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Encouraging the collection of data even after a drug is on the market is wise, and the key to doing so is flexibility. Examples in practice are the two Managed Access Agreements in place to allow patients in England with life-threatening rare diseases to access drugs while more evidence is gathered on their efficacy. This includes one for the first muscular dystrophy treatment, Translarna.
We need to more flexibility like this, as well as innovative risk-sharing agreements and variable pricing structures – an opposite direction of travel to the recent introduction of drug pricing and QALY caps. The charity Muscular Dystrophy UK has launched its Fast Track campaign to encourage this approach and to address some of the many delays and barriers that stop patients with rare genetic conditions, including muscular dystrophy, from accessing high-cost personalised treatments. A more nuanced approval process and savvier commercial negotiations with drug companies will help to ensure treatment gets to patients at an affordable price to the NHS.
No competing interests
10 October 2017
Clinical Lead and Consultant Neurologist, National Hospital for Neurology and Neurosurgery, Queen Square