ClinigenGAP_BioEurope_panelclose_editToday, in partnership with Clinigen Global Access Programs, the BIA has published a post-event report on “Early Access in Practice”. The report is based on a panel session on early access which was chaired by BIA CEO, Steve Bates earlier this month at BIO-Europe. 

Early access is a mechanism that enables patients with an unmet medical need to be provided with access to a medicine, prior to it being made available in that country. The definition sounds simple enough, but successfully navigating the rapidly evolving and highly regulated early access space is challenging. There is increasing pressure from informed, mobilised patients and their physicians as a result in part of more transparency of drug-development pipelines online. This means that pharma and biotech companies need to carefully consider their early access strategy and be prepared before receiving a first request for access to a medicine.

Organised by Clinigen Global Access Programs (GAP), the panel, entitled “Mind the GAP? Meeting an emerging demand for access to medicines in late-stage clinical trials, prior to commercial launch”, explored the practical ways that pharma and biotech can meet this demand for new therapeutic alternatives by enabling pre-launch access to drugs in late-stage clinical trials. Bringing together experts from across the sector, the panel represented the wide range of stakeholders involved in getting unlicensed drugs to patients, and there was a lively discussion of the different challenges and opportunities of early access.

ClinigenGAP_BioEurope_panel_editWhether a top 5 Pharma like Pfizer, or a smaller Biotech like PTC Therapeutics (both represented on the panel), companies can face a range of different regulations for each territory and considerable legal and logistical challenges in providing 24/7 access to a drug over a huge range of territories. Reimbursement can be a key factor for a biotech if their product is still in development and not generating revenue, but is also important for pharma for whom international access programmes can be very costly.

The UK’s Early Access to Medicines Scheme (EAMS), which was explained in a previous report from the BIA and ABPI, is now six months old and the panel evaluated the success of this approach to date and possible elements of the scheme that require review, in particular reimbursement. The scheme is one approach for one country; however it is often part of a wider access strategy encompassing multiple territories and including the cumulative regulatory hurdles, timelines, and crucially costs of different national mechanisms.

Based on their experiences, the panel also provided a number of important considerations that pharma and biotech companies must agree on before deciding to initiate an early access programme. These included; agreement of the objectives for the programme amongst all stakeholders at the outset; a consideration, acceptance and planned mitigation of risk and importantly, an agreed exit strategy.

A fundamental point to consider was whether the structure of a business is amenable to providing 24/7 access to a drug, with a requirement to receive the request and supply the drug to a critically ill patient within a matter of hours. If not, a decision whether to outsource the management and logistics to a specialised service provider should be made.

Mark Corbett, Clinigen GAP

Mark Corbett, Clinigen GAP

The panel was timely as more and more pharma and biotech companies start to receive requests for access to their drugs, in fact Mark Corbett of Clinigen GAP believes every biotech and pharma company whose drugs meet a high degree of unmet need, should have an early access strategy of some form in place as a prerequisite of their drug completing phase II trials.

This awareness of, and increasing commitment to providing new therapeutic alternatives to patients with unmet medical needs was reflected by the large turn-out to the panel and the wealth of questions posed both during the panel and afterwards to the panellists. We hope that companies can take these points into their organisations to ensure that a full and open discussion can take place, and the necessary strategies and mechanisms put in place before a patient request comes through.

Six months on from the launch of EAMS, the BIA would welcome your feedback to ensure that the scheme can maximise the benefit to patients. Please address your comments to Pamela Learmonth, BIA Director of Communications and Public Affairs.

The full report is available to download here.