Search

Understanding postoperative recovery is critical for guiding efforts to improve post-acute phase care. How recovery evolves during the first 30 days after cardiac surgery is not well-understood. A digital platform may enable granular quantification of recovery by frequently capturing patient-reported outcome measures (PROM) that can be clinically implemented to support recovery. We conduct a prospective cohort study using a digital platform to measure recovery after cardiac surgery using a PROM sent every 3 days for 30 days after surgery to characterize recovery in multiple domains (e.g., pain, sleep, activities of daily living, anxiety) and to identify factors related to the patient's perception of overall recovery. We enroll patients who underwent cardiac surgery at a tertiary center between January 2019 and March 2020 and automatically deliver PROMs and reminders electronically. Of the 10 surveys delivered per patient, 8 (IQR 6–10) are completed. Patients who experienced postoperative complications more commonly belong to the worst overall recovery trajectory. Of the 12 domains modeled, only the worst anxiety trajectory is associated with the worse overall recovery trajectory membership, suggesting that even when patients struggle in the recovery of other domains, the patient may still feel progress in their recovery. We demonstrate that using a digital platform, automated PROM data collection, and characterization of multi-domain recovery trajectories is feasible and likely implementable in clinical practice. Overall recovery may be impacted by complications, while slow progress in constituent domains may still allow for the perception of overall recovery progression. [ABSTRACT FROM AUTHOR]

Background: Drugs and high-risk medical devices are increasingly likely to receive Food and Drug Administration (FDA) approval through expedited pathways, which has implications for informed treatment consent (i.e., consent in clinical practice).Objective: To obtain expert opinion about the clinical and ethical implications of the increasing availability of new drugs and devices approved through expedited development and regulatory review pathways.Design: Qualitative study using individual semi-structured videoconference interviews.Participants: National leaders in medicine, ethics, and law (n=12) with expertise in medical product regulation, payor policymaking, bioethics, physician practice, patient advocacy, public health expertise/advocacy, clinical trials, the pharmaceutical and device industry, institutional review board oversight, and real-world evidence.Main Measures: Principal themes in 3 domains: expedited regulatory pathways, physician and patient understanding of and reliance on FDA approval, and informed treatment consent.Key Results: Respondents pointed out that more common use of expedited pathways translates to increased reliance on surrogate measures, some with uncertain clinical significance. While expedited development and review can have advantages, participants expressed worry that physicians were unaware when medical products were expedited and did not communicate about uncertainties in knowledge about new drug or device approvals effectively with patients. Many participants felt that informed treatment consent discussions about new drugs or devices should include some explanations of expedited pathways and use of surrogate measures.Conclusions: Experts identified advantages of expediting development and of FDA flexibility in applying its standards to new drugs and medical devices, but highlighted concerns that patients may not be adequately informed about the risks of shorter review times or about uncertainties in the evidence that result. There is a need to identify approaches to ensure effective clinical use of drugs and devices when approved through expedited pathways. [ABSTRACT FROM AUTHOR]

OBJECTIVE To understand if and how 2016 Medicare prescription drug plan formularies incentivize selection of brand-name drugs without bioequivalent generics compared to their corresponding therapeutically interchangeable generic drugs through tier placement and utilization management. At least one Medicare formulary covered both the brand-name and corresponding therapeutically interchangeable generic for 23 drugs (95.8%; ranolazine was not covered by any formulary), although the median proportion of formularies providing no brand-name drug coverage was 42.4% (IQR, 2.5-71.5). Nonetheless, our findings can inform the proposed 2020 indication-based formulary design, suggesting that restricted coverage of brand-name drugs and favored coverage of their therapeutically interchangeable generics might further incentivize the use of generic drugs and potentially reduce both Medicare and beneficiary spending. [Extracted from the article]

Importance: High-risk medical devices often undergo modifications, which are approved by the US Food and Drug Administration (FDA) through various kinds of premarket approval (PMA) supplements. There have been multiple high-profile recalls of devices approved as PMA supplements.Objective: To characterize the quality of the clinical studies and data (strength of evidence) used to support FDA approval of panel-track supplements (a type of PMA supplement pathway that is used for significant changes in a device or indication for use and always requires clinical data).Design and Setting: Descriptive study of clinical studies supporting panel-track supplements approved by the FDA between April 19, 2006, and October 9, 2015.Exposure: Panel-track supplement approval.Main Outcomes and Measures: Methodological quality of studies including randomization, blinding, type of controls, clinical vs surrogate primary end points, use of post hoc analyses, and reporting of age and sex.Results: Eighty-three clinical studies supported the approval of 78 panel-track supplements, with 71 panel-track supplements (91%) supported by a single study. Of the 83 studies, 37 (45%) were randomized clinical trials and 25 (30%) were blinded. The median number of patients per study was 185 (interquartile range, 75-305), and the median follow-up duration was 180 days (interquartile range, 84-270 days). There were a total of 150 primary end points (mean [SD], 1.8 [1.2] per study), and 57 primary end points (38%) were compared with controls. Of primary end points with controls, 6 (11%) were retrospective controls and 51 (89%) were active controls. One hundred twenty-one primary end points (81%) were surrogate end points. Thirty-three studies (40%) did not report age and 25 (30%) did not report sex for all enrolled patients. The FDA required postapproval studies for 29 of 78 (37%) panel-track supplements.Conclusions and Relevance: Among clinical studies used to support FDA approval of high-risk medical device modifications, fewer than half were randomized, blinded, or controlled, and most primary outcomes were based on surrogate end points. These findings suggest that the quality of studies and data evaluated to support approval by the FDA of modifications of high-risk devices should be improved. [ABSTRACT FROM AUTHOR]

