Real-World Data and Real-World Evidence For Clinical and Regulatory Decision Making

 Background

Traditionally, physicians have relied heavily on clinical evidence most notably from randomized controlled trials to guide their clinical decision making. Nowadays, clinicians have access to a wealth of additional data generated from sources outside traditional research settings, such as remote monitoring devices, wearables, disease registries, electronic health records, and claims and billing activities. Data related to patient health status and/or the delivery of health care routinely collected from a variety of sources have been defined as Real-World Data (RWD) by the FDA. On the other hand, Real-World Evidence (RWE) is described as clinical evidence about the usage and potential benefits or risks of a medical product derived from an analysis of RWD. RWD and RWE offer the possibility to bring about novel insights on patients’ clinical care, regulatory decision-making, and health technology assessment. Detailed information on data sources, data standards, data reliability, how to generate evidence, and regulatory considerations can be found on FDA’s document titled Framework for FDA’s Real-World Evidence Program (U.S. FDA, 2018).

While evidence-based data from clinical trials remains the bedrock for clinical decision making, clinicians now have the opportunity to enhance the process by incorporating findings from RWE. A recent New England Journal of Medicine opinion column proposes that RWE derived from an analysis of RWD can effectively complement “the knowledge gained from ‘traditional’ clinical trials, whose well-known limitations make it difficult to generalize findings to larger, more inclusive populations of patients or settings reflective of actual use in practice.” It is important to distinguish two key dimensions of RWE. The first is the setting in which evidence is generated, which includes the population defined by the data source as well as the specific methods used to collect and curate the data on that population. The second is the methodologic approach used to conduct the surveillance or research (Sherman et al., 2016).

There is increasing interest in the use of real‐world data (RWD) to support regulatory decision making. To directly support European Medicines Agency (EMA) committees, the EMA is routinely using three real-world databases for in-house studies and over recent years has commissioned 15 external studies, most of them multi-database and multinational. It is also well recognized that RWD are an underutilized source for assessing the public health impact of risk minimization measures, including any unintended consequences and for informing health technology assessment, pricing, and reimbursement decisions (Cave et al., 2019).

Healthcare facilities improve their capabilities on implementation of electronic medical records along with electronic prescribing. Doctors are getting actively involved in telemedicine. Patients starting to use various mobile applications to access health services. The general public’s use of computers, mobile devices, and wearable devices to gather and store health-related data has been rapidly accelerating. Data obtained from these electronic health records, cancer registries, and other RWE sources can produce valuable insights into treatments and clinical outcomes in daily practice.

There are already many examples of RWD and RWE in the context of clinical and regulatory decision making. RWD on the performance of COVID-19 vaccines provides a perspective on vaccine efficacy in non-ideal situations. RWE from cancer therapy could become even more important, the conduct of post-marketing studies could be of great value in confirming or refuting the cancer drugs’ benefit on survival in real‐world populations.

 Objectives

The goal/objectives of this online meeting include:

1. Increase understanding and awareness of the importance of real-world data and real-world evidence to improve quality in healthcare services, daily clinical practice, and regulatory decision making.
2. Providing an overview of real-world data and real-world evidence.
3. Sharing valuable information on methodologies of real-world studies.
4. Discuss the use of real-world data and real-world evidence and the exciting potential for cancer therapy, infectious disease, and vaccine.
5. Understand the challenges to use real-world data and real-world evidence as well as whether it may be sufficiently relevant and reliable for various decision makings.

 Topic of the Online Meeting

Theme : “Real-World Data and Real-World Evidence”
Topic   : “Real-World Data and Real-World Evidence for Clinical and Regulatory Decision Making”
Method: Webinar conducted through Zoom Meetings and Youtube Livestreaming.

 Participants

1. Staffs from the Ministry of Health (from several Directorates)
2. Staffs from BPJS Kesehatan (Healthcare and Social Security Agency)
3. Staffs from Badan POM (Indonesia National Agency for Drug and Food Control)
4. Members of PERSI (Indonesian Hospital Association)
5. Members of IDI (Indonesian Medical Doctor Association)
6. Members of IAI (Indonesian Pharmacist Association)
7. Academia
8. Members of IPMG (International Pharmaceutical Manufacturers Group)
9. Members of GP Farmasi (National Association of Local Pharmaceutical Manufacturer Companies)
10. Other participants

 1^st Meeting Agenda, Saturday 06 November 2021

Time	Topic	Speaker
14.00-14.05 (5’)	Opening	MC
14.00-14.10 (10’)	Welcoming Speech	Dr. dr. Eti Nurwening Sholikhah, M.Med.Ed., M.Kes. (Chair of the Department of Pharmacology and Therapy, Faculty of Medicine, Public Health, and Nursing Universitas Gadjah Mada)
14.10-14.30 (20’)	Introduction to Real-World Data and Real-World Evidence	Prof. Dr. Dra. Erna Kristin, M.Si., Apt. (Department of Pharmacology and Therapy, Faculty of Medicine, Public Health, and Nursing Universitas Gadjah Mada)
14.30-14.50 (20’)	Methodologies of Real-World Studies	Dr. dr. Rizaldy T. Pinzon, M.Kes., Sp.S. (Hospital Management Program Faculty of Medicine, Public Health, and Nursing Universitas Gadjah Mada /Bethesda Hospital Yogyakarta)
14.50-15.10 (20’)	Discussion Session 1	Moderator dr. Alfi Yasmina, M.Kes, M.Pd.Ked, Ph.D. (Faculty of Medicine, Universitas Lambung Mangkurat)
15.10-15.30 (20’)	Integrating Real-World Evidence to Clinical Decision Making	Dr. dr. Anwar Santoso, Sp.JP(K), FIHA, FASCC (Harapan Kita National Heart Center Hospital Jakarta)
15.30-15.50 (20’)	Relevance of Real-World Data and Real-World Evidence for Regulatory Decision Making	Prof. Dr. dr. Sudigdo Sastroasmoro, Sp.A(K) (Chairman of the National Committee of Health Technology Assessment 2014-2020)
15.50-16.10 (20’)	Discussion Session 2	Moderator dr. Alfi Yasmina, M.Kes., M.Pd.Ked., Ph.D. (Faculty of Medicine, Universitas Lambung Mangkurat)
16.10-16.15 (5’)	Closing	MC