The GetReal project team previously reviewed stakeholders’ policies and perspectives on using RWD for early drug development and relative effectiveness assessment. This review was supplemented by Stakeholder interviews conducted with 19 different stakeholders from 7 stakeholder groups: pharmaceutical industry, health technology assessment (HTA) agencies, regulatory agencies, healthcare payers or insurers, academia, RWD initiatives and patient organisations. The review can be found on the GetReal website.
The key findings of this review were as follows:
- Differing policies: stakeholder groups have different policies on RWD collection, partly because of their different goals and mandates. Policies on access to RWD available through pragmatic clinical trials, electronic health records and administrative claims databases, for example, vary dramatically between countries, regions, states and even between organisations. The steps that need to be taken by boards of governance to approve plans for real-world studies also vary according to the context of the studies.
- Cultural barriers: there is a cultural barrier among stakeholders that reduces the acceptability of real-world evidence (RWE) in drug development and relative effectiveness assessment. Many adhere to a hierarchy of evidence that prioritises data from randomised controlled trials (RCTs) over non-RCT data.
- Standardisation of data: the majority of authors and interviewees highlighted the lack of standardisation of RWD because it is collected for different purposes. They emphasised the need to standardise the types of RWD collected, and the methods and tools for collection and analysis. However, they also noted that stakeholders have different goals and mandates that affect what data are collected and the strengths and limitations of the data. Some authors and stakeholders stated that vast amounts of RWD are already available, for example, through health surveys, observational studies and administrative claims databases.
Since the publication of this GetReal review, progress has been made in relation to adopting the use of RWD and RWE and developing policies and guidelines that set out how it would be used in practice by the key stakeholders involved.
In this section, we list some examples of the relevant policies, guidance and/or position statements that have been published or updated since the publication of this review. Entries have been groups by type of decision-maker: regulatory agencies (for marketing authorisation), formal HTA agencies (for reimbursement and coverage), and other HTA organisations (influential in driving formal policies and decisions, but without decision-making powers). This list was last updated on 21 February 2020.
US Food and Drug Administration (FDA)Framework for FDA's
RWE Programme Submitting Documents Utilizing RWD and RWE for Drugs and Biologics
Use of RWE to Support Regulatory Decision-Making for Medical Devices Use of Electronic Health Records in Clinical Investigations
European Medicines Agency (EMA)
Therapeutic Goods Administration (Australia)
National Institute for Health and Care Excellence (NICE, UK)
Amr Makady and Wim Goettsch, Zorginstituut Nederland (ZIN)
Emily Leckenby and Dalia Dawoud (NICE)
Michael Chambers (MC Healthcare Evaluation)