Is the push for faster regulatory approval of new drugs a benefit to patients or a safety risk?
The increasing pressure from patients, pharmaceutical companies and sometimes, health professionals, eager to access new medications, needs to be handled cautiously, perhaps via new approval categories, an expert believes.
In an editorial in Australian Prescriber, Paul Kubler, a rheumatologist and clinical pharmacologist, Royal Brisbane and Women’s Hospital, said we need to be cautious about following the rapid approval path set by the US Food and Drug Administration.
He believes a three-year temporary approval category (such as that recently set up by the TGA) should used more frequently to allow for enhanced monitoring of these medicines in their initial period of use.
In March 2018, the TGA announced its provisional approval pathway that allows drugs to be available for up to six years, based on preliminary data. The anticancer drug olaratumab was the first drug to be considered for provisional approval in Australia.
“Access to new therapies is a balance between evidence (determining the risk of acceptable adverse effects versus efficacy) and the speed of availability, intersected by the issue of affordability,” Kubler wrote.
“Temporary access is akin to a learner driver receiving their provisional licence – a full licence is only granted after more experience.
Rapidly approved drugs should receive provisional registration for a period of three years and the drug company should be required to provide annual data on the post-marketing experience,” he said.
Currently the TGA requires sponsor companies to report all negative outcomes that they become aware of, “but there is no imperative for them to actively and meticulously seek out adverse events, or confirm efficacy after approval,” Kubler said.
“As pharmacovigilance relies on spontaneous voluntary reporting of adverse effects by clinicians, it is highly likely that safety concerns are under-reported”.
Dr Kubler said that improving the scientific rigor of post-marketing information to track effectiveness and safety outcomes, either through independently monitored registry studies as a condition of initial registration or data linkage (such as linking of PBS and MBS datasets), will be crucial during any provisional registration period.
“If efficacy outcomes in the real-world environment are not confirmed or a significant safety problem emerges, then the drug’s registration should be suspended, at least for previously untreated patients, until the sponsor satisfactorily addresses the problems,” he concluded.
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