The report from GBI Research expands on Health Economics and Outcome Research (HEOR), a type of analysis which is described as “a key element of market access” that covers real-world data (RWD), quality of life, budget impact, and cost-effectiveness, as well as clinical efficiency.
Capturing and analyzing data
In order to capture and analyze RWD, researchers use a wide range of tools, such as patient registries, electronic health records, routinely collected administrative data as well as other patient-level data sources.
“There are a few approaches or studies that aim to better capture RWD and these can vary in their design and complexity,” Rodrigo Gutierrez Gamboa, Managing Analyst for GBI Research told us.
For example, some pharmaceutical companies and research organizations have implemented “pragmatic” clinical trials, which are carried out post-approval and allow companies to measure a medical intervention’s effectiveness.
“This type of trial provides insights into variations between patients that occur in real clinical practice, and therefore evaluate the intervention’s effectiveness in real-world circumstances,” explained Gamboa.
Other types of trials that have seen an increase in adoption are cluster randomized trials (CRTs), a trial in which groups of subjects, as opposed to individuals, are randomized to receive one of the medical interventions being studied. According to Gamboa, CRTs reportedly enable a better approximation to the real-world setting for drug/program combinations.
“It has been widely observed that a meaningful RWD study design necessitates a significant and deep understanding of the real world environment,” he added. “This, in turn, necessitates easy access to robust and high quality real world health data and appropriate technologies and analytic capabilities to analyze the data.”
Gamboa further explained that data quality is often believed to be more essential than sample size in ensuring that a RWD study’s strategic objectives are met. “Therefore, a deep understanding of healthcare technology practices is essential to effectively assess the utility of the data,” he said.
However, there have been concerns regarding inconsistencies in the standard operating procedures and in the interpretation of RWD’s scope.
“Companies are therefore advised to consider the appropriateness of any internal procedures for these types of studies and ensure the right personnel are involved,” said Gamboa.
The Association of the British Pharmaceutical Industry (ABPI) has already taken steps to promote transparency and the integrity as it pertains to the collection and analysis of RWD in order to ensure consistency across the sector.
While some internationally-agreed standards exist, such as those in the UK, some countries still have no specific industry standards that cover all research that could be defined as an RWD study.
Industry factors encouraging RWD studies
According to Gamboa, a series of global macroeconomic and industry factors have placed “unprecedented pressures” on pharmaceutical companies, which as a result, have placed more emphasis on capturing and analyzing real-world data.
Some of these factors include, a global aging population, an increasing incidence of chronic conditions, surging drug prices, decreasing R&D productivity, and healthcare budgetary constraints in major healthcare markets.
Gamboa added that these factors “have encouraged national healthcare agencies, including Health Technology Assessment (HTA) agencies, and payers to become more sophisticated in their demand for evidence on the cost-effectiveness of new medical interventions.”
Additionally, due to the limitations of randomized clinical trials (RCTs), and the complexity of novel medical interventions, there has been an increasing interest in implementing RWD studies.
“RWD studies enable the capture of information relating to a drug’s performance, in normal clinical practice, within different age groups and genders as well as differences in disease severity and associated comorbidities,” explained Gamboa.
“It is not possible to obtain all of that data through RCT methodology.”
Addressing the issues facing the industry
Productivity in the pharmaceutical industry continues to decline – an issue that is currently forcing companies to investigate more efficient R&D models. According to Gamboa, RWD studies can provide insights early in drug development and during clinical trials, which would subsequently help companies save time and money.
“For instance, they can enable the identification of unmet needs by developing disease understanding and provide insights into patient behavior,” added Gamboa. “Clinical trial design and protocols could be aligned to current real world clinical pathways and facilitate new trial designs such as the previously described ‘pragmatic’ trials. “
RWD studies could also enable new commercial models by supporting novel contract design and contract management.
Gamboa concluded: “Some studies have suggested that if pharmaceutical companies are able to implement an integrated development model that brings together RCTs and RWD-based approaches, R&D productivity could be significantly revitalized by reducing cycle time and required R&D investment per product.”