bimaculatus. Unknown

Basch, C.H.; Hillyer, G.Clarke.; MacDonald, Z.L.; Reeves, R.; Basch, C.E., 2016: Characteristics of YouTube™ Videos Related to Mammography. With a monthly total of more than one billion unique visitors, YouTube™ is one of the Internet's most visited websites and contributes to the growing amount of health-related information on the Internet. The purpose of this study was to ana...

Peakall Jeff, 1994: Neotectonic effects on half-graben alluvial stratigraphy; a quantitative field example. International Sedimentological Congress 14: D65-D66

Costa, R.; Afonso, E.; Benedito, M.; Maricato, L., 1991: Poland's syndrome associated with chronic granulocytic leukemia. Poland's syndrome has been sporadically associated with haematological neoplasms, namely acute lymphoblastic and myeloblastic leukaemias and non-Hodgkin's lymphomas. The authors present the case of a child in whom this syndrome coexists...

Sackett D.H., 1968: Oates, NE Devonian, Pecos County, Texas. Publication - West Texas Geological Society Pages 154-155

Serrallach-Mila, N.; Saladie-Roig, J.M.; Franco-Miranda, E., 1977: Renal autotransplatation as a method of revascularization in vasculorenal hypertension. Evaluation of a technic after 11 years of its 1rst use. Actas Urologicas Espanolas 1(1): 13-16

Il'in, G.S., 1959: On the biosynthesis of nicotine.. Using nutrient solutions, with and without N, but which contained NaHC14O3, it was shown that nicotine in tobacco is a secondary synthetic product not dependent on the uptake of N by the roots.

Shigei, M., 1977: A new species of the echinothurid sea-urchin, Sperosoma nudum, from the North Pacific, off central Japan. Journal FacTokyo Univ. (Zool.): 7177,illust.

Wormell, P., 1974: The landlord with the bowler. Big farm management: 49-53

Robb-Nicholson, C., 2012: Ask the doctor. I have urinary incontinence and have heard bladder training might help. Could it stave off surgery?. Harvard Women's Health Watch 20(2): 2-2