Government regulators’ refusals of off-label drugs surged last year. Off-label use refers to prescribing drugs for unapproved indications for medical needs.
If a medical institution decides that a specific unapproved drug is medically valid for treatment through consultations with its multidisciplinary committee, it can apply for approval of the off-label use to the Health Insurance Review and Assessment Service (HIRA). HIRA makes the final decision for the off-label use.
According to HIRA, the agency rejected 69 applications for off-label use last year. It marked a rapid increase from 17 refusals in 2016, 12 in 205, 19 in 2014, and 30 in 2013.
Among them, drugs with the highest number of refusals were “Dongindang Indocyanine Green Inj.,” a diagnostic reagent for liver function and circulatory function tests, blood product “Liv-Gamma SN Inj.,” and immunotherapy “IV-Globulin SN Inj.” Their applications were turned down in four cases.
Three applications for the off-label use of Xeljanz were declined. Facing two refusals, respectively, were blood cancer treatment Truxima, immunosuppressant Rapamune, non-Hodgkin's lymphoma treatment Mabthera, and immunosuppressant Cipol-N Soft Cap.
Experts said the health authorities should make use of the accumulated clinical data on drugs in off-label use, carry out a public clinical trial, and reflect study results on registering of health insurance and deciding drug reimbursement.
Yoon Young-ho, a professor of family medicine at Seoul National University’s College of Medicine, stressed the need for clinical research for the public interest, in a debate at the National Assembly on Wednesday.
Yoon said clinical studies for the public interest would be able to improve patient access to advanced medicines, enhance the quality of care through evidence-based treatment, reduce patients’ economic burden with health insurance coverage of the drug, and expand insurance coverage through the pre-emptive implementation of preliminary reimbursements.
The Ministry of Food and Drug Safety (MFDS) agreed with Yoon that efforts should be made to turn some unapproved drugs to approved ones through public clinical trials.
“Some physicians who cannot carry out a clinical trial need the off-label use of unapproved drugs. I agree that such medicines can be granted through state-supported clinical trials,” said Kim Eun-hee, head of the ministry’s task force for evaluation of the off-label use of drugs. “However, clinical research for the public interest should be credible in terms of data and design.”
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