Therapeutics


One should consider that if the drug is charged for then this price can be used as a benchmark for pric- ing and reimbursement committees. Ultimately, the choice to offer early access – or not – is left up to the drug developer. Existing regu- lations do not force companies to offer access to drugs prior to approval or launch. When consider- ing this option, however, companies must under- take a thorough evaluation of important questions such as when to offer access and for which patients.


Ensuring supply and vetting healthcare professionals Companies must ensure that there is adequate sup- ply of the drug to complete registration studies and support the access programme in parallel. Another consideration is the resource needed to establish and run the programme, including processes for han- dling and vetting requests, the mechanisms to review physicians requesting the drug and procedures to handle adverse event reporting. Another key factor is proper vetting of physicians and pharmacists.


Taking responsibility


Liability risks are an important consideration and in most instances rest upon the treating physician. In Germany, Greece and Sweden, however, liability resides with the company. Both France and the UK distinguish between responsibility for quality aspects; this is the responsibility of the company; and clinical negligence for which the physician is responsible. Importantly, adverse event reporting is mandatory in all member states. The Czech Republic, Denmark, Finland, France, Greece, Latvia, Luxembourg, Malta, the Netherlands and Sweden have national regulations including both named and cohort programmes.


Working in partnership


Implementation and management of an access pro- gramme requires significant knowledge, experience and resources. In some cases, pharmaceutical com- panies often don’t have the specific experience in this highly regulated environment or the dedicated resources for these types of programmes, particu- larly when managing small quantities in a number of different markets. In addition, it is rare for there to be one stakeholder within a company who ‘owns’ this function.


Partnering with a strategic consultancy is crucial as they can implement these programmes for phar- maceutical companies. When selecting a company to work with, it is essential to verify that they have the expertise, experience, systems and policies in place that will provide absolute peace of mind that the programme is devised and managed effectively


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and within local regulatory guidelines. Based on past experience, working with a global specialist consultancy such as Idis, which has more than 20 years’ experience within this field, meant that the programmes were robustly implemented taking into consideration national regulatory nuances.


New horizon: embracing the need for managed access


As the pharmaceutical industry is rapidly chang- ing, it is becoming more and more likely that drug developers will find themselves on the receiving end of requests for access to drugs in the pipeline. Publicity generated by the release of updated ‘expanded access’ regulations by the FDA in 2009 created greater awareness of these programmes. At the same time, the increased transparency of clini- cal research and accessibility of powerful social media tools have led to a more informed and vocal population of patients.


For companies with drugs in development that may generate early demand, managed access pro- grammes offer a way to meet the needs of patients on a global basis in a regulated; ethical and trans- parent manner. Managed access programmes pro- vide access for patients who otherwise would not be able to participate in company-sponsored clini- cal trials and allow sponsors to engage with physi- cians, providing early, hands-on experience to potentially life-saving medicines.


For patients with life-threatening illnesses, licence approval or commercial launch of an innovative new drug may come too late. In some cases, such as those involving the EU registration and approval process, a drug may not clear reimbursement hur- dles in an individual country until well after cen- tralised approval is granted. In geographies where a formal launch is not feasible, a patient may not have any other opportunity to receive the drug other than through such a programme. In all of these scenarios, managed access pro- grammes provide an effective route to innovative drugs prior to approval or launch, potentially pro- viding patients who have run out of therapeutic options with an important lifeline.


DDW


Dr Rav Seeruthun is a member of the faculty of pharmaceutical physicians and a General Practitioner. He is currently Head of Clinical Affairs at Napp Pharmaceuticals, and previously was Head of CNS and GI at Eisai Europe where he ran a number of named patient programmes. He trained at St Mary’s Hospital Medical School at Imperial College and has a BSc (Hons) in Psychology from University College London.


Drug Discovery World Winter 2010/11


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