More and more regulators are demanding real-world outcomes data after medicinal product authorization. Even the most innovative clinical trials require outcome studies to further demonstrate the safety, effectiveness and risk/benefit evaluation of the treatment in diverse populations in China.

However, our clinical trial models at present often lack the processes and resources to gather this data. This gap can only be closed if we as an industry more proactively pursue the collection of evidence-based data through observational studies as part of our broader trial initiatives. By investing time and resources into real world late phase (RWLP) studies, we can broaden the medical research horizon, stimulate evidence-based medicine, and strengthen partnership between the medical community and industry, which drives benefits for patients, physicians, hospitals, and biopharmaceutical companies.

Certainly, RWLP research requires additional time and effort on the part of researchers, but the benefits more than make up for the dedication. These studies provide evidence of value for innovative, higher-cost medicines versus lower cost standards of care, and provide clinicians with a better understanding of long-term clinical outcomes in patients. That data can also be used as evidence in the medical guidelines and help treatment decisions made in real-life medical practice. And these RWLP studies provide information about cost-effectiveness among alternative treatment options for diseases with unmet medical needs — ultimately supporting payer decisions about what treatment the government should pay for.

We’ve already seen a trend toward more large-scale RWLP research emerging in China. The Shanghai Clinical Center for Endocrine and Metabolic Diseases (CCEMD) has conducted several major epidemiological studies in the last five years, including the 2010 China noncommunicable disease surveillance study aimed to assess and monitor the prevalence of and risk factors for noncommunicable diseases in Chinese adults. The study recruited 98,658 participants over the age of 18 in 162 cities across mainland China and has recently published a paper based on the survey’s findings in the Journal of the American Medical Association (JAMA).

Another noteworthy study is the China Cardiometabolic Registries (CCMR), funded by China’s Ministry of Health and supported by several multinational biopharma companies. This nationwide registry series were designed to better understand clinical outcomes of current treatment of cardiovascular and metabolic diseases in real world settings, and we’ve already seen compelling results.

The data collected in this study shows:

  • Achieving control of CVD risk factors in patients with Type 2 Diabetes remains a clinical challenge. That reinforces the argument that an early intervention strategy inclusive of control of integrated 3B targets (HbA1c, Blood Pressure and Blood Cholesterol) and modifying cardiovascular disease predictors is imperative to improve outcomes in adult patients.
  • The prevalence of diabetes in China is growing — with a majority of patients undiagnosed.
  • Glycemic levels were properly controlled (HbA1c<7%) in only approximately 50 percent of the patients. This demonstrates why achieving control of risk factors for cardiovascular disease in Type 2 diabetes remains a challenge in China.
  • There is a significant gap between guidelines and real-world clinical practice. 

These studies demonstrate that nation-wide, cross-sectional or longitudinal data are of significant value in China. Accessing to those high quality data will help physicians make decision among different therapeutic options in the real-life diverse patient population. Therefore, more RWLP studies are warranted to improve quality of patient care and disease burden.

Some training required

To perform these studies researcher will face more challenges than just financial and time commitment. Real-world research requires different expertise and experience from those used in clinical trials intending to bring products to the market, for example, study design and analyses methods. To stay ahead of this trend, both academic centres and biopharmaceutical companies in China need to further develop their teams’ expertise in epidemiology and biostatistics expertise as well as project management and clinical monitoring strategy.  

The demand for real-world data will only get stronger. Those companies that prioritize these studies now, will position themselves for greater success in the future.