‘Innovative drug approvals require robust scientific evidence’

Innovative drugs can win approval without the existence of specific regulatory guidance but the scientific evidence and rationale must be convincing and persuasive, an official at the drug regulator said Wednesday.

Kim Jong-won, director of the vaccine inspection division at the Ministry of Food and Drug Safety (MFDS), offered regulatory advice amid the explosive development of novel drugs in Korea which are surpassing the issuance speeds of regulatory guidance documents.

Kim, who has regulatory work experience with the World Health Organization (WHO) offered advice to drug developers gathered at BIO Korea 2023 in southern Seoul, Wednesday. 

“Regulatory approval was granted by the MFDS in the past for gene therapies and most recently for digital therapeutics (DTx) without strict regulatory guidance documents,” Kim remarked.

He stressed that developing those guidelines for innovative drugs was not a top-down approach but required the expertise of academic and industry experts to include their opinions and experience in making the guidelines.

Kim also added that for emerging microbiome therapeutics, there are already drugs that have won FDA approval.

Still, he quickly noted that gathering chemistry manufacturing and controls (CMC) data can be tricky domestically as microbiome contract research organizations (CROs) in Korea currently lack expertise compared to the U.S.

On another note, he also urged drug developers to understand the direction of each regulatory guideline as regulatory perspectives from the FDA, EMA, and MFDS can all differ.

The MFDS director pointed out four critical data submissions including GMP, CMC, non-clinical toxicity, and clinical materials which should all meet the minimum requirements to succeed in regulatory approval and market authorization.

"Particularly for biosimilars, the European regulator tends to place more emphasis on CMC data while the FDA considers other data submissions more equally," he elaborated.

Kim also noted that the Covid-19 pandemic helped to make more individuals aware of regulatory processes and just like the Covid-19 vaccines and therapeutics development were accelerated, drug developers can replicate those results but need the right strategies.

Accordingly, he mentioned collaboration with all stakeholders including scientists and physicians from the early stages of clinical trials and thinking ahead of future problems when designing clinical trials, as two examples of strategies.

“Many also believed that these accelerated approvals during Covid-19 translated to less strict regulatory guidelines,” Kim explained. “Faster regulatory approval was only possible thanks to greater human resources on both the regulatory and drug manufacturer’s end.”

He emphasized that the same can be done outside of the Covid-19 pandemic to accelerate regulatory drug approvals but drug developers should invest wisely in human resources to deliver quality data for submission.

Meanwhile, BIO Korea 2023 kicked off on Wednesday under the theme of “Connected era, Leaping towards Next Level.” The conference will continue for two more days with seminars spanning three main areas of technological innovation and R&D acceleration, digital innovation, and technology business.