The article presents a study to compare the Medicare beneficiary out-of-pocket costs through prescription drug plans (PDPs) with generic drug discount program's (GDDP) $4 cash prices. The comparison focuses on 30-day supplies of generic medications used to treat cardiovascular disease (CVD)-related conditions. It offers information about the September 2017 GDDP list from Wal-Mart Stores Inc. and identifies the generic medications used to treat seven prevalent CVD-related conditions.

The article focuses on a study which characterized the prevalence and extent of data reporting from training patients various cardiovascular device research submitted for the approval by the U.S. Food and Drug Administration (FDA). The researchers utilized the abstracted information from the Summaries of Safety and Effectiveness Data published in the Web site of the FDA. It is suggested that guidelines on the use and inclusion of results for training patients can improve accuracy on results reporting.

The article discusses a research study on gender bias in studies for cardiovascular devices that received premarket approval from the U.S. Food and Drug Administration (FDA). The researchers reviewed the demographics, comments on gender bias and analysis of results by sex for such devices between 2000 and 2007. They noted the lack of sex-specific safety and effectiveness data for high-risk cardiovascular devices before FDA approval.

The article discusses a study which investigated premarket approval (PMA) of cardiovascular devices by the U.S. Food and Drug Administration (FDA). The study also attempted to characterize the type and strength of evidence of which the FDA review process is based. The authors systematically reviewed summaries of safety and effectiveness data for 78 PMA for high-risk cardiovascular devices approved between January 2000 and December 2007. They concluded that premarket approval was often based on studies that lack adequate strength and may be prone to bias.

The article presents a study on the changes between clinical trial participants and Medicare beneficiaries in evidence used for Medicare national coverage decisions. It seeks to identify the applicability of the clinical trials depend on by the Centers for Medicaid and Medicare Services (CMS) to its beneficiaries. Results showed the significant difference between Medicare beneficiaries and the cardiovascular clinical trial participants used inform Medicare coverage decisions. Apart from this, the need for further information on Medicare beneficiaries and coverage decisions is highlighted.

The article discusses lessons from the U.S. government regulation of the Wingspan intracranial stent system. A background on how the Wingspan got to market is presented. An overview of the Stenting and Aggresive Medical Management for Preventing Recurrent Stroke in Intracranial Stenosis (SAMMPRIS) trial is provided. The reaction of the U.S. Food and Drug Administration (FDA) to the petition of the consumer advocacy organization Public Citizen is discussed. INSET: THE HUMANITARIAN DEVICE PROGRAMME: WHAT IT MEANS FOR THE US....

A letter to the editor is presented in response to the article "Strength of Study Evidence Examined by the FDA in Premarket Approval of Cardiovascular Devices," by S. S. Dhruva and colleagues in the 2009 issue, along with a response from the authors of the article.

The authors comment on the decision of the U.S. Centers for Medicare and Medicaid Services (CMS) to deny coverage of computed tomographic (CT) colonography for screening cancer. They hope that the strict application of evidence-based analysis would lead to a shift in its approach to national coverage decisions. They add that their optimism is cautious, the pressure of which will be inevitably on the CMS. They also mention that colorectal cancer screening is a procedure for which the CMS is particularly authorized to consider costs.

The authors discuss the study, How Does Medical Device Regulation Perform in the U.S. and EU? A Systematic Review, by Daniel B. Kramer and colleagues. They state that the research examines the strengths and weaknesses associated with approaches to medical device regulation in the U.S. and European Union (EU). They suggest that regulatory agencies should have sufficient funding and political backing in order to conduct thorough reviews of medical devices and ensure their safety and efficacy.

The author discusses the draft guidance released by the U.S. Food and Drug Administration in December 2011, which talks about the underrepresentation of women in medical device clinical trials and offers recommendations for ensuring adequate enrollment of women in these trials. He comments on the National Institute of Health's experience with gender bias guidances which indicates the importance of enforcement, tracking and consequences for noncompliance.

The article discusses the dangers posed by the medical device misclassifications by the U.S. Food and Drug Administration (FDA). It details how medical devices are divided into three classes by the FDA and the processes by which the agency approves medical device recalls. It reveals that more high-risk devices are cleared through the 501(k) than through the original pre-market approval (PMA) which is the required process of scientific review to ensure product safety and effectiveness.

