UK Regulator Issues First Positive Opinion in Early Access Program

Merck’s new skin cancer treatment has become the first product approved under a new accelerated review process by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA).

The product, Keytruda, has been shown to be effective in slowing the progression of advanced melanoma, a deadly form of skin cancer with few treatment options.

Accelerated Approval

Keytruda is the first product to be approved under MHRA’s early access to medicines scheme (EAMS), which was created to provide faster access to new products designed to treat “life-threatening or seriously debilitating conditions … where there is a clear unmet medical need.” The drug was also approved in the US in September 2014 after receiving breakthrough therapy designation.

Merck says Keytruda was reviewed based on the data from its Phase Ib study on anti-programmed cell death 1 therapy. In its assessment, MHRA found the product to be effective in patients who had been previously treated with ipilimumab, saying “approximately 60% of [previously treated] patients survived for at least one year.” The agency also found the side effects of the product to be “mild to moderate in severity,” but noted that there were still uncertainties about the product’s effects.

About EAMS

MHRA announced EAMS in March 2014, saying it would allow earlier access to potentially lifesaving medicines for patients with severe or life-threatening conditions.

To qualify for EAMS, companies must be granted a promising innovative medicine (PIM) designation. PIM designation is awarded based on early (Phase I and/or II) clinical data, and three criteria:

1. “The condition should be:
• Life-threatening or
• Seriously debilitating
2. With High Unmet need:
• There is no method of treatment, diagnosis or prevention available or
• Existing methods have serious limitations
3. The potential adverse effects … are outweighed by the benefits.”

Companies are also encouraged to schedule a pre-submission meeting with MHRA before applying for review under EAMS. The EAMS opinion will provide information to prescribers and patients, and allow for the use of a product before it is licensed for broader use. MHRA requires companies to provide regular updates

EAMS opinions are valid for one year, and may be renewed by the company based on the product’s use and performance. Companies must also provide regular reports to MHRA documenting patient exposure, adverse effects and other additional data collected by the company. Additionally, companies can use the reporting form to propose changes to treatment protocols and the terms of the EAMS opinion based on new data.

EAMS Public Assessment Report