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The Academy of Managed Care Pharmacy (AMCP) Nexus 2019

Data Collaborations Are Driving Improvements to the Use of Real-World Evidence

Silas Inman
As the number of expedited FDA approvals for cancer drugs that are based on surrogate end points increases, so does the need for new ways to uncover efficacy and safety data to justify the costs associated with these treatments. With the growth of data innovations and collaborations, the answer might be found in real-world evidence.
The number of expedited FDA approvals for cancer drugs that are based on surrogate end points is increasing, leading many to search for new ways to uncover efficacy and safety data to justify the costs associated with these treatments. With the growth of data innovations and collaborations, the answer might be found in real-world evidence (RWE), according to presentations at the 9th Annual Research Symposium held in conjunction with the AMCP Nexus 2019 meeting,

"We can't expect this to directly replicate clinical research, but it's really important to advance this methodology," said Jeff Allen, PhD, president and chief executive officer, Friends of Cancer Research (FOCR), a group that was instrumental in the development of the breakthrough therapy designation. "If we're going to get back to the idea of how we're going to expedite the development of treatments for patients who have no other therapeutic options, but also carry out the responsibility to continue to evaluate the effect of these therapies and benefits over time, this is a way to advance that need in a different way."

Since the launch of the breakthrough designation in 2012, the FDA has approved 150 breakthrough therapies and granted more than 332 breakthrough therapy designations, according to FOCR.1 In an analysis of the impact of the breakthrough program conducted in 2016, the designation was found to accelerate premarket drug development by 2.2 years, representing a significant trend toward rapid access to potentially lifesaving medications.2

However, this new paradigm for drug approval has generated controversy, since a majority of the regulatory decisions under the program are based on surrogate end points and not on overall survival (OS). While OS is the gold standard, there are many challenges that make it difficult to achieve, even in large, randomized phase 3 trials, Allen noted. In general, cross over between arms commonly confounds the calculation of OS, and, in some cases, the amount of time needed to show a statistical benefit is simply too long.

RWE has the potential to overcome these challenges, Allen noted, and FOCR has teamed up with several data partners, including the American Society of Clinical Oncology (ASCO)'s CancerLinQ, Cota, and Flatiron Health, to conduct a pilot study to find out exactly how it can help. The analysis has shown that patients in the real-world setting were older than their randomized controlled trial counterparts and that data collected in the pilot study could be used to compare across treatment groups for several efficacy end points.

Through the collaboration, clinical end points like progression-free survival (PFS), time to next treatment, and time to discontinuation could be tracked in a patient population that closely mirrored a true treatment setting, Allen said. Many of these end points correlated with OS; however, survival itself still remained challenging to calculate, as the date of death is not always available, he added.

"What we were able to see, at least from the initial partners who did this analysis for us, is that they were able to construct, with great detail, the different characteristics of the populations who were receiving each product," Allen said. "They were also able to show that some of these non-traditional measures, like time to treatment discontinuation, actually correlated quite well with traditional measures, like overall survival."

Advancing Cancer Surveillance
The Surveillance, Epidemiology, and End Results (SEER) program, which has been funded by the National Cancer Institute (NCI) since 1973, houses real-world data for 34% of the US population. While the registry has traditionally had several limitations, the NCI is looking to expand the reach of the database through partnerships, according to Donna Rivera, PharmD, MSc, a scientific project officer at the NCI.

Through these partnerships, the NCI is looking to add data on comorbidities, adverse drug events, treatment success, second and third courses of treatment, and health outcomes, such as survival. SEER has traditionally only collected diagnosis, staging information, tumor characterization, and initial course of treatment.

"The main goal for the enhancement is to create a system that represents population-level, real-world data to supplement clinical trials and understand the effectiveness of oncology care outside the clinical setting," said Rivera. "Clinical trials represent 3% to 5% of the adult oncology population, however, that leaves 95% of patients outside of clinical trials, for which we need quality data."

The new initiative looks to add further biomarkers, through partnerships with Genomic Health and Myriad/Invitae. Additionally, partnerships with CVS and Walgreens will provide data on outpatient prescription use for oral medications, with the ability to measure not only utilization but also adherence. The collaboration will also tap into electronic health records (EHRs) through ASCO's CancerLinQ program and will include information on radiation oncology through partnerships with Varian/Elekta.



 
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