Regulator criticised for 'unbelievably arrogant' approval process

Two charities representing patients with rare medical conditions criticised the national body’s process for evaluating new drugs, with one claiming it had to “fight every step of the way”.

NHS England’s clinical priorities advisory group advises the regulator on which specialist services and treatments should be prioritised for funding. The final decision is then made by a specialised commissioning oversight group.

Minutes from December’s meeting of the all-party parliamentary group on rare, genetic and undiagnosed conditions revealed discussions around the approval process for the drug Selexipag, which has been developed for people with pulmonary arterial hypertension.

Gerry Coghlan, a consultant at the Royal Free London Foundation Trust, told the committee that “evidence provided by patients… was excluded” from the CPAG process. It is unclear why and NHSE has refuted this claim.

A London-based doctor involved in treating tuberous sclerosis complex, who asked not to be named, also told HSJ that patient evidence had not been considered during the assessment process for using the drug Everolimus to treat the condition.

Both drugs have since been approved for funding.

Since these concerns were raised NHSE has piloted a new process, called a “lived experience template”, to standardise the way it engages with patient charities during a drug appraisal.

Susan Walsh, the head of the charity Primary Immunodeficiency UK, was one of the first to complete the template and said it was “unbelievably arrogant” of NHSE to give her just 20 working days to do so.

She said: “What teeth do these reports have? [NHSE] said it will not change the decision-making methodology and indicated they will be quality controlled when submitted. [There] was no assurance it would be read. We don’t know what happens in that process. It is very opaque.

I had to use my wages paid for by the charity to fill out this form and we are operating on less than one full-time equivalent. I don’t think our situation is unique”.

Iain Armstrong, chair of the Pulmonary Hypertension Association, also criticised the process on similar grounds.

He told HSJ: “[Engaging with NHSE was] painful and raised quite concerning issues around openness and transparency, not only through a lack of interest but also about building a massive barrier around patients. We had to fight every step of the way to get patients heard.”

NHSE told HSJ that PHA is part funded by the manufacturer of Selexipag. The charity mentions pharma funding on its website but does not name companies.

Dr Armstrong also said NHSE accepted some information of the patient experience of living with pulmonary hypertension “on the grounds it could inform but not influence the decision. What is the point of information if not to influence?”

The NHSE guidance paper on the new process said some small patient organisations “may” receive some funding to complete the report.

An NHSE spokeswoman said: “To be criticised about ‘issues around openness and transparency’ on this decision by an organisation [PHA] receiving tens of thousands of pounds from the manufacturer of Selexipag, is questionable at best.

“We remain committed to ensuring the patient voice is heard and are currently testing ways to give charities and patient representatives an additional opportunity to contribute to [the CPAG] process.”

Jayne Spink, CEO for Genetic Alliance UK, an umbrella group for charities representing rare diseases, said: “Many patient groups, especially those operating on limited resources, are likely to struggle to develop their submissions. For this reason, dedicated resource to support patient groups as they engage with NHSE would be very welcome.”