A letter to the editor is presented in response to an article by James D. Chambers and co-authors on the coverage of interventions by the U.S. Centers for Medicare and Medicaid Services (CMS) in the February 2015 issue.

A letter to the editor is presented in response to the article written by Samantha Roberts and coauthors in the July 2011 issue which discusses the available cancer medications in the U.S.

Background: Beta-blockers, angiotensin receptor-neprilysin inhibitor (ARNI), mineralocorticoid receptor antagonists, and sodium-glucose cotransporter-2 inhibitors (SGLT2i), known as quadruple therapy, are recommended for patients with heart failure with reduced ejection fraction (HFrEF).Objectives: This study sought to determine Medicare coverage and out-of-pocket (OOP) costs of quadruple therapy and regimens excluding ARNI or SGLT2i.Methods: This study assessed cost sharing, prior authorization, and step therapy in all 4,068 Medicare prescription drug plans in 2020. OOP costs were determined during the standard coverage period and annually based on the Medicare Part D standard benefit, inclusive of deductible, standard coverage, coverage gap, and catastrophic coverage.Results: Tier ≥3 cost sharing was required by 99.1% of plans for ARNI and 98.5% for at least 1 SGLT2i. Only ARNI required prior authorization (24.3% of plans), and step therapy was required only for SGLT2is (5.4%) and eplerenone (0.8%). The median 30-day standard coverage OOP cost of quadruple therapy was $94 (IQR: $84-$100), including $47 (IQR: $40-$47) for ARNI and $45 (IQR: $40-$47) for SGLT2i. The median annual OOP cost of quadruple therapy was $2,217 (IQR: $1,956-$2,579) compared with $1,319 (IQR: $1,067-$1,675) when excluding SGLT2i and $1,322 (IQR: $1,025-$1,588) when including SGLT2i and substituting an angiotensin-converting enzyme inhibitor or angiotensin receptor blocker for ARNI. The median 30-day OOP cost of generic regimens was $3 (IQR: $0-$9).Conclusions: Medicare drug plans restrict coverage of quadruple therapy through cost sharing, with OOP costs that are substantially higher than generic regimens. Quadruple therapy may be unaffordable for many Medicare patients with HFrEF unless medication prices and cost sharing are reduced. [ABSTRACT FROM AUTHOR]

The article reports that the Centers for Medicare and Medicaid Services (CMS) finalized its national coverage determination for aducanumab (Aduhelm) and other monoclonal antibodies for Alzheimer's disease. Topics include considered Aducanumab was approved by the Food and Drug Administration (FDA) on the basis of a post hoc analysis of one trial which had been terminated for futility.

Objective: Implantable loop recorders (ILRs) are increasingly used for long-term rhythm monitoring after ischaemic and cryptogenic stroke, with the goal of detecting atrial fibrillation (AF) and subsequent initiation of oral anticoagulation to reduce risk of adverse clinical outcomes. There is a need to determine the effectiveness of different rhythm monitoring strategies in this context., Methods: We conducted a retrospective cohort analysis of individuals with commercial and Medicare Advantage insurance in Optum Labs Data Warehouse who had incident ischaemic or cryptogenic stroke and no prior cardiovascular implantable electronic device from 1 January 2016 to 30 June 2021. Patients were stratified by rhythm monitoring strategy: ILR, long-term continuous external cardiac monitor (>48 hours to 30 days) or Holter monitor (≤48 hours). The primary outcome was risk-adjusted all-cause mortality at 12 months. Secondary outcomes included new diagnosis of AF and oral anticoagulation, bleeding, and costs., Results: Among 48 901 patients with ischaemic or cryptogenic stroke, 9235 received an ILR, 29 103 long-term continuous external monitor and 10 563 Holter monitor only. Mean age was 69.9 (SD 11.9) years and 53.5% were female. During the 12-month follow-up period, patients who received ILRs compared with those who received long-term continuous external monitors had a higher odds of new diagnosis of AF and oral anticoagulant initiation (adjusted OR 2.27, 95% CI 2.09 to 2.48). Compared with patients who received long-term continuous external monitors, those who received ILRs had similar 12-month mortality (HR 1.00; 95% CI 0.89 to 1.12), with approximately $13 000 higher costs at baseline (including monitor cost) and $2500 higher costs during 12-month follow-up., Conclusions: In this large real-world study of patients with ischaemic or cryptogenic stroke, ILR placement resulted in more diagnosis of AF and initiation of oral anticoagulation, but no difference in mortality compared with long-term continuous external monitors., Competing Interests: Competing interests: SSD also receives research support from the Department of Veterans Affairs. SSD and RFR report serving on the Institute for Clinical and Economic Review (ICER) California Technology Assessment Forum. JM is a full-time employee of UnitedHealth Group and OA and KC (currently) and CEC (previously) are full-time employees of Optum Health, an arm of UnitedHealth Group and own stock in the company. OA had full access to all the data in the study and takes responsibility for the integrity of the data and the accuracy of the data analysis., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Background: Remote monitoring (RM) of pacemakers and implantable cardioverter-defibrillators (ICDs) is a Class 1, Level of Evidence A recommendation because of its multitude of clinical benefits. However, RM adherence rates are suboptimal, precluding patients from achieving these benefits. There is a need for direct-to-patient efforts to improve adherence., Methods: In this national randomized, controlled trial conducted in the Veterans Health Administration (VHA), 2120 patients with a pacemaker or ICD who had not sent an RM transmission for ≥1 year (and usually ≥3 years) while under VHA care for their device were randomly assigned to be mailed a postcard (n = 1076) or a detailed letter (n = 1044). The postcard described what RM does and its key benefits (reduced mortality and fewer in-person visits). The letter provided a similar message but included more details about RM benefits and the process. The primary outcome was an RM transmission sent within 90 days of mailing, and a secondary outcome was an RM transmission sent within 365 days., Results: The primary outcome was achieved in 121 (11.3%) in the postcard and 96 patients (9.2%) in the letter group (p = .12). The secondary outcome was achieved in 266 (24.7%) and 239 (22.9%), respectively (p = .32)., Conclusions: This randomized trial showed no significant difference in the proportion of chronically non-adherent patients who sent an RM transmission after receiving a low-cost postcard or a detailed, higher-cost letter encouraging their participation in RM. However, as only a minority of patients responded to either, further work is needed to engage patients in the life-saving benefits of RM., (Published 2023. This article is a U.S. Government work and is in the public domain in the USA.)

