fast tracking rare disease patient access to orphan drugs under eams

Orphan Drugs and the UK Early Access to Medicine Scheme

In Market Access, Regulation & Government by Caroline Hornby

On April 7th 2014 the UK Early Access to Medicine Scheme was launched, changing the playing field for fast tracking orphan drugs to patients.

As defined by the Medicines and Healthcare products Regulatory Agency, the EAMS “aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need.” Under this voluntary new scheme, the MHRA will undertake a benefit/risk analysis of the medicine, and, when appropriate, grant access to the medicine before it has received licensing approval. This process will not replace normal licensing procedures, but it will speed up patient access to vital treatment. As reported by The Telegraph, the UK “Government envisages that a new drug could be given the green light for use with specific patient groups – subject to their consent and doctor approval – as soon as the data show safety and benefit advantages to the target users. This could bring drugs to patients anything from two to 10 years earlier than would otherwise be the case.”

In regards orphan drugs, the key part of the above MHRA definition is the phrase “clear unmet medical need.” Rare diseases are often debilitating by nature so there is certainly an obvious medical need. Furthermore, rare diseases often don’t have a huge list of possible treatments and therapeutics, so this medical need in many cases remains unmet. This means that under the EAMS rare disease patients may be able to cut down the wait time and access new medicines with the potential to treat their diseases before these medicines go through lengthy licensing procedures.

Early examples of this may include treatments for the rare muscle disorder, Duchenne Muscular Dystrophy. Glyn Edwards, chief executive of Summit plc, a UK pharmaceutical company currently developing treatments for DMD, has told The Telegraph that it would be “first in line” to apply for the early access scheme. Already in Phase 1b, early access for this drug seems possible, and would certainly give patients much faster access than usual if granted by the MHRA.

As well as vastly improving the wait time for patients, the EAMS will also impact the economics of orphan drug development. Understanding this scheme is integral to the future success of companies who are developing orphan drugs. For more information on the EAMS and other fast tracking schemes, join key opinion leaders in London on the 10th of July at the Fast Tracking Orphan Drugs Briefing.

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