Access to life-saving drugs for seriously ill patients, or easy money for pharmaceutical companies?
We have just had our latest paper published, in JRSM Open. A little different from our usual studies, this one is about the safety of a scheme run by the UK regulator, the Medicines and Healthcare products Regulatory Agency (MHRA). The Early Access to Medicines Scheme (EAMS) was established in 2014 to provide access to medicines that don’t yet have a marketing authorisation for UK patients with life threatening or seriously debilitating conditions. Sounds good? We were interested to see whether, ten years on, it is benefiting patients. Strangely though, we discovered that patients have not been asked for their views, nor have any reviews of the scheme considered the perspective of patients, the people for whom it was ostensibly set up. Instead, we found that reviews focused on the benefits of the scheme to pharmaceutical companies – such as early guidance from the regulator and access to patient populations – and to the UK economy.
Our analysis focuses on 48 submissions to the scheme, relating to 48 indications (mostly cancer) and 32 individual drugs. Unfortunately, we found that the quality of evidence used by pharmaceutical companies to support their submissions was poor, with most studies using surrogate and/or survival outcomes and only 7% using double-blind, placebo controlled randomised trials. Even more worryingly, almost half the expedited drugs had elevated rates of suspected adverse reaction reports according to the pharmacovigilance data we examined. Shockingly, given the paucity and quality of evidence upon which most expedited drugs are approved, the MHRA does not even demand post-approval clinical trials to check whether the promised benefits actually exist.
Perhaps we should not be surprised about this. During her time at the MHRA, Dame June Raine, its Chief Executive Officer, has changed the culture of the agency from one that sees its responsibility as scrutinising pharmaceutical company data on behalf of the public, to one that makes it as easy as possible for companies to market their products. And she is quite open about this. In 2022 she gave a talk at Oxford entitled, ‘From Watchdog to Enabler’ and in 2020 she proclaimed to the House of Commons Science and Technology Committee and the Health and Social Care Committee, ‘The agency, which is an independent regulator, but formerly seen perhaps as a watchdog, should now become an enabler …’. (Q780)
Can a regulator make it as easy as possible for companies to market their products and at the same time protect the public health? Read our paper here: https://journals.sagepub.com/doi/10.1177/20542704251317916
