Dealing with real-world data (RWD) might be complicated, but it can be a highly useful tool in drug research and development. Still, considering the complexity and unfamiliarity, some trial teams might still be hesitant to take the RWD plunge.
Outsourcing-Pharma spoke with D.J. Tang, senior vice president of data services with Firma Clinical Research, about the potential benefits of harnessing RWD, as well as the possible pain points and how to overcome them.
OSP: You describe RWD as “messy”—could you please explain what you mean by that?
DT: That is due to the comparison with the clinical trial data which is collected based on the well-designed clinical trials and patient populations are controlled by the inclusion and exclusion criteria. The real-world data is usually collected from a wider patient population that might include patients with certain characteristics not studied in the clinical trials. Some of such characteristics may be confounded to the treatment effect. In addition, without randomization, the variation of the data is much larger than the clinical trial data.
OSP: Even though it’s messy, RWD is still pretty handy in the drug development process—could you please share some of the ways in which it’s beneficial?
DT: The usefulness of the RWD comes from two aspects. One is that it is much easier and more efficient to collect. Unlike in clinical trials, the RWD exists wherever patients take medicines regardless if it is internal or unintentional. The other is that the RWD can really help the drug developers to verify or estimate the treatment assumptions more accurately or provide supplemental information which is difficult to obtain due to the limitations of the clinical trials.
OSP: Please talk a bit about the data explosion of recent years—why the pile has grown bigger, the reasons why more data than ever is available, etc.
DT: The RWD is always available. For example, the physicians who prescribe the medicines would have the patient narratives for the treatment and reported side effects. The pile has grown bigger due to the mindset changes. People are more focused on the clinical trial data than before since it could provide needed information for drugs to be developed. However, more and more researchers and drug developers have seen the values of the RWD and would like to utilize them to help the developing process better and faster.
OSP: How has the 21st Century Cures Act impacted data collection and use, and the acceptance of using RWD?
DT: The US lawmakers signed the 21st Century Cures Act on December 13, 2016, to help accelerate drug development and bring new innovations to patients who need them faster and more efficiently. The FDA puts much effort into the implementation of the 21st Century Cures Act. As mentioned above, RWD is one of the areas to serve the purpose. There are multiple examples recently of the utilization of the RWD to help design the clinical trials and the products were approved. It is very encouraging for both patients and drug developers.
OSP: Please discuss where and how PIs, biometrics teams, and other key players can best leverage this type of data in their work.
DT: For PIs, they usually collect the RWD on individual patients. It will be helpful for them to see the summary of the RWD based on some particular purposes. It can provide them additional information on the treatment and make adjustments as needed. From the sponsor side, maximizing the utilization of the RWD is a booster to the development process. It should be always considered throughout the whole development duration, from the protocol development to post-marketing activities.
OSP: What kinds of tools and technology might teams put to use to help maximize their collection and utilization of RWD?
DT: In terms of the statistical methodology to analyze the RWD, many researchers have been working in this area and multiple new methods have been developed, either published or ongoing. The key challenges are mainly two items. One is on methodology - how to mine non-randomized or highly correlated data to get meaningful information. Much progress has been made for this item.
The other is to integrate the RWD from different sources. It is not technically difficult, but it takes much effort to put the data from different places together. The formats may be totally different for different physicians, clinics, hospitals, regions, and countries. Creating a usable database will usually take much longer time than analyzing the data itself.
OSP: Do you have anything to add?
DT: In this area, a lot of work has been done and even more work is to be done. Introducing a standardized data collection approach would save much time and effort. The RWD should and will be a key part of drug development.