By Angela Woodall, Healthcare and Environmental Reporter.
Challenges faced by researchers trying to translate stem cell based therapies into real-life treatments could be likened to the 1969 lunar landing: Even though they’re operating at a different scale, both had to start with a number of unknowns and build the necessary infrastructure along the way. The big hurdle in the stem cell world now involves answering questions as elemental but challenging as to which cells are productive and most effective.
2019 will likely be one in which the industry as a whole works to build bridges between big ideas and effective, safe results. Granted, the recent scandal involving the CONCERT-HF trial, an experimental stem cell therapy in cardiac patients, didn’t help.
The same forecast holds true for clinical trials in general. Complexity is on the rise everywhere, whether the study is in very different fields such as orthopedics or ophthalmology. The work varies from managing the kind of interdisciplinary teams recommended for today’s clinical trials to wrangling “real world” big data projects, which could have far-reaching impact on study design and personnel.One startup claims to have reproduced results of a clinical trial using information gathered from patient records, while the Food and Drug Administration (FDA)has greenlighted the first randomized clinical study using data from the Sentinel electronic health records system (the IMPACT-Afib trial).
Passed two years ago, the 21st Century Cures Act emphasizes the use of what is called real world data (RWD) to support regulatory decision making. According to the FDA, data gathered from mobile devices, medical records, administrative records and other sources, and the findings produced from them, are being used to support clinical trial designs and observational studies to generate innovative, new treatment approaches.This in turns raises another topical issue: How to evaluate the quality of data, which, as the Center for Drug Evaluation and Research at the FDA points out, are seldom produced with research goals in mind.
More guidance is likely to come in 2019: Already the FDA has proposed a slate of new guidelines for expansion cohorts, adaptive trial designs, and master protocols. Detectable is the attempt to balance a measured approach that protects patients with demands by the very same patients and their advocates for results not caution.
The pressure speaks to the increased influence of trial participants and nonprofit advocacy organizations. It’s worth noting that the FDA just launched a digital device to “help capture real world data from patients to help inform regulatory decision-making” and recently authorized a third party company to charge patients for pre-approval access to a novel placenta-based cell therapy despite the participants’ ineligibility for the company’s Phase 3 clinical trial of the product.
On the pharmaceutical side, the industry is eager to bring down the cost of drug development while speeding up delivery to market. They’ll be watching as politics reaches directly into their goals as the White House and government attempt to push through a drug price-cutting agenda. The debate swivels between warnings that price controls will cripple R&D, and thus clinical trials, and arguments that prescription drug prices are unjustifiably high and must be lowered for the well-being of patients.
The year ahead promises to be an interesting one. In that spirit, here is a summary of what else 2019 might have in store for clinical trials:
- More states legislating reimbursements to patients for expenses associated with participation in a clinical trials, which has already caught the attention of Lyft and Uber.
- The pharmaceutical industry will explore the potential of clinical trials in China to increase access to trial participants while reducing costs.
- Imaging and biomarkers will continue to be a growing factor in clinical trials.
- Endpoints will continue to be renegotiated to keep pace with advances in regenerative therapies.
- CROs must recognize the heterogeneity of patients in trial designs and cater more closely to their needs in order to improve participation.
- U.S. lawmakers will feel more pressure to drop the obstacles to cannabis trials as the domestic market grows and other nations move forward with studies.
It’s your turn! What are your predictions for 2019?
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