Therapeutics


Figure 1: What do you need to consider?


l The Directive 2001/83/EC states that an unli- censed drug may be made available in response to a bona fide unsolicited order by a healthcare pro- fessional for use by their individual patient in their direct personal responsibility. l The EU Directive 726/2004 outlines the type of patient who can be considered for these pro- grammes based on compassionate use grounds. According to the Directive, unlicensed drugs can only be made available on compassionate grounds to patients with a chronically or seriously debili- tating disease or whose disease is considered to be life-threatening and who cannot be treated satis- factorily with an authorised medicinal product.


The latter regulation also underlines that man- aged access programmes may not interfere with or replace clinical trials, ie patients should always be considered for inclusion into a clinical trial first. In addition to this general framework laid down on a European level, the 27 member states of the European Union – all of whom nominally come under the jurisdiction of the European Medicines Agency (EMA) – each have their own nationalised regulations regarding the import of investigational or pre-launch medications for NPPs. National pro- grammes differ widely from one member state to another due to differences in national medical practices, resources available, funding of the prod- uct, hospital structures and national insurance sys- tems. The terminology also differs, but broadly


56


speaking, access is created via named patient pro- grammes.


Implementing managed access programmes


Putting into place a managed access programme can often be a complicated process (see Figure 1). My experience has involved implementing three NPPs, including one for a drug with orphan status from the EMA and the US Food & Drug Administration (FDA) for Lennox Gastaut Syndrome. The programme was implemented both on a pan-European and domestic level during reim- bursement discussions in a number of the EU27 member states.


Orphan drugs do provide an interesting chal- lenge to many companies. To be designated an orphan drug in the EU, the condition treated needs to occur in less than five per 10,000 patients in the European Union. This differs from the United States where less than 200,000 patients need to have been diagnosed with the condition. As these conditions are rare the numbers of patients included in the pre-licence trials are often limited. The orphan drug legislation creates an incentive for pharmaceutical companies to develop drugs that due to the rareness of the conditions may have limited marketing gain. Often these conditions such as Lennox-Gastaut Syndrome are catastrophic in their nature. Therefore an early access pro- gramme is an ethical approach to supplying orphan


Drug Discovery World Winter 2010/11


Page 1  |  Page 2  |  Page 3  |  Page 4  |  Page 5  |  Page 6  |  Page 7  |  Page 8  |  Page 9  |  Page 10  |  Page 11  |  Page 12  |  Page 13  |  Page 14  |  Page 15  |  Page 16  |  Page 17  |  Page 18  |  Page 19  |  Page 20  |  Page 21  |  Page 22  |  Page 23  |  Page 24  |  Page 25  |  Page 26  |  Page 27  |  Page 28  |  Page 29  |  Page 30  |  Page 31  |  Page 32  |  Page 33  |  Page 34  |  Page 35  |  Page 36  |  Page 37  |  Page 38  |  Page 39  |  Page 40  |  Page 41  |  Page 42  |  Page 43  |  Page 44  |  Page 45  |  Page 46  |  Page 47  |  Page 48  |  Page 49  |  Page 50  |  Page 51  |  Page 52  |  Page 53  |  Page 54  |  Page 55  |  Page 56  |  Page 57  |  Page 58  |  Page 59  |  Page 60  |  Page 61  |  Page 62  |  Page 63  |  Page 64  |  Page 65  |  Page 66  |  Page 67  |  Page 68  |  Page 69  |  Page 70  |  Page 71  |  Page 72  |  Page 73  |  Page 74  |  Page 75  |  Page 76  |  Page 77  |  Page 78  |  Page 79  |  Page 80