18th EURORDIS Round Table of Companies workshop looks at corporate responsibility and innovative policies for improving access to orphan medicinal productsApril 2013
The EURORDIS Round Table of Companies (ERTC) met on 27 February in Brussels to debate Corporate Responsibility in Improving Access to Orphan Medicinal Products in the context of the current economic situation across Europe, taking into consideration the national plans for rare diseases that EU Member States are urged to develop by the end of 2013.
The ERTC offers its members a unique opportunity to interact with other biopharmaceutical companies, as well as leading rare disease patient advocates and scientists, and national or European health authorities. Workshops every six months enhance relationships between these relevant stakeholders to accelerate the development and availability of treatments in Europe. This latest ERTC workshop gathered more than 80 participants from 11 countries – the EU, the USA and Canada.
Thomas Heynisch, (European Commission, DG Enterprise) presented the progress on creating the Mechanism of Coordinated Access to Orphan Products (MoCA) as part of the European Platform on Access to Medicines in Europe – a two year policy discussion process in which EURORDIS represents patients. The MoCA process seeks to provide affordable and sustainable patients’ access to orphan medicines. The essence of MoCA is to create a framework for dialogue between payers from EU Member States involving medical experts, patient representatives and the Marketing Authorisation Holder, building on the EU regulatory process and the anticipated growing interfacing between the European Medicines Agency (EMA) and Health Technology Assessment (HTA) in the field of orphan medicines. Mr Heynisch emphasised that the patients’ organisations had and will continue to have a role to play by convincing their Member States to become proactive in this process.
As countries determine their national rare disease strategies, the workshop looked at how initiatives shaped at EU level between Member States and with participation from other stakeholders could dovetail with the national measures needed to improve access to orphan medicinal products. Which measures can be identified and promoted to improve access? The European Transparent Value Framework developed by MoCA will enable payers from EU Member States to determine a Common Assessment of the Value of an Orphan Medicine Product to support well-informed national pricing decisions, thus already reducing delays and duplication. Experience garnered would build trust between involved parties and help identify what each party would gain in future coordinated price negotiations based on this common value assessment, aggregated volume of patients from volunteering EU Member States, and post-marketing research activities generating additional evidence that reduces uncertainties and identifies the appropriate place of a product in the therapeutic strategy. It was highlighted that the Clinical Added Value of Orphan Medicinal Products (CAVOMP) and the MoCA complement each other, proposing a continuum of approaches aiming at an informed and robust decision-making process while improving patient access.
Johan van Calster, (former Head of Belgian Medicines Agency) reported on discussions in which EURORDIS is closely involved on Differential Pricing as an additional and potentially important policy innovation to improve access to medicines. Differential Pricing refers to the strategy of selling the same medicinal product at different prices to different markets, here based upon a country’s GDP and disease prevalence. Differential Pricing has improved access to medicines in developing countries. The benefits and risks of its use inside the EU for orphan medicines were considered and it was pointed out that External Reference Pricing is not compatible with Differential Pricing and could diminish company willingness to participate. Parallel Trade was not felt to be a current or potential issue for orphan products. A call was made for transparent Market Entry Plan/ Market Entry Agreement with a commitment of participating Member States to apply Differential Pricing principles and a commitment of companies to launch medicinal products within two years of receiving a marketing authorisation.
European Federation of Pharmaceutical Industry Association Director General Richard Bergström discussed the industry perspective on Differential Pricing by raising key issues, such as processes needed to ensure a common European political and legal framework that keep pricing decisions at the Member State (or even regional) level and ensure industry willingness to participate. Differential Pricing was evoked as an important medium/long-term objective to facilitate orphan drug access, which should remain voluntary, requiring no legislative change. Net price confidentiality would be a central part of a differentiated pricing approach and payers must be engaged early in the process.
Throughout the daylong workshop, the interaction between CAVOMP, MoCA, Market Entry Agreement, and Differential Pricing as a set of innovative policies to improve access to orphan medicinal products became increasingly understood and supported. Pilots are needed for these innovative approaches to gather more experience and understanding.
In the afternoon, the impact of the economic crisis on medicine access in the EU countries was debated. A panel discussion focused on the situation in Greece and other countries and the factors that are aggravating the situation.
The 18th Workshop of the ERTC successfully moved forward understanding of the complex issues involved in the process toward more equitable and rapid medicinal product access for people living with a rare disease across Europe.
Author: Louise Taylor, Communications and Development Writer, EURORDIS