The most innovative new cancer drugs are taking longer to pass through clinical trials, licensing and eventual appraisal by the National Institute for Health and Care Excellence (NICE), an analysis carried out by the Institute of Cancer Research (ICR) has suggested.
European Medicines Agency (EMA) authorisation data from 2000 to 2016 showed that the numbers of cancer drugs being authorised has increased in the past two decades; however, the most innovative treatments — including those that have a novel target or mechanism of action — took 3.2 years longer to move from patent filing to NHS patients.
Further scrutiny by researchers at the ICR, published on 29 January 2019 in
Drug Discovery Today
, revealed that much of this delay seemed to happen in the period between starting phase I trials and authorisation by the EMA which lasted an average of 8.9 years for the most innovative drugs.
The ‘most innovative drugs’ were categorised as those that acted against a new molecular target or via a novel mechanism; represented a novel class of compound in an area of high unmet need; was novel in its application; or offered improved targeting through use of a biomarker. The equivalent delay for the least innovative drugs was 6.8 years.
Examples of delayed cancer drugs include mifamurtide (Mepact; Takeda UK), which took 20 years to go from patent to NICE approval for osteosarcoma, and trabectedin (Yondelis; Immedica), which took 22 years to navigate clinical trials and approval processes for advanced soft tissue carcinoma.
For some cancer types, including brain and oesophageal cancer, there were no new treatments licensed from 2000–2016. Only 8% of the drugs licensed in the same time period were for use in children, the study found.
The research team — led by Paul Workman, chief executive of the Institute of Cancer Research, has called for streamlined clinical trial regulations and licensing processes, as well as stronger incentives for innovation in drug discovery and development.
The team pointed out that NICE had reduced the lag time between EMA authorisation and starting the technology appraisal process from 21.0 months to 6.5 months over the 16-year period, although the appraisal process was taking the same amount of time.
Workman said that while the study highlights major progress in the number of new drugs coming through the process, it does highlight shortcomings in the regulatory process.
“[Our study] makes clear that our regulatory systems are not keeping pace with advances in the science. It is taking longer for new drugs to reach patients and, alarmingly, the delays are longest for the most exciting, innovative treatments, with the greatest potential to transform the lives of patients.”
Commenting on the research, Simon Cheesman, lead pharmacist for cancer at University College London Hospitals NHS Foundation Trust, said the results were not completely surprising, but that ensuring a treatment really did confer benefits to patients was tricky to balance against speed of access.
“It can be hard to assess the true benefit, safety and cost-effectiveness of a new medicine following initial clinical trials with surrogate endpoints and short-term follow up, and to balance this with rapid access to get the drug to those patients who might benefit as soon as possible,” he said.