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New ISPE White Paper Provides Guidance on Development and Implementation of Regulatory-Compliant Patient Access Programs

New ISPE White Paper Provides Guidance on Development and Implementation of Regulatory-Compliant Patient Access Programs

Panel of industry experts chaired by SVP Clinigen Global Access Programs offers best practices for programs enabling access for patients with unmet medical needs

Burton-on-Trent, UK – 7 August 2014 –  Clinigen Group (‘Clinigen’) (AIM: CLIN), the specialty global pharmaceutical company, announces the publication of a new International Society for Pharmaceutical Engineering (ISPE) white paper developed in collaboration with an interdisciplinary team of industry experts chaired by Mark Corbett, SVP Clinigen Global Access Programs (Clinigen GAP). The white paper describes regulatory-compliant mechanisms that enable access to medicines for patients who have exhausted all other treatment options.

Improving Access for Patients with Unmet Medical Needs – Overview and Best Practices for Success, describes these programs which are known by various names around the world including expanded access, named patient programs and compassionate use, and offers best practices for implementation.

The white paper can be downloaded via the Clinigen Group website1 and provides a sampling of country-specific regulatory guidelines that govern access outside the clinical and commercial framework. In addition, some guidance related to patient eligibility, stakeholder involvement, timing and logistics is included.

“For many patients, access to new medicines through well-known and well-defined clinical trial processes or through traditional commercial channels is not an option,” said Mark Corbett, Senior Vice President, Clinigen GAP. “Access programs can provide a robust and ethical solution, both locally and globally, for relevant patients in need. Unfortunately, low awareness of these programs and confusion as to how and when they are best applied limit their use in the proper circumstances. We believe this white paper will help address these issues and demonstrate the value of these important programs.”

Recent events continue to cast a spotlight on the need for improved access and an understanding of all available options.  A well-publicized plea for access to an anti-viral medication on behalf of a young patient with Adenovirus infection in the US made headlines across a number of days in early 2014 and generated robust debate as to who was responsible for granting access. In May, Colorado, in addition to a number of other states, passed a “right to try” law, giving terminally ill patients access to experimental drugs without approval from the FDA. The UK, in an effort to facilitate access for patients with life-threatening or seriously debilitating conditions introduced a new Early Access to Medicines Scheme earlier this year.

“We have seen recent regulatory changes in what is a continually evolving landscape, highlighting the critical role access programs provide for patients with a high degree of unmet medical need,” noted Corbett. “The more we can do to help educate drug developers, patients and physicians about how these programs work and their proper use and value, the better positioned we are to help those in need in an ethical and compliant manner.”

The report represents the input of industry experts including:

Elizabeth Cooper
Manager, Supply Chain Operations
Delphine Fabry
Project Leader, Clinical Supplies
Mark Corbett
Senior Vice President, Clinigen GAP
Clinigen Group
Rachel Huskisson
Regulatory Affairs Manager
Clinigen Group
Marie DeGayner Kuker
Kuker Regulatory Consulting
Sheri Smith
Courante Oncology








Notes To Editors

About Clinigen Group
The Clinigen Group is a specialty global pharmaceutical company headquartered in the UK, with offices in the US and Japan. The Group, dedicated to delivering ‘the right drug, to the right patient at the right time’, has three operating businesses; Specialty Pharmaceuticals (Clinigen SP), Clinical Trials Supply (Clinigen CTS), and Global Access Programs (Clinigen GAP). Clinigen GAP develops and implements global access programs for biotechnology and pharmaceutical companies and has provided access to unlicenced, licenced and end-of-lifecycle products to thousands of patients. Clinigen has experience in the implementation of more than 30 access programs worldwide. For more information, please visit

About ISPE
ISPE, the International Society for Pharmaceutical Engineering, is the world’s largest not-for-profit association serving its Members through leading scientific, technical and regulatory advancement throughout the pharmaceutical lifecycle. The 20,000 members of ISPE are building solutions in the development and manufacture of safe and effective pharmaceutical and biologic medicines and medical delivery devices in more than 90 countries around the world. Founded in 1980, ISPE has its worldwide headquarters in Tampa, Florida, USA. Visit for more information.


Issued for and on behalf of Clinigen Group by Instinctif Partners.

To contact the Clinigen team at Instinctif Partners, email

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Melanie Toyne-SewellManaging Partner