What's patient-centered safety in the biopharmaceutical era?

Kymriah and Zolgensma

From the controversy over safety and accessibility to efforts to develop cutting-edge drugs in Korea,

The Ministry of Food and Drug Safety's GBC, policy discussion, Yoon's approval and salary are expected to proceed simultaneously

Along with the application of the world's first CAR-T cell therapy Kymriah and Zolgensma, an alternative treatment for spinal proximal phagocytosis (SMA), the biopharmaceutical era has opened in Korea.

 

Controversy over safety and accessibility has not yet been resolved in the process of innovative biopharmaceuticals being developed and used to treat patients.

 

Seo Kyung-won, director of NIFDS, who attended the debate, said, "It seems to be the first session related to patient safety during the 8th session of the GBC," adding, "Thinking about how and at what point patients' opinions should be contained should begin from now on." Director Seo said, "Although it was not actually implemented due to the spread of COVID-19, we wanted to provide a place to listen to the stories of patients who participated in clinical trials of anticancer drugs developed in Korea at expert meetings such as the Central Pharmaceutical Review Committee." Director Seo expressed his willingness to directly hold the patient's opinion, saying, "It is a task I really want to do in the future." What is the controversy over biopharmaceutical safety and accessibility?

 

Lee Hyung-ki, a clinical pharmacology professor at Seoul National University Hospital who was in charge of the topic presentation, pointed out safety problems such as unexpected side effects in clinical trials on patients due to the nature of biopharmaceuticals.

 

Professor Lee said, "A skin injection reaction that did not occur in a preclinical trial of an innovative biopharmaceutical company in Korea occurred in a clinical trial of a patient," adding, "Biopharmaceuticals have unexpected problems, and the safety of long-term administration has not been clinically confirmed." Professor Lee's opinion is that as more innovative biopharmaceuticals have been developed and used to treat patients, the issue of safety should not be underestimated after approval.

 

Regarding biopharmaceutical safety, the industry showed a slightly different idea.

 

Choi In-hwa, executive director of Roche Korea, said, "Stability is under follow-up management such as management and post-marketing management to carefully review data when the Ministry of Food and Drug Safety approves products." Executive Director Choi said, "There are drugs that have been approved in foreign countries, but have not been approved in Korea, and in some cases, benefits are applied after two to three years of approval due to complicated benefit processes." Executive Director Choi said, "We have no choice but to be upset from the perspective of patients who are threatened with rare and incurable diseases or lives." Choi added, "There are products from the introduction of innovative new drugs such as rapid approval system, rare disease designation system, permit-drug link system, rare disease law, RSA, and PE exemption, but improvement is needed because there are not enough systems that reflect patient opinions." Critics of drug accessibility were also pointed out by patient groups.

 

Lee Eun-young, director of the Korean Association of Patient Organizations, said, "The right to access drugs should start with permission." She noted, "Kymriah took more than a year to get approval from the Ministry of Food and Drug Safety after FDA approval, and it took 13 months to register benefits." Director Lee said, "Quick permission and benefit registration are important issues," adding, "The Yoon Seok-yeol government will be established and the Ministry of Health and Welfare will carry out pilot projects and main projects so that approval and drug price negotiations can be carried out at the same time for rapid registration." An important phrase here is the establishment of a cooperative relationship with the Ministry of Food and Drug Safety.

 

Director Lee said, "If the Ministry of Food and Drug Safety does not grant prompt permission, rapid registration is useless," adding, "If a pharmaceutical company applies for an item permission, the Ministry of Food and Drug Safety must approve it and then go through the HIRA and the NHIS procedures." At this time, he emphasized that "the accessibility of new drugs will be stabilized only when patients are centered, and it will be a patient-centered policy that the Ministry of Food and Drug Safety is talking about." The association also agreed that the Ministry of Food and Drug Safety's patient-centered policy can be realized only when patients have access to new drugs.

 

Park Jung-tae, vice chairman of the Korea Biopharmaceutical Association, said, "In foreign cases, patient safety is reflected in the policy," adding, "It is important to secure therapeutic access to patients by opening the way for active participation in regulatory science." The Ministry of Food and Drug Safety also expressed a consensus on this point, but explained why it is practically difficult.

 

Jung Hyun-chul, head of the Ministry of Food and Drug Safety's bio-policy department, said, "The keywords of today's debate are the same as rapid screening and rapid approval," adding, "With the introduction of the Breakthrough Designation system in the U.S.

 

in 2015, Korea also enacted the Breakthrough Drug Development Promotion Act the following year, but it was scrapped." The scrapped bill calls for introducing an occasional review to review the results of each product development process even before applying for permission to boost the development of miracle drugs and shorten the approval period.

 

It also includes the basis for the conditional permission system, such as priority screening before other drugs so that innovative drugs can be supplied to terminal cancer patients in a timely manner, submitting clinical trial data that can confirm the effectiveness of the drug, or prescribing only specific trained medical personnel.

 

"Because rapid approval and screening are really necessary for high-tech biopharmaceuticals, the 2019 Advanced Biopharmaceutical Act was passed, and a bill related to drugs responding to the public health crisis was prepared due to the spread of COVID-19 in 2020," said Jeong.

 

He said, "As it requires a lot of manpower as well as the burden of reviewers, recruitment of manpower should be done." There was also an explanation for the approval of the therapeutic purpose of overseas clinical trial drugs currently being promoted.

 

"Overseas clinical trial drugs are expected to be used for a small number of patients," Jeong said.

 

"The problem is that there should be a great burden as the KFDA judges have to proceed with permission only with doctor's medical certificates and opinions without seeing any non-clinical data and quality data."