Therapeutics


MAKING EARLY ACCESS REAL


getting treatments to patients with no other option


For patients who have no other option to treat their condition, early access to drugs in clinical development can be critical. As a trained physician, there is an intrinsic understanding that early access to investigational or pre-launch drugs can potentially offer life-saving treatment options when no other option is available. The challenge for the pharmaceutical industry is to successfully and ethically manage early access to their drugs.


P


atients with unmet medical needs can gain access to drugs in clinical development through ethical and regulated managed access programmes known as Expanded Access Programs (EAP) in the US and Named Patient Programmes (NPPs) in the rest of the world. The growing focus on controlled access to pre-approved therapies is a reflection of patients’, physicians’ and the industry’s increased awareness of the important role that pharmaceutical companies can play in the management of access to their medicines.


The role of managed access programmes Managed access programmes facilitate access to investigational drugs for individual patients or groups of patients with unmet medical needs. These pro- grammes also allow access, in specific circumstances, to drugs that are approved in other countries but not yet approved in a patient’s home country pending ongoing pricing and reimbursement negotiations. In Europe, NPPs are controlled and restricted access programmes set up by pharmaceutical and biotechnology companies under which physicians


Drug Discovery World Winter 2010/11


and pharmacists can gain access to investigational therapies on a ‘named’ patient basis. At the same time, they also offer physicians the opportunity to utilise new medicines and stay at the forefront of medical advances. For pharmaceutical and biotechnology companies, NPPs can help address demand and build a network of physicians and knowledge about who, where, and in which patient populations the drug can be used effective- ly. There are limitations on data that can be col- lected during an NPP such as efficacy data. This understanding of the impact of a new drug can enable more informed strategic decisions and feed into pre-launch plans that can be shared with- in a company and may indeed help influence future marketing and reimbursement strategy.


Facilitating access in a range of scenarios and different markets There are several EU Directives that apply to man- aged access programmes. They provide a set of rules under which patients can receive investiga- tional or pre-launch drugs:


55 By Dr Rav Seeruthun


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