Leveraging RWE and Managed Entry Agreements in Asia Pacific

Published Nov 1, 2022

Summary Report from the ISPOR Asia Consortium Industry Committee Meeting

Prepared by Sang-Soo Lee, PhD, MBA, Chair, ISPOR Asia Consortium Industry Committee, Sr. Director, Health Care Economics and Government Affairs (HCE&GA); Medtronic North Asia (Korea and Japan), and Head of Center of Expertise (COE), HCE&GA, Medtronic Asia Pacific, Seoul, South Korea

The ISPOR Asia Consortium Industry Committee held a virtual meeting on June 29, 2022 to gather stakeholders interested in health policy and health technology innovation in the Asia Pacific region, and for updates on trending topics in healthcare. The meeting featured 2 presentations from 1) Xin Sun, PhD, Professor and Director, Chinese Evidence-based Medicine Center, West China Hospital of Sichuan University, Chengdu, China, and 2) Ying-Li (Tommy) Chen, BS Pharmacy, MHS in Health Economics, Board of Director of HTAsiaLink and HTA Researcher of Center for Drug Evaluation (CDE), Taipei, Taiwan.

“Use of RWE for Regulatory and Reimbursement Decisions in China” presented by Professor Xin Sun, NMPA Hainan Key Laboratory of Real-World Data Research and Evaluation; Chinese Evidence-based Medicine Center, Huaxi Hospital, Sichuan University, Sichuan, China; ISPOR West China Chapter

In recent years, real-world data (RWD) and real-world evidence (RWE) have gained substantial attentions in China. Since the concept of the RWE study was introduced in 2009, the RWE methodologies and data infrastructure, including the establishment of routine RWD and patient registry collection pathways, the release of government regulations on data privacy and quality for research purposes, and the publication of a series of methods guides or holding training courses by academic organizations, have rapidly developed in China. All these initiatives laid a foundation for evidence-based decision-making in healthcare.

In particular, the needs for drug development and decisions, such as clinical evaluation and approval, post-approval regulatory studies, and coverage decisions, have promoted the production and use of RWE. In China, as the National Drug Administration Conference (2019) explicitly promoted regulatory science research, one focus of the research was incorporating RWE into drug approval and improving regulatory decision making. A series of technical guidance documents were also released to speed up the clinical development of new drugs and medical devices. In addition, the regulatory body put forward an innovative RWE framework for Traditional Chinese Medicine (TCM) products approval, which combined TCM theory, RWE and classical trials. Among those, an important initiative was to develop the Key Laboratory of Real-world Data Research and Evaluation in Hainan under the guidance of the National Medical Products Administration (NMPA). This is the first RWE key lab in China, with joint efforts between West China Hospital of Sichuan University, Hainan Medical Products Administration, and Lecheng. The key lab owns a special healthcare environment and is the primary place that facilitates generation of RWE to support clinical evaluation or regulatory approval of drugs and medical devices which are approved by major regulatory bodies overseas but not in China. Up to now, there have been cases for drug and medical device approval using Boao RWE.

The use of RWE for coverage decision is still at an early stage in China, although unsystematic efforts and sporadic examples were available to demonstrate the potential of RWE for coverage. The Hainan RWE program has the potential to support drugs or medical devices specially approved in initial coverage. The Key lab has set out to construct a complete workflow of Boao RWE studies for regulatory or coverage decisions. Hainan RWE program will be a helpful platform for a feasible approach in the exploration process of regulatory science research.

“Experience and Lessons Learned: Utilizing Managed Entry Agreements and Real-World Evidence in Taiwan’s Health Policy”, presented by Ying-Li Chen, Board of Director of HTAsiaLink and HTA Researcher of Center for Drug Evaluation (CDE), Taipei, Taiwan

The National Health Insurance Administration (NHIA) proposed the “general rules of reimbursement submission for high-cost drugs” and indicated that all drugs cost over 500 million NT dollars (16.6 million USD) must propose a managed entry agreement (MEA) model and a local pharmacoeconomic study in their reimbursement applications. In Taiwan, 3 types of MEA models were applied, (1) mutual share by negotiation, (2) financial-based and (3) performance-based. The main idea of MEAs is to facilitate access to new treatments while sharing the risk of uncertainty between stakeholders. Taking the reimbursement of Immune checkpoint inhibitors (ICIs), a new category of high-cost drugs in cancer treatments, is one of the examples. The NHIA set an 800-million budget for 3 ICIs in 8 indications and requested all patients receiving ICIs must be registered. With the MEAs, the NHIA kept the listing prices of ICIs at a level preferable to pharmaceutical companies while being able to treat as many patients as possible within a capped budget.

CDE was commissioned to continuously review the efficacy of ICIs by analyzing data from National Immune Checkpoint Inhibitor Registry Database (ICIRD), the database that contains clinical data from patients received ICIs. The real-world evidence (RWE) generated had shown that the patient with urothelial carcinoma or renal carcinoma had better outcomes compared to other indications. At the same time, the response rate of hepatocellular carcinoma and gastric cancer were the lowest. The NHIA suspended the new applications of hepatocellular carcinoma and gastric cancer based on the result. The MEA is an important tool to share the risk of uncertainty for drugs with uncertain clinical efficacy. It does not apply only to cancer drugs but any drugs that the insurance administration considered necessary. Moreover, RWE is another important tool for agencies to keep track of the usage and efficacy of drugs reimbursed, and agencies may revise the reimburse criteria based on the RWEs.

Suggested reading: Hsieh ST, Ho HF, Tai HY, Chien LC, Chang HR, Chang HP, Huang YW, Huang JJ, Lien HJ, Huang LY, Lee PC. Real-world results of immune checkpoint inhibitors from the Taiwan National Health Insurance Registration System. Eur Rev Med Pharmacol Sci. 2021 Nov;25(21):6548-6556. doi: 10.26355/eurrev_202111_27097. PMID: 34787857.

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