Demands for access to new therapies: are there alternatives to accelerated access?

Abstract

Patients deserve timely access to new therapies, but the rhetoric surrounding accelerated access impedes rational policy making.It is almost impossible to turn on the television or open a newspaper without hearing about the “miraculous” benefits of the latest medicines. The targeted cancer therapy idelalisib, for example, was touted as a revolutionary treatment that would “melt away” your cancer,1 while the new leukaemia drug venetoclax has been described as being so innocuous that it is “like taking Panadol [paracetamol].”2 While much of this rhetoric centres on cancer medicines, new treatments for other chronic and life threatening conditions such as diabetes,3 cystic fibrosis,4 and Duchenne muscular dystrophy5 are also described as miracle cures. The mass media is replete with stories of terminally ill patients who have been given a second chance by these new miracle drugs. However, alongside such stories of triumph are darker stories—of patients having access to these life saving drugs denied or compromised by excessively conservative regulators or cost conscious public or private insurers (payers). Headlines over the past few years include “Aussie patients denied funding for 30 life-saving drugs,”6 “Dying mum fights for life-prolonging drugs the NHS won’t fund due to cost,”7 and “Company denies drug to dying child.”8 This rhetoric is indicative of an increasingly pervasive social expectation, which we refer to as the access imperative. By this we mean the view that patients with severe or life threatening diseases should not have to wait (as long as they do) for regulatory approval or formal subsidy before they can access medicines. This access imperative seems to be gaining in strength, leading to numerous recent inquiries into the adequacy of existing regulatory and reimbursement systems including in the UK and Australia and calls to expedite access to promising new treatments.Key messages Both patients and clinicians are immersed in rhetoric emphasising the potential benefits of and urgency of access to new medicines Policy makers are under increasing pressure to approve and fund medicines with poor quality evidence on safety, efficacy, and cost effectiveness Alternative approaches are needed to meet the desire for quicker access and protect the interests of current and future patients as well as the broader community