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Rare Disease Day 2013 Improving patients’ access to orphan medicinal products in Europe based on increased EU coordination « Rare Disease Blogs.

Rare Disease Day 2013 Improving patients’ access to orphan medicinal products in Europe based on increased EU coordination « Rare Disease Blogs.

February 25th, 2013

Rare Disease Day 2013 Improving patients’ access to orphan medicinal products in Europe based on increased EU coordination

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Paris, 21 February 2013 – A press conference to launch the sixth Rare Disease Day focused on discussions underway with the European Commission and other stakeholders to accelerate access to medicines for rare diseases and to streamline national processes for pricing and reimbursement decisions.
Building on the ongoing policy debate on the EU Directive on Transparency, the EU Regulation on Clinical Trials, the recently adopted EUCERD Recommendation on the Clinical Added Value of Orphan Medicinal Products (CAVOMP), the final discussions on the Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA) within the process on Corporate Responsibility in the field of Pharmaceuticals, rare disease patient organisations present at the Paris-based press conference expressed hope that on-going discussions with the European Commission will lead to a value-based pricing approach associated with improved transparency.
It was pointed out that while the Orphan Drug Regulation in Europe has facilitated the centralisation of the designation and authorisation processes for orphan drugs and successfully promoted the development of rare disease treatments many procedures are still conducted and negotiated in isolation at the national level. Upstream, clinical trial authorisation remains a national process, even though rare disease trials are frequently multi-centred, taking place across several countries, with scarce experts, limited knowledge and few patients from each country. Downstream, the processes for price and reimbursement negotiations is decentralised, leading to delayed and unequal patient access to rare disease products across Europe, diverging requirements from national competent authorities and leaving little chance to generate the additional level of evidence needed to address uncertainties at time of marketing authorisation.
A bid was made for more collaboration between the European Medicines Agency (EMA), the Health Technology Assessment (HTA) agencies and the national competent payers. The EUCERD Recommendation on the Information Flow on the Clinical Added Value of Orphan Medicinal Products provides a conceptual framework and four concrete steps to implement it – with a large consensus of 26 out of 27 EU Members States. The EMA has started to involve HTA agencies in numerous pilot discussions at the stage of Protocol Assistance, an important experience in the right direction, which has only been applied once so far to an orphan medicinal product. While some countries are taking the initiative in this area, stakeholders would like to see more. This would allow national agencies evaluating the clinical added value of orphan medicines to cooperate early in the regulatory process, from the time of protocol assistance as well as to coordinate their requirements for post-marketing studies.
A possible mechanism to coordinate pricing and reimbursement processes between EU Member States is already moving forward, with 12 countries participating in the Working Group on the Mechanism of Coordinated Access to Orphan Medicinal Products within the framework of the Process on Corporate Responsibility in the field of Pharmaceuticals(learn more). For a value-based approach to pricing within such a coordinated process, a company planning to introduce a orphan medicinal product would negotiate the price, with the 12 or so participating countries simultaneously, together with medical experts and patient representatives, on the basis of the three following elements: first to evaluate the “value” of the product, based on a commonly agreed “European Transparent Value Framework”; second to define the “volume” of patients in all countries, based on the prevalence of the therapeutic indication as defined in the marketing authorisation; and third to confirm its agreement on the post-marketing research activities defined between the EMA and HTA agencies. This price would not be imposed on countries, as Member States have the exclusive competence in this field and are the end payers from their hospital budget or social security budget. Member States would make their well informed decisions at the national level based on this value, agreed at the European level. With such a mechanism, the EU Member States would be able to better regulate the economic advantage provided at EU level with the 10 to 12 year market exclusivity of the orphan status, companies would get faster market access and better economic visibility, and patients and doctors will get faster access to medicines and better clinical practices based on progressive evidence generation.
In addition, EU Members States should explore the mutual benefit based on fairness and equitability of access of a coordinated approach on differential pricing whereby the agreed price of reference at European level could have a margin of +/- 10% depending on the GNP and population. Pricing would also be subject to fluctuation over time depending on the number of patients to treat and new evidence generated to re-assess the clinical added value, and therefore update both the “value” of the medicine and the “volume” of patients.
Another proposition raised during the press conference concerned the need for changes in the clinical evaluation of medicines for rare diseases. For studies involving very small numbers of patients, grouping Phase I and II studies could be more efficient. Placing products on the marketplace with a Conditional Marketing Authorisation could be considered at the end of Phase II studies. Such conditional licensing, allowing faster access to medicines for patients, would necessitate a carefully planned expansion of prescriptions in the hospital environment based on the adjustment of inclusion criteria, and detailed post-marketing research studies. Conditional marketing authorisation status could then be reviewed annually until confirmation or withdrawal.
Each Rare Disease Day helps to move forward crucial policies and strategies that allow people living with rare diseases to access the care and treatment they need. Rare Disease Day 2013 is continuing this positive trend!

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