Monday, October 31, 2016

Open Access Publications from CMTP: "Barriers to NGS" and "Clinicians and Pragmatic Trials"

The Center for Medical Technology Policy (CMTP) in Baltimore has published two recent, open access publications on genomic test policy and evidence collection.

The first, in Applied & Translational Genomics, is "Barriers to clinical adoption of next generation sequencing: Perspectives of a policy Delphi panel."   By Messner et al., find the open access paper here.  (Abstract clipped below the break).

The second, in Contemporary Clinical Trials & Communications, is "Understanding practice-based research participation: The differing motivations of engaged vs. non-engaged clinicians in pragmatic clinical trials."   Also by Messner et al., find the open access paper here.

In 2008, Messner, now Senior Vice President of CMTP, wrote a very interesting PhD thesis at University of Edinborough on the historical development of Fast Track policy at the FDA.   (The thesis is that there are a variety of statutory, regulatory, subregulatory, and practice-based policy changes all occurring in various stages of coordination and completion, at any given time.)   The PhD thesis is online here.

For the paper on "Barriers to clinical adoption..." the abstract is:
This research aims to inform policymakers by engaging expert stakeholders to identify, prioritize, and deliberate the most important and tractable policy barriers to the clinical adoption of next generation sequencing (NGS). A 4-round Delphi policy study was done with a multi-stakeholder panel of 48 experts. The first 2 rounds of online questionnaires (reported here) assessed the importance and tractability of 28 potential barriers to clinical adoption of NGS across 3 major policy domains: intellectual property, coverage and reimbursement, and FDA regulation. 
We found that: 1) proprietary variant databases are seen as a key challenge, and a potentially intractable one; 2) payer policies were seen as a frequent barrier, especially a perceived inconsistency in standards for coverage; 3) relative to other challenges considered, FDA regulation was not strongly perceived as a barrier to clinical use of NGS. 
Overall the results indicate a perceived need for policies to promote data-sharing, and a desire for consistent payer coverage policies that maintain reasonably high standards of evidence for clinical utility, limit testing to that needed for clinical care decisions, and yet also flexibly allow for clinician discretion to use genomic testing in uncertain circumstances of high medical need.
For the paper, "Understanding practice-based research participation," which is highly relevant to pragmatic trials, real world evidence, and registries, the abstract is:
Pragmatic clinical trials (PCTs) represent an increasingly used strategy for “real-world” trials. Successful PCTs require inclusion of community-based practices. 
However, community clinicians often have limited interest or experience in clinical research. Many barriers to practice-based research have been described, but possible motivations to participate among community practices not active in research have not been well explored. The tendency is for researchers to assume similar motivations and priorities across all candidate practices. This is not necessarily the case. A better understanding of the range of reasons clinicians might see for participating in pragmatic trials could be key to promoting this type of practice-based research.

Separately, a 2016 paper on barriers to precision medicine, see Bertier et al., Genome Medicine, here.