COMMUNICATION IN MEDICINE:
Policies, Pressures and Practice Patterns:
Long-Term Evaluation Needed
May 25, 2021
In 2016, the 21st Century Cures Act (Cures Act) directed the Food and Drug Administration (FDA) to accept statistical methods that include Real-World Data (RWD) and Real-World Evidence (RWE). The next year, the Center for Devices and Radiological Health (CDRH) and Center for Biologics Evaluation and Research (CBER) issued guidance regarding the submission of RWD and RWE to the FDA.
The Role of RWD and RWE
Both RWD and RWE have important roles as defined by the legislation to:
- monitor post-market safety and adverse events that result in regulatory decisions
- expedite approval of innovative therapeutic approaches, RWD and RWE support clinical trial designs, including approval of new indications for approved drugs
- develop therapeutic guidelines and decision support tools for clinical practice
- support healthcare insurance coverage decisions
Sources of RWD
RWD may come from various government and commercial sources including:
- Electronic health records
- Claims databases
- Billings databases
- Registries: product and disease
- Patient-generated data
- Health status data from mobile devices
To mitigate potential bias, the use of RWD requires a careful study design, protocol, and analysis plan that is comparable to a more traditional investigation, like a randomized controlled clinical trial, with strict inclusion/exclusion criteria and clinical endpoints.
There are some challenges with prospective, randomized, controlled clinical trials. Two major drawbacks:
- Large numbers of patients are required to conduct the study
- Slow patient accrual; strict patient eligibility
Study needs, research ideas and business strategy including synthetic control arms, also known as historical control arms or external control arms, are important to consider.
This type of control arm is defined as a group of patients treated at an earlier time (historical control) or a group treated during the same time period, in another setting.
Emergence of RWD and RWE provides new opportunities to efficiently conduct large-scale studies that have many of the benefits of randomized controlled clinical trials, while being generalizable to broad populations.
Discovering and analyzing RWD provides information regarding the clinical outcomes associated with a therapeutic approach. These outcomes, from a wide range of study designs, generate RWE.
When RWE is submitted to the FDA, both the RWD and RWE must conform to recognized data standards for file formats and data structures, using standardized variables and definitions.
Advantages and Outcomes
Historical or external control arms overcome many barriers to the completion of clinical trials, and provide a representative, rather than a highly selected, patient population in randomized controlled trials.
In cancer trials, there are many advantages including patient access to therapeutic breakthroughs.
Watch this video to learn more about this important topic
Medical paradigms are changing. Inefficiencies when conducting clinical trials are being addressed. Therapeutics are no longer siloed by medical subspecialty. Long recognized risks for development of disease are better understood by interconnecting biological pathways.
Outcomes are more than survival; they now also reflect the importance of controlling symptoms of disease and treatment. Greater emphasis is placed on outcomes beyond the clinical trial and in real world populations. Outcomes within real-world experiences reflect both the personal and societal cost of disease.
Nora Janjan, MD, MPSA, MBA
FACP, FACR, FASTRO, FASCO
Chief Medical Officer
Dr. Janjan enhances our team with her broad experience in regulatory affairs, development of national and international standards for healthcare, and unique academic credentials to enrich our knowledge base and provide high-quality clinical expertise to our team. As one of a few physicians nationally with a master’s degree in public service administration and business administration, her expertise includes clinical trials, health economics and outcomes research, healthcare policy, and patient-reported outcomes.
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