By Dawn Marie Stull, scientific director and lead of INSIGHT MM, Takeda Oncology
There is more focus than ever before on tailoring treatment plans to the unique needs of patients. One way to ensure we as an industry are delivering the most value is by examining real-world data (RWD) throughout the drug development process.
According to the U.S. Food and Drug Administration (FDA),1 RWD is playing an increasing role in healthcare decisions, making it more important than ever for pharmaceutical companies to adapt to the changing landscape. In fact, more than half of leading life science companies are developing or significantly improving their RWD capabilities, according to a 2017 Deloitte report.2
Randomized controlled trials (RCTs) are the gold standard in establishing the efficacy and safety profile of a drug. This means, by design, the trials have strict criteria to eliminate bias and maintain the integrity of the collected data. A specific hypothesis is tested that requires one treatment be tested against another. The comparator arm is typically considered a current standard of care treatment in the population being studied. This data remains critical to the drug development process; however, it does not tell the whole story.
Gaps often exist between information gathered about a drug or treatment in RCTs and that same drug or treatment when used as part of routine clinical practice in the real world. Pharmaceutical companies should consider investing in the design and implementation of RWD studies that will increase the understanding of a medication’s impact outside of the rigors of RCTs. Because real-world studies have less stringent eligibility criteria and include a more diverse patient population, they often can help to fill in these gaps and provide a more complete understanding of the true therapeutic benefit that can be achieved when these same drugs or treatments are employed in the real world.
Observational trials are one type of study that provides RWD. The results may help healthcare providers understand and optimize treatment plans based on a patient’s specific circumstance and, if patient reported outcomes (PROs) are included, they can help contextualize the impact treatments have on the patient’s quality of life. Recognizing the immense potential that RWD has in terms of elucidating the true benefit of treatments when they are prescribed as part of routine clinical practice, Takeda is investing in this type of research.
In 2016, Takeda launched INSIGHT MM, the largest global, prospective, non-interventional observational study on multiple myeloma to date. The study plans to follow patients for an extended period of time in an effort to track patterns in disease presentation, patient characteristics, treatment, and associated outcomes, including the impact of the disease and its management on the patient’s quality of life. INSIGHT MM, with its long duration of follow-up, will help inform the optimal sequence of treatments that result in the best outcomes – which is one of the most critical questions in multiple myeloma treatment today.
Validated PRO questionnaires are utilized to enhance the understanding of the real-world experience of patients with multiple myeloma. PROs collected during observational studies capture the impact of the patient’s disease and associated treatments on the patient’s quality of life in routine clinical practice. This information is valuable for physicians and patients to have when making treatment decisions, in addition to traditional efficacy endpoints like response rates, progression-free survival (PFS), and observational studies. Observational studies such as INSIGHT MM are non-interventional, meaning that participants do not alter their established treatment plans in any way in order to participate and there are no additional appointments required. We know firsthand from our multiple myeloma Patient Leadership Council (PLC) at Takeda, as well as the patient, caregiver, and advocacy representatives that sit on the trial’s steering committee, that traveling to appointments can be very cumbersome for patients. Because no additional clinic visits are required for this study, the PRO results will allow researchers and healthcare providers to see how the required routine care and follow-up needed for multiple myeloma patients impacts their quality of life.
Leading healthcare organizations are in the process3 of establishing frameworks for assessing RWD. In addition, regulatory bodies such as the FDA and EMA have developed guidelines for researchers working on real-world data sets to optimize the trial design in hopes the data can be used to support drug approvals. In addition, the FDA recently released guidance for sponsors4 on how they can incorporate patients with more challenging health conditions into oncology clinical trials. Inclusion of patients with comorbidities as well as those from underrepresented demographics can make trials more illustrative of real-world oncology care and is an increasing focal point for the industry.
The ultimate goal for the INSIGHT MM study is to better inform treatment decisions so that the best possible outcomes are achieved in as many patients as possible. The design of INSIGHT MM, the first truly global pharma-sponsored trial, enables the participation of a larger variety of patients around the world, with more than 130 study locations in 15 countries and across four different continents. Earlier this year, the study reached its goal of enrolling 4,200 participants globally.
An early analysis of the data from INSIGHT MM, conducted in 2017, showed 40 percent of patients enrolled in the trial would not have been eligible for a Phase 3 RCT in multiple myeloma, proving that INSIGHT MM allows us to capture the experience of patient groups that aren’t represented in RCT data. Observational trials like this allow physicians access to data for a wider variety of patient subgroups with diverse ethnicities and comorbidities. This data helps to address the growing need for more robust data sets that include a more diverse patient population treated in routine clinical practice across the globe.
As we look to the future, RWD may have increasing importance in the future of drug approvals and label expansions. In many countries, RWD is a key part of securing drug approval and access, and the U.S. FDA has indicated that it is open to considering RWD. In order to further understand treatment options as they pertain to patients’ daily lives, pharmaceutical companies should consider the value of observational real-world studies that evaluate how patients respond to treatment outside of the clinical setting.
- U.S. Food and Drug Administration. Real World Evidence. https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence. Accessed August 27, 2019.
- Deloitte. Getting real with real-world evidence. https://www2.deloitte.com/content/dam/Deloitte/us/Documents/life-sciences-health-care/us-ls-2017-real-world-evidence-survey-031617.pdf. Accessed August 27, 2019.
- Julie C. Locklear, PharmD, MBA. What is real-world evidence, and why do we need it? STAT News, https://www.statnews.com/sponsor/2017/02/16/real-world-evidence-need-2/. Accessed August 27, 2019.
- U.S. Food and Drug Administration. FDA In Brief: FDA takes new steps to broaden patient participation in cancer clinical trials, advancing policies to promote inclusion of pediatric patients and patients with medical conditions that can occur alongside cancer. https://www.fda.gov/news-events/fda-brief/fda-brief-fda-takes-new-steps-broaden-patient-participation-cancer-clinical-trials-advancing. Accessed August 27, 2019.
About The Author:
Dawn Marie Stull, Pharm.D., BCOP, is the scientific director and lead of INSIGHT MM at Takeda Oncology. Prior to joining the pharmaceutical industry, she worked as part of multidisciplinary healthcare teams providing clinical care to hematology/oncology patients. Stull has over two decades of experience and has held several positions within the pharmaceutical and healthcare industries. Most recently her focus has been in the area of global medical affairs and the generation of real-world data.