Objectives: To quantify differences in prices paid and procedural complications incurred in hospital outpatient departments (HOPDs) and freestanding ambulatory surgery centers (ASCs)., Study Design: Observational study using deidentified 2019-2020 insurance claims from Blue Cross Blue Shield insurance plans nationally, with information on prices paid and complications incurred for colonoscopy, knee or shoulder arthroscopy, and cataract removal surgery., Methods: The data include 1,662,183 patients who received a colonoscopy, 53.5% of whom were treated in HOPDs; 259,200 patients who underwent arthroscopy, 61.0% of whom were treated in HOPDs; and 173,664 patients who had cataract removal surgery, 34.7% of whom were treated in HOPDs. Multivariable linear regression methods were used to identify the associations between HOPD and ASC site of care, prices, and complications after adjusting for patient demographics, risk, and geographic market location., Results: After adjusting for patient characteristics, risk, and geographic market location, prices paid in HOPDs were 54.9% higher than those charged in ASCs for colonoscopy (95% CI, 53.6%-56.1%), 44.4% higher for arthroscopy (95% CI, 43.0%-45.8%), and 44.0% higher for cataract removal surgery (95% CI, 42.9%-45.5%). Adjusted rates of complications were slightly higher in HOPDs than ASCs for colonoscopy over a 90-day interval but similar over the 7- and 30-day intervals. Rates were statistically and clinically similar between the 2 sites of care for arthroscopy and cataract removal., Conclusions: The higher prices charged in HOPDs for the 3 ambulatory procedures were not balanced by better quality-as measured by rates of procedural complications-compared with procedures performed in nonhospital ASCs.

Background: Hospitals can leverage their position between the ultimate buyers and sellers of drugs to retain a substantial share of insurer pharmaceutical expenditures., Methods: In this study, we used 2020-2021 national Blue Cross Blue Shield claims data regarding patients in the United States who had drug-infusion visits for oncologic conditions, inflammatory conditions, or blood-cell deficiency disorders. Markups of the reimbursement prices were measured in terms of amounts paid by Blue Cross Blue Shield plans to hospitals and physician practices relative to the amounts paid by these providers to drug manufacturers. Acquisition-price reductions in hospital payments to drug manufacturers were measured in terms of discounts under the federal 340B Drug Pricing Program. We estimated the percentage of Blue Cross Blue Shield drug spending that was received by drug manufacturers and the percentage retained by provider organizations., Results: The study included 404,443 patients in the United States who had 4,727,189 drug-infusion visits. The median price markup (defined as the ratio of the reimbursement price to the acquisition price) for hospitals eligible for 340B discounts was 3.08 (interquartile range, 1.87 to 6.38). After adjustment for drug, patient, and geographic factors, price markups at hospitals eligible for 340B discounts were 6.59 times (95% confidence interval [CI], 6.02 to 7.16) as high as those in independent physician practices, and price markups at noneligible hospitals were 4.34 times (95% CI, 3.77 to 4.90) as high as those in physician practices. Hospitals eligible for 340B discounts retained 64.3% of insurer drug expenditures, whereas hospitals not eligible for 340B discounts retained 44.8% and independent physician practices retained 19.1%., Conclusions: This study showed that hospitals imposed large price markups and retained a substantial share of total insurer spending on physician-administered drugs for patients with private insurance. The effects were especially large for hospitals eligible for discounts under the federal 340B Drug Pricing Program on acquisition costs paid to manufacturers. (Funded by Arnold Ventures and the National Institute for Health Care Management.)., (Copyright © 2024 Massachusetts Medical Society.)

