Articles
Statement from FDA Commissioner Scott Gottlieb, M.D., on FDA's New Strategic Framework to Advance Use of Real-World Evidence to Support Development of Drugs and Biologics (12/6, Scott Gottlieb, M.D., FDA) comments “...The strategic Framework we're announcing today will serve as a roadmap for more fully incorporating RWD and RWE into the regulatory paradigm. The Framework outlines a number of important RWE-related efforts to explore the potential for using RWE to help support approval of new indications for approved drugs or to support or satisfy post-approval study requirements. This new Framework will help the FDA and industry evaluate the potential use of RWE to support regulatory decisions that have visible outcomes for consumers.” Full
FDA Will Allow Observational Studies to Support Effectiveness Determinations, New Framework Says (12/6, Zachary Brennan, Regulatory Focus) reports “...As far as what the framework will guide FDA on specifically, the agency said its RWE program will evaluate the potential use of RWE to support changes to labeling about drug product effectiveness, including adding or modifying an indication, such as a change in dose, dose regimen or route of administration; new populations; or the addition of comparative effectiveness or safety information. ‘The RWE framework will also consider the evaluation of observational clinical studies using RWD to support product effectiveness determinations,’ FDA said.” Full
The Case for Self-Funded Health Benefit Plans and Reference-Based Pricing (12/6, Daniel MacLeod, Employee Benefit News) comments “...[W]hat's behind the recent trend toward reference-based pricing for smaller employers?...First, a self-funded health benefit plan design that uses referenced-based pricing can mean less expensive coverage for employees and employers...Second, reference-based pricing can provide employees more flexibility when it comes to choosing a provider.” Full
The Extraordinary Importance of Sleep (12/2018, Susan L. Worley, P&T) reports “...‘Perhaps the most important aim these days when developing and implementing any type of large-scale clinical research study is to incorporate the patient's perspective,’ says Dr. Kushida, who is currently analyzing the results of a comparative effectiveness sleep study sponsored by [PCORI]. The study, designed and conducted by a team at Stanford, introduced a new model of patient-centered, coordinated care and tested it against conventional outpatient treatment for sleep disorders.” Full
What Diet Is Best to Manage Crohn's Disease? This Study Aims to Find Out (12/5, Lara DeSanto, HealthCentral.com) reports “...Researchers have launched a clinical research study, funded by the Foundation and [PCORI], to help remedy this issue. The study, called DINE-CD, is the first-ever national randomized trial of two dietary interventions to treat Crohn's disease. HealthCentral spoke with James Lewis, M.D., the primary investigator of DINE-CD, about the study's goals and the important role diet plays in Crohn's treatment.” Full
Treating the Individual, Not the 'Average' Patient (12/6, John M Mandrola, MD, Medscape) comments “Hospital leaders often tell clinicians to strictly embrace evidence-based medicine because patients will do better and the hospital will avoid pay-for-performance losses. In chart reviews, EBM looks easy: diagnosis A means the clinician uses medicines B and C. I love and fully embrace evidence. But this sort of nudging tramples on the founding principle of EBM. One sentence from the late David Sackett, MD, a pioneer in EBM, reveals the challenge: Evidence-based medicine is the conscientious explicit and judicious use of current best evidence in making decisions about the care of individual patients.” Full
Video: Allison Brennan Outlines Concerns With a Faster Move to Risk for ACOs (12/6, The American Journal of Managed Care) “Pushing accountable care organizations to take on risk faster will likely result in ACOs that aren't ready just dropping out of the program and could impact participation in value-based care, said Allison Brennan, MPP, senior vice president of government affairs for the National Association of ACOs.” View Video
Press Releases
ICER Opens Nominations for New Members of Its Voting Panels (12/5, ICER Press Release) “...Prospective nominees should have familiarity with the interpretation and application of medical evidence in health care delivery. This may include (but is not limited to) training in fields such as clinical epidemiology, health services research, or health economics, or experience in public health administration, consumer/patient engagement, or policy efforts to improve access to health care...To reduce the influence of possible conflicts of interest, prospective nominees cannot be directly involved in making medical policy decisions for any state health agency, or be employees of any private insurer or life sciences industry company.” Full
NICE Recommends XELJANZ®? (tofacitinib citrate) as a New Treatment Option for Adults with Moderately to Severely Active Ulcerative Colitis (12/6, Pfizer Press Release) “Pfizer announced that the National Institute for Health and Care Excellence (NICE) has published final guidance recommending the use of XELJANZ® (tofacitinib citrate), 5 mg and 10 mg twice daily twice-daily oral tablet, as a treatment option for adult patients with moderately to severely active ulcerative colitis who have had an inadequate response, lost response, or were intolerant to either conventional therapy or a biologic agent.” Full
Journals
Editorial: Evaluating Patient and Public Involvement in Research Antoine Boivin, et al. December 6, 2018, BMJ BMJ
Health Plan Strategies for Value-Based Care: Closing Gaps in Care Means Getting Serious About Prevention Joshua Sclar, MD, MPH December 6, 2018, The American Journal of Accountable Care AJMC