Executive Summary
England’s health technology appraisal institute, NICE, has published its real-world evidence framework.
NICE LAUNCHES RWE FRAMEWORK
With its newly published real-world evidence (RWE) framework, NICE, England’s health technology appraisal (HTA) institute, is making “a real push into real-world data in a big way,” Sam Roberts, NICE’s chief executive, said last week at an online event to launch the initiative. The framework, published on 23 June, is intended to be a “living document” that will broaden overtime according to need.
With respect to RWE, NICE is aware that a “step change is needed for the future,” said Roberts, pointing to two recent developments. The first is the recently launched Innovative Medicines Fund, which provides access to promising new medicines via the National Health Service while more data is gathered to address uncertainties regarding their clinical and cost-effectiveness. (Also see “England Launches Innovative Medicines Fund” – Pink Sheet, 8 Jun, 2022.) “If we’re going to do that, we really need a robust framework where we all agree on what is the right data to collect, if it is good quality and how can we do that,” she said of the IMI.
The second “big thing” signalling the need for a stronger approach to RWE is NICE’s new early value assessments, which will see the institute look at early indicators of value for digital technologies, medical devices and diagnostics that may not have a “full evidence package.”
NICE’s RWE journey will not end with these initiatives given that the rate of innovation is increasing and that data are becoming available on a magnitude not seen before, commented Roberts. As pressures on health systems increase, RWE will have a role in “solving some very tricky problems,” she said. For example, it can be used to assess new technologies associated with uncertainty, or to monitor the uptake of new technologies to ensure patients are benefitting as much as they did in clinical trials, she said.
The framework is therefore a “living document” and will be updated in the years to come, said Roberts. It is part of the NICE 2021-2026 Strategy, which emphasized that RWD is integral to the institute’s “strategic ambitions.” These include rapid, robust and responsive technology evaluations, dynamic living guideline recommendations, effective guidance uptake, and leadership in data, research and science. (Also see “UK: RWD At Center Of NICE Five-Year Strategy” – Pink Sheet, 21 Apr, 2021.)
According to Seamus Kent, a senior advisor at NICE, the framework describes when and how RWE can be used to improve recommendations by the institute with regard to which health care products should be funded by the NHS. It does not attempt to “reinvent the wheel” or set out minimum standards, he said. Instead, the institute has collated best practice research and put it together in “a way that is meaningful for NICE and useful to stakeholders,” he said, speaking at the same event.
The framework uses a broad definition of real-world data to mean data collected outside of highly controlled clinical trials, explained Kent. The data can relate to patient health and experiences or care delivery, and can be quantitative or qualitive, including, for example, free text written by the doctor or patient reported outcomes, said Kent. He added that it can come from a number of sources, including health service administration records, patient health records or more structured sources like patient registries or observational cohort studies.
By analyzing the data, it can be used to form RWE that can be used in making recommendations. It can help the institute characterize conditions, understand the natural history of a disease, validate economic models, and assess the generalizability of a clinical trial to NHS patients and look at the effects on patients’ outcomes, said Kent.
There are three overarching principles for the framework:
- Generating evidence in a transparent way “with integrity from study planning to study conduct and reporting”
- Ensuring that the data is of good provenance, that it is trustworthy and fit for purpose
- The use of appropriate analytical methods that minimize the risk of bias and characterize uncertainty.
The framework provides recommendations on three main areas. The first is planning, conducting and reporting RWE studies. It applies mainly to quantitative data but several aspects can also be applied to qualitative studies. It also recognizes that the acceptability of evidence will depend on the type of evidence concerned and other contextual factors.
The framework advises clear specification of research questions, early planning of studies and clear descriptions of data sources and data curation data sources. It is important to show the journey from “source data to analytical datasets,” said Kent. He added that transparency is essential so that independent researchers can reproduce the results and ensure that NICE reviewers critically appraise the studies and be confident in trusting their use in making recommendations.
Another section of the framework sets out best practices on assessing data suitability. This describes the information necessary for assessing data provenance, the quality of the data and its relevance for addressing specific research questions.
A third section describes methods for real-world studies of comparative effects, including specific recommendations for conducting non-randomized studies such as traditional observational studies and clinical trials that form an external control arm using real-world data.
According to Kent, the framework will also be extended to other priority topics. Some areas have already been identified in a public consultation, including the need for more information on dealing with small sample sizes and the use of patient experiences and qualitative data, he added.
Advice
Jacoline Bouvay, technical director at NICE, advised companies that they can seek help from NICE with respect to “making the best use” of the framework. Companies anticipating a NICE evaluation could benefit from the institute’s scientific advice program so that they can see advice on clinical development plans. A “number of offerings” are possible she said, including joint advice with other HTAs, such as Canada’s CADTH, or with UK medicines regulator, the MHRA.
She also pointed to NICE International, an advisory service to support other countries in improving decision making in health and care. This could help other organizations around the world use the principles in the NICE RWE framework as part of their methods and processes or to develop their own versions of the framework.
Source: https://pink.pharmaintelligence.informa.com/PS146414/England-HTA-Body-NICE-Makes-Big-RWE-Push