Pharmaceutical Executive Europe - October 2008 - (Page 19)

Meeting Unmet Needs In this era of greater transparency of drug development pipelines and fingertip access to information about pre-launch drugs, named patient programmes are becoming more prevalent. Natalie Douglas explains how drug companies can use these programmes to help meet patients’ unmet medical needs. Natalie Douglas T oday’s population of patients is more educated and empowered than ever before, and those desperate for potentially life-saving or life-prolonging drugs, are increasingly demanding access to pre-launch medications. Recent, wellpublicised examples of patients taking this kind of action include Pamela Northcott from Wales, who challenged her local trust and the Welsh Assembly government for access to a pre-launched cancer therapy,1 and Connie Loughman, who garnered worldwide attention through a YouTube video she created, advocating access to a pancreatic cancer treatment for herself and fellow patient, actor Patrick Swayze. Governments worldwide have created provisions for granting access to prelaunch drugs for patients who have exhausted all alternative treatment options and do not match clinical trial entry criteria. Often grouped under the labels of compassionate use, expanded access, or named patient supply, these programmes are of course governed by rules that vary by country. Named patient programmes enable physicians, on behalf of patients, to access medicines approved or nearing approval in other countries, in a legal and ehtical way, before marketing approval has been granted in their home country. Regulatory evolution Nearly 20 years ago, a European Union (EU) Council Directive2 laid out the framework for the supply of unregulated medicines in response to unsolicited requests for use by individual patients “on his personal responsibility.” The current legal basis for access to prelaunched medicines in the EU is Article 5 of Directive 2001/83/EC.3 This legislation offered the possibility of pre-launch use as an exception to the rule that medicines must be authorised before use or used within the context of an approved clinical trial. With the expansion of the EU in 2004, Regulation 726/2004 evolved compassionate use programmes in order to better meet the expectations of patients.4 While pre-launch use is permitted by EU legislation, these programmes are governed by the individual member states; each of the 30 member states of the European Economic Area (EEA) has its own nationalised regulations regarding access to pre-launch medicines. Additionally, even after a drug receives approval, authorities in each country can take several months to make reimbursement decisions, which can delay the ability of patients to access these drugs. In a majority of EU member states, an authorisation from a competent authority is required to initiate named patient supply; in other countries it may be retrospective. For example, the French regulatory agency (AFSSAPS) is primarily concerned with the science supporting a patient’s request for access to a prelaunch drug and has in place a proactive approval system. In contrast to the French system, in Ireland, the prime concern is transparency of supply and patient safety, so retrospective notification is required. Despite these differences, the reporting of adverse events is expected in all EU member states (set forth in Article 83). The marketing authorisation holder or manufacturer must report serious adverse reactions (SARs) occurring within the EU to the competent authority of the member state in which the incident occurred. Beyond the EU Many countries outside of the EU offer similar types of pre-launch programmes. 22 Pharma in Turkey Interviews with the country’s leading executives 37 Last words Who said what in pharma’s last four weeks 3 News The monthly round-up of European stories 6 Calendar Upcoming events in Europe and around the world

Table of Contents Feed for the Digital Edition of Pharmaceutical Executive Europe - October 2008

Pharmaceutical Executive Europe - October 2008 Contents News and Analysis Calendar BioFutures Strategy Fundamentals Executive Recruitment: Small Company, Big Career Executive Recruitment: Instant Pharma Named Patient Programmes Executive Review: Turkey

Pharmaceutical Executive Europe - October 2008

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