Principles for consistent value assessment and sustainable funding of orphan drugs in EuropeOrphanet Journal of Rare Diseases


Laura Gutierrez, Julien Patris, Adam Hutchings, Warren Cowell
Medicine (all) / Pharmacology (medical) / Genetics (clinical)


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Pedro Franco

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Dennis P. Swanson


POSITION STATEMENT p arr tio o e are


M er kin on ic population (between 27 and 36 million people [4]), greater momentum in OMP development and approval [10-13]. Such variations can be due to restrictions of reimbursement or funding, either because of a negaGutierrez et al. Orphanet Journal of Rare Diseases (2015) 10:53

DOI 10.1186/s13023-015-0269-yFor example, in the United Kingdom (UK) the National3Dolon Ltd, 17-185 Gray’s Inn Road, London, UK

Full list of author information is available at the end of the articlewill be necessary if the public health burden of rare diseases is to be addressed.

Whether this goal is reached will be determined in part by the outcome of an ongoing debate about the value of OMPs and the mechanisms by which tive assessment by policymakers or because the drug has not been considered for reimbursement [10,11,14-17].

Negative reimbursement decisions can reflect problems in applying standard pricing and reimbursement processes to OMPs [18]. An awareness of this issue has led some European countries to adapt their processes to reflect the specificities of OMPs [6,12,19-22].* Correspondence: adam.hutchings@dolon.comlong-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.

Keywords: Orphan medicinal products, Rare diseases, Value assessment


The growth in the number of approved orphan medicinal products (OMPs) in Europe since the introduction of legislation in 2000 has been a success for rare disease patients, the scientific and medical community,

European and national policymakers, and the pharmaceutical industry. To date, one hundred and twelve (112) OMPs [1] have received marketing authorisation in Europe for the treatment of rare diseases associated with high unmet need and limited suitable therapeutic options [2,3]. Given the estimated 5000–8000 rare diseases that affect 6–8% of the European Union (EU)

European healthcare systems assess, reimburse and fund such treatments [5-9]. While the regulatory framework has been adapted to the specificities of

OMPs by creating an expert Committee for Orphan

Medicinal Products (COMP) within the European

Medicines Agency (EMA), many national pricing and reimbursement systems have not been adapted to respond to the specific challenges of these types of drugs.

Regional and local inequalities of access to OMPs have previously been observed in Europe, where

Member States have responsibility for health policyPrinciples for consistent v sustainable funding of or

Laura Gutierrez1, Julien Patris2, Adam Hutchings3* and W


The European Orphan Medicinal Products (OMP) Regula treatments for rare diseases resulting in the authorisation designation and marketing authorisation are made at th made at the national level. OMP value and affordability regarding their pricing and funding are highly complex. value should be assessed and inequalities of access to O background, policy makers in many countries are consid proposes ten principles to be considered when underta manufacturer. We recommend the continued prioritisati alignment between payer and regulatory frameworks, pr© 2015 Gutierrez et al.; licensee BioMed Centr

Commons Attribution License (http://creativec reproduction in any medium, provided the or

Dedication waiver (http://creativecommons.or unless otherwise stated.Open Access alue assessment and han drugs in Europe en Cowell4 n has successfully encouraged research to develop f new OMPs in Europe. While decisions on OMP

European Union level, reimbursement decisions are high priority issues for policymakers and decisions ere is currently no European consensus on how OMP

Ps have previously been observed. Against this ing reforms to improve access to OMPs. This paper g such reforms, from the perspective of an OMP of rare diseases by policymakers, an increased ing centred on OMP value, and mechanisms to ensureal. This is an Open Access article distributed under the terms of the Creative, which permits unrestricted use, distribution, and iginal work is properly credited. The Creative Commons Public Domain g/publicdomain/zero/1.0/) applies to the data made available in this article,

Institute for Health and Care Excellence (NICE) recently created a separate programme for ultra-rare drugs that uses different assessment criteria to those applied to treatments for more common disorders [23,24].

Despite important initiatives to improve conformity of OMP assessment methods for reimbursement purposes at a European level, there is as yet no consensus on how OMP value should be evaluated [20]. This uncertainty has important consequences for healthcare policy makers, manufacturers and patients. Healthcare policymakers may lack confidence in the ability of their healthcare systems to ensure optimal patient access to

OMPs while ensuring value for money and long-term financial sustainability. Manufacturers often face an unDiscussion

Value assessment of OMPs

Principle 1: National pricing and reimbursement processes should acknowledge the COMP’s assessment of therapeutic benefit

The European OMP Regulation defines rare diseases as having a prevalence of no more than 5 in 10,000 people, and as diseases that are life-threatening or chronically debilitating [2,3]. Due to their inherent rarity and complexity, these diseases are often less well characterised or understood. There is often limited information on rare disease pathophysiology, mechanisms or genesis, a lack of relevant animal models and problems with accurate diagnosis [25]. Information on the disease process, epidemiology, prognosis, and bures dis t el inc m sur ter d t an

Gutierrez et al. Orphanet Journal of Rare Diseases (2015) 10:53 Page 2 of 9clear, inconsistent and changing array of systems and policies that create uncertainty about the long-term return from OMP development and provide little clarity of the data requirements to adequately ensure patient access [14].