Hot Topics From Around the World

Proponents of real-world evidence say it will yield information that is both more targeted and more thorough than the evidence that clinical trials can provide. But that doesn’t mean real-world evidence research is easy to do—or inexpensive—notwithstanding the relative cost of the randomized trial. What’s more, electronic health records and insurance claims may contain a wealth of information, but neither are designed for medical research. And if wearable devices were to live up to merely half of their hype, even more data would come pouring into the mix. Collecting, organizing, and understanding data requires skill, time, and money and many current computer systems just aren’t up to dealing with that data firehose. Read more.

2. MHRA Posts Suite of Post-Transition Guidances
(Regulatory Focus)

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in September released more than 2 dozen guidance documents explaining how drugs, medical devices, clinical trials, and more will be regulated after the Brexit transition period expires on January 1. Read more.

3. For the First Time, Drug Makers and PBMs Must Jointly Face an Insulin Price Fixing Lawsuit
(Pharmalot)

A federal judge ruled that Harris County, TX, USA, can proceed with a lawsuit accusing several drug makers and pharmacy benefit managers of conspiring to fix prices for insulin. This marks the first time that these companies will have to collectively defend their role in the rising cost of the life-saving diabetes medicine. In a lawsuit filed last year, Harris County officials claimed taxpayers were “fraudulently overcharged” for ongoing and drastic price hikes for a medication that has not substantively changed in many years. From 2013 to 2018, the county maintained it paid $27.5 million for insulin due to an allegedly misleading pricing scheme involving both drug companies and the biggest pharmacy benefit managers. Read more.

4. MA Consults on Using Registry Studies as Real-World Evidence Source (Pink Sheet)

A draft EU guideline explains how disease registries can be used to supplement evidence in the pre-authorization phase and provide infrastructure for post-authorization evidence generation. Read more.

5. Democrats Slam Teva, Celgene Execs for Large Price Hikes
(Fierce Healthcare)

House Democrats tore into several pharmaceutical executives for continuing to raise prices for certain drugs even as they allocated limited funding to research and development. The House Committee on Oversight and Government Reform held the first in a 2-day hearing September 30, 2020 on unsustainable drug prices. The hearing follows a report released by the committee that found drug maker Celgene, now part of Bristol Myers Squibb, raised prices for cancer drug Revlimid multiples times over the years to meet sales targets, and that Teva Pharmaceuticals raised prices for multiple sclerosis drug Copaxone despite low research and development costs. Read more.

6. Drugs Aren’t the Reason the United States Spends So Much on Healthcare
(Kaiser Family Foundation)

Voters care a lot about drug prices, but they’re not the main reason the United States spends so much on healthcare. The United States spends twice as much per person as other wealthy nations, according to a new Peterson-Kaiser Tracker analysis, and hospitals and outpatient care are the primary culprits. Read more.

7. CMS Releases its Annual IPPS Final Rule: Key Updates for Manufacturers (PRMA Consulting)

On September 2, 2020, the Centers for Medicare & Medicaid Services (CMS) released its annual Inpatient Prospective Payment System (IPPS) final rule, which applies to hospital discharges occurring on or after October 1, 2020 and will impact approximately 3200 acute care hospitals across the United States. While the ruling is made up of enormous detail across a 2160-page document, there are several key updates that will have implications on manufacturers in months and years to come. Read more.

8. ICER to Assess Treatment for Alzheimer’s Disease
(ICER)

The Institute for Clinical and Economic Review (ICER) announced September 29, 2020 that it will assess the comparative clinical effectiveness and value of aducanumab (Biogen) for treatment of Alzheimer disease. An FDA decision on aducanumab is expected in early 2021. The assessment will be publicly discussed during a meeting of the California Technology Assessment Forum in May 2021, where the independent evidence review panel will deliberate and vote on evidence presented in ICER’s report. Read more.

9. Pseudo-Understanding: An Analysis of the Dilution of Value in Healthcare
(BMJ Quality & Safety)

Management concepts cycle through healthcare in trends lasting 3-5 years. This may hinder policymakers, healthcare managers, researchers, and clinicians from grasping the intricacies of a management concept and prevent organizations from realizing the potential of these concepts. Researchers sought to characterize how the newest management concept, value-based healthcare, is used and understood in the scientific literature. Read more.

10. Utilizing Patient and Public Involvement in Stated Preference Research in Health: Learning From the Existing Literature and a Case Study
(The Patient–Patient-Centered Outcomes Research)

Publications reporting discrete choice experiments of healthcare interventions rarely discuss whether patient and public involvement activities have been conducted. This paper presents examples from the existing literature and a detailed case study from the National Institute for Health Research-funded PATHWAY program that comprehensively included patient and public involvement activities at multiple stages of preference research. The paper describes different stages at which it is possible to effectively incorporate patient and public involvement across preference research, including the design, recruitment, and dissemination of projects. Read more.