Edition of 16 February 2006

The European Platform for Patients’ Organisations, Science and Industry (EPPOSI) has released key outcomes and recommendations from its Sixth Workshop on Partnering for Rare Disease Therapy Development, held in London in late October 2005.

Amongst the conclusions reached, consensus building between stakeholders figured as being vital in order to foster understanding of each player’s specific needs. Concrete examples of this principle include understanding patients’ medical needs and the degree of risk patients are willing to accept; appreciating the challenges industry faces in developing innovation; ensuring equal access to screening and diagnostic services; blending epidemiological data gathering and information with demonstration of treatment safety and efficacy in order to create a better understanding of benefits; and harmonising treatment review and approval requirements amongst regulators.

Other key points mentioned include trust between all players, which could be fostered by increased communication. Partnerships were named as key to addressing specific issues impacting the research, manufacturing and distribution of treatments and ensuring that patients get proper access. Specifically, it was decided that patients must play an active role in identifying their treatment needs and taking part in risk versus benefits decisions. They should also have input in decisions involving health economics. Industry needs to take advantage of opportunities to develop rare disease products by sharing potential product candidates with third parties, exchanging expertise with other companies, and possible new clinical trial designs. Research needs to support academia and collaborate internationally; networking must be encouraged.

Regulators must find ways to harmonise clinical trial requirements in order to overcome the challenges of small populations, such as the agreement made by the EMEA and FDA on a common approach to annual reporting for orphan drugs. Biomarkers and surrogate end-points are other issues upon which regulators must come together.

Diagnostic availability for very rare diseases needs a framework that will ensure equity, perhaps modelled on the US network of public laboratories established to continue maintaining technology and expertise initially developed by research groups.

Other key outcomes stress the pivotal role of patients; the need for further awareness to stimulate research efforts, perhaps via a specific commitment to orphan drugs in each EU member state; the lack of formal authorisation for treatments for children which is being addressed by specific legislation; cultivating a truly global focus emphasising cooperation across borders in partnership with international organisations such as the WHO; and treatments for patients in developing countries via a shared responsibility approach involving manufacturers. Twinning research and clinical departments between developed and developing countries would benefit patients in poorer countries. Finally, sustained research funding was identified as an ongoing issue needing coordination internationally as well as by individual countries. A full report of the recommendations is available.

The European Medicines Agency (EMEA) has started to produce simplified summaries of their European public assessment reports (EPARs) written in a shorter, non-technical manner especially designed for patients and members of the general public. Containing descriptions of how the treatment functions, its indications, benefits and risks, and why it was accorded a positive authorisation recommendation by the EMEA, the simplified EPARs are part of a series of new provisions of the revised EU pharmaceutical legislation on the availability of improved medicine information. Written in a question and answer format, the summaries will only be published for newly authorised medicines initially, but eventually will be made available retroactively for all centrally authorised products on the market.