Survey by BioCentury and Happy Life Technologies finds high hopes but delayed expectations for RWE to deliver value in China R&D
BY LAUREN MARTZ, SENIOR EDITOR AND RIMMEL SHEKHA, BIOPHARMA ANALYSTNovember 10, 2021 2:09 PM PSTBioCentury & Getty Images
Real-world evidence is working its way into global clinical development and commercialization strategies with an impact that’s expected to grow, but implementation is lagging in China.
Recent policy changes to promote use of RWE in drug development in China could mean a surge in adoption is on the horizon, once awareness rises and other barriers to implementation come down.
Strict data privacy laws, data integrity concerns and a lack of institutional experience with RWE studies are among the biggest perceived barriers to RWE uptake in China, according to a survey conducted by BioCentury and Happy Life Technologies (HLT) Group (Figure 1).
Figure 1https://media.graphcms.com/t3P2yRBaSbarzuP14eui
The survey results also conclude that though some companies are using RWE for post-market assessments, there’s opportunity to add value in pre-market clinical development applications, and in reimbursement negotiations both in China and globally.
The survey of more than 80 biopharma executives, professional service providers, investors, and government and non-profit groups was conducted via live polling from Oct. 6-25.
Historically, RWE has been an important type of evidence for monitoring vaccine and therapeutic safety after approval. With the growing body of electronic medical data, tools to analyze the data and innovation in clinical trial design, more applications are emerging.
The pandemic represented a turning point for RWE, drawing industry attention to its potential to supplement standard clinical trial data, and accelerating its adoption. Alternative clinical development tools capable of speeding evidence generation and capturing effects across diverse demographic groups became important as traditional clinical trials became more difficult to execute.
RWE also emerged as a valuable tool for tracking outcomes after authorization of medical countermeasures that had been rapidly developed.
The applications are now clear, but a set of internal and external barriers has created hesitancy among both companies and regulators. The survey results suggest those barriers are being dismantled, and that the reach of RWE should start to extend past the post-market monitoring applications where it has a solid footing to begin making more contributions to trial designs, regulatory decisions and payer negotiations.
Global optimism for RWE
Executives surveyed by BioCentury and HLT agreed the momentum for RWE generated during the pandemic is likely continue building in the coming years.
Of the 81 participants surveyed, 91% envision the use of RWE changing over the next five years, with nearly half expecting a large increase in adoption of RWE by companies and acceptance by regulators.
Figure 2https://media.graphcms.com/9c1MCJtxSS2xGE09HKsy
Figure 3https://media.graphcms.com/96qAOhd1TpmWrVCcPxJ2
Buy-in from companies and regulators may need to go hand-in-hand because companies aren’t motivated to perform the studies without a reasonable expectation they’ll be accepted by regulators, and regulators can’t have confidence in RWE studies without research from companies to validate them.
Of the two disease areas where respondents expect RWE to make its biggest mark, cancer and rare diseases (Figure 3), the latter may stand to benefit more from incorporation of RWE in the development pathway.
Due to scarcity of patients, using real-world data to guide site selection, stratify patients, and substitute for placebo control arms is particularly valuable in rare diseases.
Meeting clinical endpoints in a cost-effective way can be challenging for many rare diseases, suggesting development could be incentivized by use of RWE as a postmarket tool to confirm benefit after approvals, based on limited or surrogate data.
Across disease areas, RWE has already penetrated postmarket settings, and survey respondents believe that is still where these data are likely to be most effectively used in the next five years.
While safety assessments are a validated application of RWE, survey participants are most excited about the use case of postmarket efficacy assessment (Figure 4).
With the expanding use of accelerated approval pathways to help drugs reach patients faster, there’s an increased need for post-market research tools such as RWE to provide a complete picture of clinical efficacy and safety. This year’s BioCentury Back to School collection calls for greater use of RWE in confirmatory trials for accelerated approval.
Figure 4https://media.graphcms.com/kfDohXBeTdqizrpVbKD9
Bringing RWE to China
Real-world evidence was once perceived as off-limits in China due to privacy, data and regulatory barriers, but policy has recently started to change to enable broader use of RWE, particularly by Western companies bringing therapies to China.
Several guidelines covering the use of RWE in clinical development have been released in China since the beginning of last year.
Use of RWE is also now encouraged in Hainan Province’s Bo’ao Lecheng International Medical Pilot Zone, where drugs approved in the U.S., Europe or Japan, but not yet in China, can be deployed by physicians and RWD in Chinese patients can be generated. Several products have been approved by China’s NMPA based on RWE generated in the pilot zone.
Despite the warming regulatory environment, growth in use of RWE remains slow, in part because of a lack of awareness.
According to the BioCentury/HLT survey results, most industry representatives aren’t aware of Hainan’s Pilot Zone.
The survey captured responses from 39 people in North America, 34 in Europe and 6 in Asia. Overall, 34 (43%) of the respondents said they have business in China.
Less than one-third of those who answered the question (19/62) said they were aware of free trade zones that allow use of RWE in regulatory submissions to NMPA, and the use of therapeutics and devices approved in other regions (Figure 5). However, 44% (26/59) expect the free trade zones have a high or very high likelihood of driving future use of RWE in China (Figure 6).
Figure 5https://media.graphcms.com/1kzDYnNxSojUrl8WMzoQ
Figure 6https://media.graphcms.com/vNHJGJ0QL6t0sTEqFlYE
Figure 7
Among the companies that said they do business in China, less than one-third said they’ve used RWE in the country; however, another third are considering it (Figure 7).
The most common applications for RWE in China are related to marketing and commercialization, according to survey responses (Figure 8). These are the low-hanging fruit for RWD implementation, but the results also suggest the number of companies using RWE in China may be overrepresented by the survey. Some companies consider market research such as surveys to be RWE, but that doesn’t fall under BioCentury’s definition of evidence generated from data collected in claims databases and electronic medical records.
Emerging opportunities for RWE include clinical research and reimbursement negotiations, both of which are being pursued by some companies in China. In clinical trials, RWE can feed into trial design by identifying the best trial sites based on patient concentrations and providing information about disease prevalence and natural history. It can also serve as historical control arms to avoid unnecessary placebo administrations.
In payer negotiations, RWE can provide a broader picture of safety, efficacy and durability in clinically relevant populations than can be captured in standard randomized controlled trials.
Figure 8
Remaining barriers
Despite the growing regulatory acceptance in China, companies globally still see data privacy, integrity and regulatory acceptance as problems in the region (Figure 9).
Data integrity issues extend beyond China to the global state of data quality and standardization. One problem with data for RWE, which usually comes from electronic medical records and insurance claims databases, is that the data aren’t necessarily structured for research purposes.
Data can be incomplete, not digitized, and not standardized in scales, collection or recording methods.
China’s later entry to RWE generation gives it an opportunity to build an infrastructure that’s standardized and allows companies to extract data relevant to drug development from the start.
Source: https://www.biocentury.com/article/640602/bringing-down-real-world-evidence-barriers-in-china