CER Daily Newsfeed

The Comparative Effectiveness Research Daily Newsfeed®, known for short as the CER Daily Newsfeed®, offers the latest news, research and related information on comparative effectiveness research, real-world data and evidence, value assessment and other important health care topics. 

News from Wednesday, June 22, 2022

Articles

New York Times Piece on Drug Market Ignores the Data (and Reality)

(6/22, John M. O'Brien, Real Clear Policy) comments “...The truth is that the majority of the increase in new pharmaceutical spending is not caused by price increases. It is the result of more people using newer drugs more frequently, and more and more of that additional spending is going to middlemen who neither discovered nor developed the drug. In 2020, more than half of spending on brand drugs went to someone other than the developer, up from one-third in 2013...It is also true that on a net basis, in 2020 prescription drug spending was $359 billion, less than 9% of total health care expenditures. Net prices for new drugs are actually growing more slowly than inflation. More broadly, good research should cite net spending, as the important difference between list and net prices used in research is well documented in research literature.” Full

 

Moving Toward Broader Value: Expanding Value Assessment Beyond Clinical Drug Trial Data

(6/22, Beth Beaudin-Seiler, et al., Health Affairs Forefront) comments “...Rapid growth in real-world data can capture information on costs and health outcomes for all types of services, thereby adding value to existing information in limited administrative claims. As discussed by Brett McQueen, real-world data can help us measure what matters to patients and caregivers across the continuum of care. Greater use of data sources outside of FDA-required trials opens more possibilities to measure value in ways that align with patients’ preferences and recognizes how the outcomes important to patients can vary across disease areas and individuals.” Full

 

Pfizer Is First Big Pharma to Tap Truveta’s Real-World Data Platform

(6/22, Phil Taylor, PharmaPhorum) reports “...Pfizer is the first big pharma company to sign up to make use of the platform, which used machine learning and automation to sift through the clinical data points to detect patterns that could help to inform patient care – and potentially spot an emerging safety issue in the early stages. It is ‘one of the most timely and complete datasets available in the US,’ said Pfizer’s head of worldwide medical and safety Dr Aida Habtezion. Using the resource, Pfizer can ‘learn directly from de-identified patient data at an unprecedented pace and scale,’ she added.” Full

 

Resources, Roles, and Returning Value: Lessons from Patient- and Participant-Driven Research Communities (Part 2)

(6/21, Claudia Grossmann, PhD, Michelle Johnston-Fleece, MPH and Julianna Paris, MPH, The PCORI Blog) comments “...In partnership with NORC and AcademyHealth, PCORI assembled a Learning Network of eight PDRCs to create a forum for peer-to-peer learning. Three workgroups were subsequently formed to address challenges identified by learning network members with the goals of providing forums for sharing learnings and identifying generalizable lessons that could be shared broadly. Ultimately, 3 priority gap areas were identified: improving representation, enhancing digital engagement, and supporting sustainability.” Full

 

International Regulators Plan Roadmap For Collaborative RWE Studies

(6/22, Ian Schofield, Pink Sheet) reports “...Experiences during the COVID-19 pandemic are expected to help identify future opportunities for regulatory cooperation on real-world evidence and avoid divergent decision-making for products including advanced therapies and personalized medicines.” Paid Subscription Required

 

UK: NICE Opts to Reject Acorda’s MS Drug Fampyra

(6/22, John Pinching, PharmaTimes) reports “...The National Institute for Health and Care Excellence has announced its decision to reject Fampyra – also known as fampridine – for use in England. The drug was developed by Acorda Therapeutics and is manufactured by Elan Corporation. The treatment can significantly improve walking ability and quality of life for people with multiple sclerosis, but will not be used across the NHS in England.” Full

Press Releases

The Science of Patient Engagement: How PCORnet® Is Leading the next Hot Topic in Clinical Research

(6/22, PCORnet Press Release) “...[A] new manuscript published in Learning Health Systems shares how the PCORnet infrastructure can serve as a model for the burgeoning science of patient engagement, providing much-needed insights on how large research networks can effectively engage patients. ‘If we can learn how to effectively partner with patients across the research continuum—from defining research questions through study design, conduct, and dissemination—we can generate findings that are more relevant, complete, and trusted by communities,’ said Elizabeth Cope, senior director of public and population health at AcademyHealth and lead author of the manuscript.” Full

Journals

Stakeholder Engagement Infrastructure to Support Multicenter Research Networks: Advances from the Clinical Research Networks Participating in PCORnet

Elizabeth L. Cope, et al.

May 12, 2022, Learning Health Systems

Wiley

 

From Scientific Discovery to Covered Treatments: Understanding the Payer Perspective as a Keystone to Achieving High-Value Care

Carlos Blanco, MD, PhD; Shari M. Ling, MD; Wilson M. Compton, MD, MPE

June 21, 2022, JAMA

JAMA

 

Performance Metrics for Models Designed to Predict Individualized Treatment Effect

Carolien C.H.M. Maas, et al.

June 21, 2022, medRxiv

medRxiv