OBJECTIVES

: Patient advocacy groups (PAGs) are increasingly involved in shaping health policy. This is particularly true in the UK, where health authorities gain extensive input from patient organisations. As the role of PAGs in determining policy rapidly evolves, this research aims to assess their impact on drug appraisal and commissioning in England.

METHODS

: Three high-profile case studies were analysed, with insights validated in thirty-minute interviews conducted with three UK payer advisors.

RESULTS

: Three recent episodes demonstrate the extent to which PAGs can influence access decisions in England. In 2016, the National AIDS Trust won a funding appeal when the UK courts confirmed NHS England’s lawful ability to commission preventative medicines and overturned the initial refusal to fund HIV pre-exposure prophylaxis. Likewise, last year, the National Institute for Health and Care Excellence (NICE) reversed its decision to restrict drugs for relapsing multiple sclerosis (MS) after the MS Trust argued that NICE’s appraisal failed to consider critical patient factors. More recently, several parents of children suffering from cystic fibrosis (CF) have formed a ‘buyer’s club’ to purchase generic CF drugs from abroad and bypass the negotiation deadlock between NHS England and the originator manufacturer.

Payer advisors recognise the increased involvement of PAGs in decision-making and the growing opportunity to leverage them to achieve a more informed and patient-centric approach when determining health policy. However, payers highlight that greater PAG engagement will require increased collaboration between health authorities and drug manufacturers to guarantee timely access to innovative therapies and prevent patient disengagement.

CONCLUSIONS

: PAGs are progressively shaping access for novel medicines in England. Partnerships between PAGs, health authorities and drug manufacturers can be mutually beneficial and lead to improved long-term health outcomes for patients. A strong collaboration amongst the key stakeholders is essential to ensure that patient representation results in maximised patient-centricity in drug appraisal and commissioning.