Physicians' knowledge of Food and Drug Administration (FDA) approval processes is important in informing clinical decisions and patient discussions. Among a randomly selected national sample of 509 internists, cardiologists, and oncologists, 41 percent reported moderate or better understanding of the FDA's drug approval process, and 17 percent reported moderate or better understanding of the FDA's medical device approval process. Nearly all physicians thought that randomized, blinded trials that met primary endpoints should be very important factors required to secure regulatory approval. Also, nearly all physicians thought that the FDA should revoke approval for accelerated-approval drugs or breakthrough devices that did not show benefit in postapproval studies. Our findings suggest that physicians commonly lack familiarity with drug and medical device regulatory practices and are under the impression that the data supporting FDA drug and high-risk device approvals are more rigorous than they often are. Physicians would value more rigorous premarket evidence, as well as regulatory action for drugs and devices that do not demonstrate safety and effectiveness in the postmarket setting.

Importance: The US Food and Drug Administration (FDA) and Centers for Medicare & Medicaid Services (CMS) have different statutory authorities; FDA evaluates safety and effectiveness for market authorization of medical devices while CMS determines whether coverage is "reasonable and necessary" for its beneficiaries. CMS has recently enacted policies automatically providing supplemental reimbursement for new, costly devices authorized after designation in FDA's Breakthrough Devices Program (BDP) and in June 2023 issued notice for a new Transitional Coverage for Emerging Technologies pathway, accelerating coverage for Breakthrough devices., Observations: Aiming to incentivize innovation, FDA awards Breakthrough designations early in device development to expedite market authorization and can accept greater uncertainty in benefit and risk, contingent on postmarket evidence generation. Since 2020, Breakthrough designation has effectively automatically qualified devices to receive supplemental Medicare reimbursement after CMS waived a long-standing requirement that devices demonstrate "substantial clinical improvement" for beneficiaries. Using publicly available information, 3 examples of cardiovascular devices illustrate that the BDP may allow for FDA authorization based on less rigorous evidence, such as single-arm trials focused on surrogate end points with short-term follow-up whose participants are often not representative of Medicare beneficiaries. In 1 case, Breakthrough designation allowed a 30% decrease in enrollment of a trial used to support approval. Initial positive findings for some devices have remained unverified, and in 1 case even partially nullified, by postmarket studies. Manufacturers have also used Breakthrough designations to set the price of devices to facilitate additional pass-through payments, leading to higher short-term and long-term costs to CMS and health care systems., Conclusions and Relevance: The BDP may qualify new, costly devices for higher and automatic Medicare reimbursement despite evidence not being representative of CMS beneficiaries and persistent uncertainty of benefit and risk. To ensure the best evidence is generated to inform clinical care, FDA could apply more selectivity to BDP eligibility, specify objective criteria for revoking Breakthrough designation when appropriate, and ensure timely postmarket evidence generation, whereas CMS could independently review clinical evidence, advise manufacturers about standards for coverage review, and make supplemental payments and long-term device reimbursement contingent on clinical outcome benefit and postmarket evidence generation.

In June 2021, the US Food and Drug Administration (FDA) granted accelerated approval to aducanumab, a monoclonal antibody indicated for the treatment of Alzheimer's disease. The accelerated approval decision was controversial due to concerns about the use of an unvalidated surrogate measure, beta-amyloid, as the basis for approval and a lack of clinical outcome benefit. Between October 2021 and September 2022, we conducted a survey of a nationally representative group of internists, medical oncologists, and cardiologists to understand perspectives around aducanumab's approval and how this FDA decision may influence trust in other drugs approved through the accelerated approval program. Among 214 physician respondents familiar with the accelerated approval of aducanumab, 184 (86%) would not prescribe or recommend aducanumab. Further, 143 (67%) physicians reported losing trust in other drugs approved through the accelerated approval program due to the FDA's decision with aducanumab. As a growing number of similar novel Alzheimer's disease treatments are on the horizon, the first of which, lecanemab, already has received accelerated approval in January 2023, our survey findings provide insight into the impact of the FDA's regulatory decisions on the perspectives and prescribing behavior of physicians concerning these novel drug treatments., (© 2023 The Authors. Clinical Pharmacology & Therapeutics © 2023 American Society for Clinical Pharmacology and Therapeutics.)

