The Ministerial Industry Strategy Group has called for “a programme of work” to explore the possibility of developing a regime that would help speed up access to innovative therapies in the UK.

Since its creation in 2001, The MISG - a high-level group bringing together government and pharmaceutical industry representatives - has been meeting twice a year to discuss strategic issues regarding the industry and healthcare in the country.

Last September, it organised a Forum to mull over whether making medicines available to patients at an earlier stage of their development, such as at the end of Phase II clinical testing, as suggested by Sir David Cooksey in his Review of UK Health Research Funding, is “feasible and desirable”.

Sir Cooksey’s report recommended the clinical use of unapproved drugs under strict controls, as well as a programme of pilot studies of conditional licensing for new drugs at an earlier stage to allow their use by specialists but not GPs.

The MISG Forum, which brought together stakeholders from all sides of the industry, considered a number of possible scenarios under which patients could be allowed access to as yet unapproved medicines, including:

- Expanding existing European Union conditional licensing laws to include less serious diseases representing unmet medical and/or health needs, as well as “exceptionally promising” candidate drugs;

- A two-step “staged” approval process, allowing access to new products provisionally approved on the basis of clinical data derived from studies other than large-scale clinical trials, with subsequent monitoring of patients in a “real world” setting; and

- A compassionate use system allowing reimbursed managed access a la the French Autorisations Temporaires d’Utilisation (ATU) system, introduced in 1994 to provide early access to new promising treatments where a genuine public health need exists.

The Forum concluded that earlier access would indeed benefit patients, but only under certain conditions. For example, it was stressed that any early access programme should only be implemented where there an unmet medical need is identified, and if there is “clear evidence” of efficacy and confidence – as far as possible - about safety, “with a good system for collecting evidence afterwards”.

Cost, benefit and liability
Furthermore, it pointed out that earlier discussions between sponsors and health technology assessment agencies on which cost/benefit measures to include in clinical trials will be crucial to marking sure that treatments are “appropriately valued for early access”, and that “reimbursement systems should assist, and not frustrate, the concept of earlier access”.

It also underscored the importance of determining just what constitutes “informed consent” and how to ensure that patients get all the necessary information, including any uncertainties surrounding the drug. In line with this, the problem of litigation and who could be considered liable if a product was found to be harmful needs to be addressed, it said.

The group concluded that a programme of work should be commissioned to “explore the possibility of developing a regime that would provide earlier access to medicines under certain circumstances in the UK”, paying particular attention to issues such as: ensuring patient safety and availability of a robust system for data; arrangements for reimbursement and timing of health technology assessment; and ensuring a UK initiative remained compatible with current EU provisions.

"Any proposal that could bring medicines more quickly to patients who need
them is worthy of serious consideration," a spokesperson for the Association of the British Pharmaceutical Industry told PharmaTimes UK News.

"This would be particularly valuable in medical conditions that have a high unmet clinical need, where availability of new treatments is even more crucial for patients. However, the ABPI would echo the paramount need for patient safety as a top priority," he added.