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Patients with rare conditions face postcode lottery

Research shows that patients with rare conditions have less chance of accessing 'orphan' medication if they live in England

Seriously ill patients with life-threatening rare diseases are being denied vital drugs because of a postcode lottery across the NHS that campaigners say is frustrating and unfair.

New research reveals that patients with a rare condition have much less chance of accessing "orphan" medication if they live in England rather than Scotland or Wales. "Orphan" drugs treat patients with a condition affecting fewer than five in 10,000 people.

While the European Medicines Agency (EMA) has approved 68 drugs for use with patients with rare conditions such as unusual forms of cancer and epilepsy since 2000, the National Institute for Health and Clinical Excellence (Nice) has assessed only 18 of them, recommending that seven become available on the NHS in England and Wales, and another five under certain restrictions.

In contrast Nice's equivalents in Scotland and Wales have made 28 and 21 respectively available in some way.The Scottish Medicines Consortium (SMC), has examined 56 of the 68 drugs and made 14 fully available and 14 more in some circumstances.

Similarly, the All Wales Medicine Strategy Group (AWMSG) has looked at 51 and made 12 available and nine others in some cases.

The figures are contained in a new report by the think-tank 20:20 Health, which claims the disparity between the Celtic countries and England shows the NHS is providing an unequal service.

"There's inconsistency in access across the UK, with different assessment bodies looking at different orphan drugs so some are being recommended and some not. In terms of approval of drugs, it's worse in England by a long way", said Julia Manning, the think-tank's chief executive.

"Access to life-saving drugs should not be determined by your postcode. The NHS should work for people living in all parts of the UK. There's not a level playing field; there's a postcode lottery.

"These are seriously ill people with a rare disease for whom these drugs are possibly the only treatment on the market", added Manning.

The drugs, which can cost up to £40,000 for a course of treatment, treat conditions such as chronic myeloid leukaemia; a rare form of epilepsy called severe myoclonic epilepsy, which affects infants; renal cell carcinoma, a rare form of lung cancer; and multiple myeloma, a blood cancer.

Nice insisted the low number of such drugs it had examined was due to a lack of referrals from the Department of Health, as it can only assess the cost effectiveness of medications which ministers there ask them to look at.

But the DoH denied patients with rare conditions were missing out and insisted that the absence of a Nice appraisal should not stop them getting what they need. "There is no evidence of a 'postcode lottery' in access to these drugs", said a spokesman.

"The NHS must not refuse funding for a drug because of the lack of a Nice appraisal. The NHS Constitution sets out patients' rights to expect rational local funding decisions on drugs where there is no Nice guidance", he added.