Background: Current methods to identify cardiovascular implantable electronic device lead failure include postapproval studies, which may be limited in scope, participant numbers, and attrition; studies relying on administrative codes, which lack specificity; and voluntary adverse event reporting, which cannot determine incidence or attribution to the lead., Objective: The purpose of this study was to determine whether adjudicated remote monitoring (RM) data can address these limitations and augment lead safety evaluation., Methods: Among 48,191 actively monitored patients with a cardiovascular implantable electronic device, we identified RM transmissions signifying incident lead abnormalities and, separately, identified all leads abandoned or extracted between April 1, 2019, and April 1, 2021. We queried electronic health record and Medicare fee-for-service claims data to determine whether patients had administrative codes for lead failure. We verified lead failure through manual electronic health record review., Results: Of the 48,191 patients, 1170 (2.4%) had incident lead abnormalities detected by RM. Of these, 409 patients had administrative codes for lead failure, and 233 of these 409 patients (57.0%) had structural lead failure verified through chart review. Of the 761 patients without administrative codes, 167 (21.9%) had structural lead failure verified through chart review. Thus, 400 patients with RM transmissions suggestive of lead abnormalities (34.2%) had structural lead failure. In addition, 200 patients without preceding abnormal RM transmissions had leads abandoned or extracted for structural failure, making the total lead failure cohort 600 patients (66.7% with RM abnormalities, 33.3% without). Patients with isolated right atrial or left ventricular lead failure were less likely to have lead replacement and administrative codes reflective of lead failure., Conclusion: RM may strengthen real-world assessment of lead failure, particularly for leads where patients do not undergo replacement., (Published by Elsevier Inc.)

Importance: In the US, nearly all medical devices progress to market under the 510(k) pathway, which uses previously authorized devices (predicates) to support new authorizations. Current regulations permit manufacturers to use devices subject to a Class I recall-the FDA's most serious designation indicating a high probability of adverse health consequences or death-as predicates for new devices. The consequences for patient safety are not known., Objective: To determine the risk of a future Class I recall associated with using a recalled device as a predicate device in the 510(k) pathway., Design and Setting: In this cross-sectional study, all 510(k) devices subject to Class I recalls from January 2017 through December 2021 (index devices) were identified from the FDA's annual recall listings. Information about predicate devices was extracted from the Devices@FDA database. Devices authorized using index devices as predicates (descendants) were identified using a regulatory intelligence platform. A matched cohort of predicates was constructed to assess the future recall risk from using a predicate device with a Class I recall., Main Outcomes and Measures: Devices were characterized by their regulatory history and recall history. Risk ratios (RRs) were calculated to compare the risk of future Class I recalls between devices descended from predicates with matched controls., Results: Of 156 index devices subject to Class I recall from 2017 through 2021, 44 (28.2%) had prior Class I recalls. Predicates were identified for 127 index devices, with 56 (44.1%) using predicates with a Class I recall. One hundred four index devices were also used as predicates to support the authorization of 265 descendant devices, with 50 index devices (48.1%) authorizing a descendant with a Class I recall. Compared with matched controls, devices authorized using predicates with Class I recalls had a higher risk of subsequent Class I recall (6.40 [95% CI, 3.59-11.40]; P<.001)., Conclusions and Relevance: Many 510(k) devices subjected to Class I recalls in the US use predicates with a known history of Class I recalls. These devices have substantially higher risk of a subsequent Class I recall. Safeguards for the 510(k) pathway are needed to prevent problematic predicate selection and ensure patient safety.

Importance: Spinal cord stimulators (SCSs) are increasingly used for the treatment of chronic pain. There is a need for studies with long-term follow-up., Objective: To determine the comparative effectiveness and costs of SCSs compared with conventional medical management (CMM) in a large cohort of patients with chronic pain., Design, Setting, and Participants: This was a 1:5 propensity-matched retrospective comparative effectiveness research analysis of insured individuals from April 1, 2016, to August 31, 2018. This study used administrative claims data, including longitudinal medical and pharmacy claims, from US commercial and Medicare Advantage enrollees 18 years or older in Optum Labs Data Warehouse. Patients with incident diagnosis codes for failed back surgery syndrome, complex regional pain syndrome, chronic pain syndrome, and other chronic postsurgical back and extremity pain were included in this study. Data were analyzed from February 1, 2021, to August 31, 2022., Exposures: SCSs or CMM., Main Outcomes and Measures: Surrogate measures for primary chronic pain treatment modalities, including pharmacologic and nonpharmacologic pain interventions (epidural and facet corticosteroid injections, radiofrequency ablation, and spine surgery), as well as total costs., Results: In the propensity-matched population of 7560 patients, mean (SD) age was 63.5 (12.5) years, 3080 (40.7%) were male, and 4480 (59.3%) were female. Among matched patients, during the first 12 months, patients treated with SCSs had higher odds of chronic opioid use (adjusted odds ratio [aOR], 1.14; 95% CI, 1.01-1.29) compared with patients treated with CMM but lower odds of epidural and facet corticosteroid injections (aOR, 0.44; 95% CI, 0.39-0.51), radiofrequency ablation (aOR, 0.57; 95% CI, 0.44-0.72), and spine surgery (aOR, 0.72; 95% CI, 0.61-0.85). During months 13 to 24, there was no significant difference in chronic opioid use (aOR, 1.06; 95% CI, 0.94-1.20), epidural and facet corticosteroid injections (aOR, 1.00; 95% CI, 0.87-1.14), radiofrequency ablation (aOR, 0.84; 95% CI, 0.66-1.09), or spine surgery (aOR, 0.91; 95% CI, 0.75-1.09) with SCS use compared with CMM. Overall, 226 of 1260 patients (17.9%) treated with SCS experienced SCS-related complications within 2 years, and 279 of 1260 patients (22.1%) had device revisions and/or removals, which were not always for complications. Total costs of care in the first year were $39 000 higher with SCS than CMM and similar between SCS and CMM in the second year., Conclusions and Relevance: In this large, real-world, comparative effectiveness research study comparing SCS and CMM for chronic pain, SCS placement was not associated with a reduction in opioid use or nonpharmacologic pain interventions at 2 years. SCS was associated with higher costs, and SCS-related complications were common.

