Report warns of UK's rare disease 'postcode lottery'

There is still a ‘postcode lottery’ for patients getting access to vital rare disease treatments in the UK, according to a new report.

The new report, ‘Leaving no-one behind: Improving access to medicines for rare diseases patients’, from Roche, IQVIA and Public Policy Projects, highlights key issues facing the access system in England.

Produced in conjunction with a panel of rare disease policy experts, the report calls for reform so people with rare diseases are able to access new treatments no matter where they live in the UK.

The report points to a fragmented decision making process with gaps, inconsistencies, and poor transparency, causing delays in patient access.

It noted that four separate decisions are currently being made on the same treatment across the UK, with not all of them reaching the same conclusion, leading to inequality of access.

In England, NICE’s Highly Specialised Technology appraisal aims to look at only three very rare disease medicines each year.

This restriction on appraisals, and the opaqueness of the process where they are chosen, means the current process will not be able to cope as the number of emerging therapies continues to increase.

The number of licensed orphan drugs approved in Europe is predicted to grow to 75 in 2020, compared with 19 in 2016, according to the report.

Under existing arrangements where very rare disease drugs cost below £100,000 per Quality Adjusted Life Year, these are automatically reimbursed.

Above that there is a new range of up to £300,000 per QALY, which is acceptable to NICE if therapies extend patients’ lives by 30 quality years or more.

However, the report said that no drug will be likely to match this criterion – although authors noted that seven very rare disease drugs are recommended for funding in England so far, such as GlaxoSmithKline’s Strimvelis for the immune disease “bubble boy” syndrome, which is to be sold off to Orchard Therapeutics.

Report authors recommended greater flexibility in assessing medicines, to include wider improvements to patients’ lives and those of their families.

Evaluation should be aligned across the UK, and the patients’ voice should be included in the process.

The government should also produce a full impact assessment of Brexit for the rare disease community, according to report authors.

Rav Seeruthun

Dr Rav Seeruthun, Roche’s UK Medical Director, said: “Looking to the future we are keen to see reform to the system to ensure people with rare diseases across the country are able to access medicines which can improve the lives of patients and their families. There is a welcome focus from Government on ensuring the UK is a global leader in life sciences post-Brexit, as reflected in the agenda set out in the Life Sciences Industrial Strategy and Sector Deal. Improving rare diseases outcomes needs to be a key part of this agenda.”