Plans to give patients with rare diseases access to certain drugs before they have been appraised by the National Institute for Health and Clinical Excellence have received significant backing from all sides, including patient groups, the industry and the National Health Service.
Late last year the government said it was stumping up £25 million to help fund a three-year pilot of the Innovation Pass scheme, under which patients with rare diseases will get access to certain licensed innovative medicines on the NHS before they are assessed by the cost watchdog, allowing manufacturers to garner sufficient evidence – often a challenge with small patient populations – to support their use before a cost evaluation.
In addition, proposals were announced for the creation of a new expert advisory group to help strengthen and expand the current process for the national commissioning of services for extremely rare conditions, enabling more technologies that may not be appropriate for assessment by NICE to be considered and thereby reach more patients.
Now, results from two separate public consultations over the initiatives, which the DH says are both intended to give patients with very rare conditions access to a wider range of drugs and services on the NHS, have revealed widespread backing for the plans.
Health minister Mike O’Brien said he is “extremely pleased to see support for the proposals” that will make “a real difference” to patients suffering from rarer diseases.
However, while noting that they could indeed “potentially” improve access to treatments for people with rarer cancers, charity Macmillan Cancer Support insists the plans are only “a small part” of the solution. “NICE needs to be more flexible in its approach to evaluating drugs for rarer cancers and that drugs for very rare cancers should automatically be assessed by national specialised commissioning,” it said.
