Print“The scheme is intended to enable patient access to medicines for treatment of life threatening or seriously debilitating conditions where there is an unmet need, says Ian Hudson, CEO of the Medicines and Healthcare Products Regulatory Agency.”
After much debate, the UK’s drug regulatory agency will launch the Early Access to Medicines Scheme in April, its version of the U.S. Food and Drug Administration’s Breakthrough Therapy designation. The program aims to give patients with life threatening or seriously debilitating conditions access to medicines that have not yet received marketing approval.
“The scheme is intended to enable patient access to medicines for treatment of life threatening or seriously debilitating conditions where there is an unmet need,” says Ian Hudson, CEO of the Medicines and Healthcare Products Regulatory Agency, or MHRA. “This is a major new development in medicines policy in the UK.”
It offers a way for promising experimental drugs to be made available to patients before approval to benefit public health, Hudson says, and enables companies to gain additional knowledge and experience of these medicines in clinical use.
The program will allow limited access to products in mid- to late-stage testing for conditions that have limited or no treatment options. MHRA will give a scientific opinion on a new medicine or indication that has demonstrated a positive risk/benefit ratio. Medicines that qualify will be designated as a promising innovative medicine, or PIM, by the agency, based on early clinical data that could be given several years before the product is approved.
Medicines eligible for the Early Access to Medicines Scheme must have the PIM designation. Like the FDA’s breakthrough therapy designation, companies developing products designated as promising innovative medicines will have access to advice from MHRA and NICE during development.
The BioIndustry Association, the UK biopharmaceutical trade group, welcomes the new scheme for its “all hands on deck approach” to speedily progress promising innovative therapies to the patients that need them. But it has reservations about the fact that companies must foot the bill for all medicines dispensed under the scheme.
“As it is currently envisaged, without centrally funded reimbursement, the scheme runs the risk of being under-utilized,” says Steve Bates, CEO of the BioIndustry Association. “The BIA remains committed to working in partnership with all stakeholders to ensure that this is not the case and the scheme remains globally competitive.”
Bates says that for the program to work so that patients receive breakthrough therapies while industry objectives are met, “companies will need to be reimbursed at an earlier stage in development at a price that recognizes the uncertainty of the effectiveness of early stage products.”
March 14, 2014
http://www.burrillreport.com/article-uk_to_launch_early_access_to_medicines.html