Policy Points Low-value care is common in clinical practice, leading to patient harm and wasted spending. Much of this low-value care stems from the use of medical device-based procedures. We describe here a novel academic-policymaker collaboration in which evidence-based clinical coverage for device-based procedures is implemented through prior authorization-based policies for Louisiana's Medicaid beneficiary population. This process involves eight steps: 1) identifying low-value medical device-based procedures based on clinical evidence review, 2) quantifying utilization and reimbursement, 3) reviewing clinical coverage policies to identify opportunities to align coverage with evidence, 4) using a low-value device selection index, 5) developing an evidence synthesis and policy proposal, 6) stakeholder engagement and input, 7) policy implementation, and 8) policy evaluation. This strategy holds significant potential to reduce low-value device-based care., (© 2022 Milbank Memorial Fund.)

Background: Percutaneous ventricular assist devices (PVADs) have been replacing intra-aortic balloon pumps for hemodynamic support during percutaneous coronary intervention (PCI), even though data supporting a benefit for hard clinical end points remain limited. We evaluated diffusion of PVADs across US markets and examined the association of market utilization of PVAD with mortality and cost., Methods: Using the 2013 to 2019 Medicare data, we identified all patients aged ≥65 years who underwent PCI with either a PVAD or intra-aortic balloon pump. We used hospital referral region to define regional health care markets and categorized them in quartiles based on the proportional use of PVADs during PCI. Multilevel models were constructed to determine the association of patient, hospital, and market factors with utilization of PVADs and the association of PVAD utilization with 30-day mortality and cost., Results: A total of 79 176 patients underwent PCI with either intra-aortic balloon pump (47 514 [60.0%]) or PVAD (31 662 [40.0%]). The proportion of PCI procedures with PVAD increased over time (17% in 2013 to 57% in 2019; P for trend, <0.001), such that PVADs overtook intra-aortic balloon pump for hemodynamic support during PCI in 2018. There was a large variation in PVAD utilization across markets (range, 0%-85%), which remained unchanged after adjustment of patient characteristics (median odds ratio, 2.05 [95% CI, 1.91-2.17]). The presence of acute myocardial infarction, cardiogenic shock, and emergent status was associated with a 45% to 50% lower odds of PVAD use suggesting that PVADs were less likely to be used in the sickest patients. Greater utilization of PVAD at the market level was not associated with lower risk mortality but was associated with higher cost., Conclusions: Although utilization of PVADs for PCI continues to increase, there is large variation in PVAD utilization across markets. Greater market utilization of PVADs was not associated with lower mortality but was associated with higher cost.

Prescription drugs and medical devices are increasingly coming to market through expedited US Food and Drug Administration (FDA) pathways that require only limited evidence of safety and efficacy, such as nonrandomized, unblinded trial data in small numbers of patients, or the use of surrogate end points. Reliance on more limited evidence means that there is often greater uncertainty about risks and benefits. Using a modified Delphi process, we sought to identify promising policy approaches that address physician-patient decision-making needs about the use of such drugs and medical devices. We convened 13 national leaders from academia, government, nonprofits, payors, and industry who had expertise in medical product regulation, payor policymaking, bioethics, physician practice, patient advocacy, public health expertise/advocacy, clinical trials, the pharmaceutical and device industry, institutional review board oversight, and real-world evidence. Through multiple rounds of voting and meetings focused on evaluating the feasibility and impact of various interventions, the 13 participants reached the broadest consensus on 4 interventions: strengthening FDA post-approval study requirements to ensure postmarket evidence is generated in a timely manner, better informing patients about the risks and benefits and level of evidence supporting therapies via simplified and patient-centered product information "boxes" modeled on nutrition labels, limiting prices for drugs and medical devices approved based on surrogate end point data until confirmatory clinical evidence is generated, and improving health professional education about FDA regulation to better support clinician use of drugs and devices as well as communication with patients., (© 2022 The Authors. Clinical Pharmacology & Therapeutics © 2022 American Society for Clinical Pharmacology and Therapeutics.)

