Clinigen_debbie AingeEarlier this month, the International Society for Pharmaceutical Engineering (ISPE) launched a white paper offering guidance on the development and implementation of regulatory-compliant patient access programmes. Dr Debra Ainge, Head of Project Management at Clinigen Global Access Programs (Clinigen GAP), whose colleague  Mark Corbett chaired the interdisciplinary team behind the paper, outlines the rapidly evolving access programme space and the key groups involved in the supply of unlicensed medicine to patients with unmet medical needs.

Providing access to investigational products can be a challenging business for Pharma and Biotech companies.

The treatment of two Ebola victims with an experimental medicine in the US this month and the huge social media campaign surrounding a well-publicised plea for access to an anti-viral medication on behalf of a young patient with Adenovirus infection in the US has also raised awareness of the need for better understanding of access programmes across both industry and wider society.

An access programme is often needed most when a company is busy focussing on clinical trials and the approval, launch and commercialisation of a product when time, resources and the ability to scale up supply of the product is limited.  These programmes can also be extremely complex to set up, and many companies both large and small lack the infrastructure and experience to deliver the medicine.

Planning early is key, but companies can sometimes struggle to gain internal consensus on how a programme should be run. Education and the development of clear policies and procedures to ensure readiness to respond to requests in an ethical and compliant manner can help overcome this, and is an area where Clinigen GAP is frequently asked to provide support.

Early access – strategies for a successful programme

The white paper Improving Access to Patients with Unmet Medical Needs – Overview and Best Practices for Success, describes these regulatory-compliant mechanisms that enable Pharma and Biotech companies to provide access to investigational or unlicensed medicines to patients who have exhausted all other treatment options.

The paper looks at country-specific regulatory guidelines on access to medicines outside the clinical and commercial framework, patient eligibility guidance, stakeholder involvement, timing and logistics. The majority of countries have mechanisms in place to allow access to these medicines for those with serious of life-threatening conditions where no licensed alternatives are available; it is the navigation of these regulatory systems that can be the challenge.

Mark Corbett, who heads Clinigen GAP speaks regularly on these mechanisms and strategies for delivering successful early access programmes. He will present the findings of the white paper at the ISPE Annual Meeting and will speak at the BIA’s UK Bioscience Forum on early access to unlicensed drugs in October.

In November, Clinigen GAP will host a panel at BIO-Europe in Frankfurt, chaired by the BIA’s Steve Bates and featuring Luc Dochez, Chief Business Officer at Prosensa and Pfizer’s Lead Oncology Europe, Africa and the Middle East, Dr Markus Kosch, entitled ‘Mind the GAP? Meeting an emerging demand for access to medicines in late-stage clinical trials, prior to commercial launch’. The panel will explore the industry environment, regulatory environment with a focus on the new UK Early Access to Medicines Scheme (EAMS).