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MEETING ABSTRACT Open Access
A coordinated EU approach to informed accessdecisions: CAVOD process proposals – thepossibility to turn concept into reality?Wills Hughes-Wilson
From 6th European Conference on Rare Diseases and Orphan ProductsBrussels, Belgium. 23-25 May 2012
BackgroundThe European orphan legislative framework has beensuccessful in delivering approved new treatments sinceits establishment in 2000. But the data needed to reach apositive risk-benefit analysis for regulatory approval oftenfalls short of that needed by Member States to justifyreimbursed availability to patients. This gap leads to dif-ferences in availability of new orphan medicines. TheCAVOD proposals build on more than 12 years of formalcollaborative approaches to European orphan drug pol-icy, which have consistently agreed on the need to gatherand share information at a European level. They wouldestablish a process for the exchange of knowledge fromthe earliest stage in a drug’s development, through to in-life outcomes after a treatment is available to patients,with the objective of bundling fragmented know-how toallow the timely production of well-informed decisionson national pricing and reimbursement, while respectingexisting roles, responsibilities and competences.
Methodology and contentThe approach intends to optimise processes, notably atfour key time points. All activities should be based onexisting and planned roles, responsibilities and legislativeframeworks, and in collaboration between all partiesinvolved at Member State and EU level, including regula-tors, HTA bodies, payers, patients and the sponsor. Earlydialogue between the EMA, regulators and HTA bodiesshould be instigated at the time of orphan designation andthe assumption of significant benefit. This should continuethrough protocol assistance to the Marketing Authorisa-tion and confirmation of the significant benefit. Between
the CHMP opinion and the granting of the MarketingAuthorisation, the available information on a drug shouldbe gathered in a useable form and additional informationrequirements defined, together with a plan for develop-ment to be undertaken by the Marketing AuthorisationHolder. This will give HTA bodies access to the widestpool of in-use data on a pan-European basis, although theappropriate methodological tools to evaluate orphan drugswill need to be developed.
Proposed resultsIt is intended that the collaboration at European level onthe clinical added value of an orphan medicinal productwill bridge the gap of data generation and availabilitybetween what is needed to reach a positive risk-benefitanalysis and what is needed to facilitate understanding ofthe appropriate positioning of a product in the therapeuticarsenal for a given rare condition within national health-care systems. This should be a voluntary process carriedout on a case-by-case basis. Uptake will be a key measureof success, because use of the system will largely dependon its ability to deliver a more streamlined approach.Implementation could start already where the elementsare in place, while additional required elements are beingdeveloped.
Published: 22 November 2012
doi:10.1186/1750-1172-7-S2-A25Cite this article as: Hughes-Wilson: A coordinated EU approach toinformed access decisions: CAVOD process proposals – the possibilityto turn concept into reality? Orphanet Journal of Rare Diseases 20127(Suppl 2):A25.
Correspondence: [email protected] – Swedish Orphan Biovitrum, EU Committee of Experts on RareDiseases (EUCERD
Hughes-Wilson Orphanet Journal of Rare Diseases 2012, 7(Suppl 2):A25http://www.ojrd.com/content/7/S2/A25
© 2012 Hughes-Wilson; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the CreativeCommons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, andreproduction in any medium, provided the original work is properly cited.