Importance: Most adults 65 years or older have multiple chronic conditions. Managing these conditions with prescription drugs can be costly, particularly for older adults with limited incomes., Objective: To estimate hypothetical out-of-pocket costs associated with guideline-recommended outpatient medications for the initial treatment of 8 common chronic diseases among older adults with Medicare prescription drug plans (PDPs)., Design, Setting, and Participants: This retrospective cross-sectional study used 2009 and 2019 Medicare prescription drug plan formulary files to estimate annual out-of-pocket costs among hypothetical patients enrolled in Medicare Advantage or stand-alone Medicare Part D plans. A total of 3599 PDPs in 2009 and 3618 PDPs in 2019 were included after inclusion and exclusion criteria were applied. Costs associated with guideline-recommended medications for 8 of the most common chronic diseases (atrial fibrillation, chronic obstructive pulmonary disease [COPD], heart failure with reduced ejection fraction, hypercholesterolemia, hypertension, osteoarthritis, osteoporosis, and type 2 diabetes), alone and in 2 clusters of commonly comorbid conditions, were examined., Main Outcomes and Measures: Annual out-of-pocket costs for each chronic condition, inflation adjusted to 2019 dollars., Results: Among 3599 Medicare PDPs in 2009, 1998 were Medicare Advantage plans and 1601 were stand-alone plans; among 3618 Medicare PDPs in 2019, 2719 were Medicare Advantage plans and 899 were stand-alone plans. For an older adult enrolled in any Medicare PDP in 2019, the median annual out-of-pocket costs for individual conditions varied, from a minimum of $32 (IQR, $6-$48) for guideline-recommended management of osteoporosis (a decrease from $128 [IQR, $102-$183] in 2009) to a maximum of $1579 (IQR, $1524-$2229) for guideline-recommended management of atrial fibrillation (an increase from $91 [IQR, $73-$124] in 2009). For an older adult with a cluster of 5 commonly comorbid conditions (COPD, hypertension, osteoarthritis, osteoporosis, and type 2 diabetes) enrolled in any PDP, the median out-of-pocket cost in 2019 was $1999 (IQR, $1630-$2564), a 12% decrease from $2284 (IQR, $1920-$3107) in 2009. For an older adult with all 8 chronic conditions (atrial fibrillation, COPD, diabetes, hypercholesterolemia, heart failure, hypertension, osteoarthritis, and osteoporosis) enrolled in any PDP, the median out-of-pocket cost in 2019 was $3630 (IQR, $3234-$5197), a 41% increase from $2571 (IQR, $2185-$3719) in 2009., Conclusions and Relevance: In this cross-sectional study, out-of-pocket costs for guideline-recommended outpatient medications for the initial treatment of 8 common chronic diseases varied by condition. Although costs generally decreased between 2009 and 2019, particularly with regard to conditions for which generic drugs were available, out-of-pocket costs remained high and may have presented a substantial financial burden for Medicare beneficiaries, especially older adults with conditions for which brand-name drugs were guideline recommended.

Importance: With a growing interest in the use of real-world evidence for regulatory decision-making, it is important to understand whether real-world data can be used to emulate the results of randomized clinical trials., Objective: To use electronic health record and administrative claims data to emulate the ongoing PRONOUNCE trial (A Trial Comparing Cardiovascular Safety of Degarelix Versus Leuprolide in Patients With Advanced Prostate Cancer and Cardiovascular Disease)., Design, Setting, and Participants: This retrospective, propensity-matched cohort study included adult men with a diagnosis of prostate cancer and cardiovascular disease who initiated either degarelix or leuprolide between December 24, 2008, and June 30, 2019. Participants were commercially insured individuals and Medicare Advantage beneficiaries included in a large US administrative claims database., Exposures: Degarelix or leuprolide., Main Outcomes and Measures: The primary end point was time to first occurrence of a major adverse cardiovascular event (MACE), defined as death due to any cause, myocardial infarction, or stroke, analogous to the PRONOUNCE trial. Secondary end points were time to death due to any cause, myocardial infarction, stroke, and angina. Cox proportional hazards regression was used to evaluate primary and secondary end points., Results: A total of 32 172 men initiated degarelix or leuprolide for prostate cancer; of them, 9490 (29.5%) had cardiovascular disease, and 7800 (24.2%) met the PRONOUNCE trial eligibility criteria and were included in this study. Overall, 165 participants (2.1%) were Asian, 1390 (17.8%) were Black, 663 (8.5%) were Hispanic, and 5258 (67.4%) were White. The mean (SD) age was 74.4 (7.4) years. Among 2226 propensity score-matched patients, no significant difference was observed in the risk of MACE for patients taking degarelix vs those taking leuprolide (10.18 vs 8.60 events per 100 person-years; hazard ratio [HR], 1.18; 95% CI, 0.86-1.61). Degarelix was associated with a higher risk of death from any cause (HR, 1.48; 95% CI, 1.01-2.18) but not of myocardial infarction (HR, 1.16; 95% CI, 0.60-2.25), stroke (HR, 0.92; 95% CI, 0.45-1.85), or angina (HR, 1.36; 95% CI, 0.43-4.27)., Conclusions and Relevance: In this emulation of a clinical trial of men with cardiovascular disease undergoing treatment for prostate cancer, degarelix was not associated with a lower risk of cardiovascular events than leuprolide. Comparison of these data with PRONOUNCE trial results, when published, will help enhance our understanding of the appropriate role of using real-world data to emulate clinical trials.