Using the quality of health economic studies instrument to assess pharmacoeconomic studies evaluating recent food and drug administration drug approvals

Individuals with dialysis-dependent kidney failure experience considerable disease- and treatment-related decline in functional status and overall well-being. Despite these experiences, there have been few substantive technological advances in KRT in decades. As such, new federal initiatives seek to accelerate innovation. Historically, integration of patient perspectives into KRT product development has been limited. However, the US Food and Drug Administration recognizes the importance of incorporating patient perspectives into the total product life cycle (i.e., from product conception to postmarket surveillance) and encourages the consideration of patient-reported outcomes in regulatory-focused clinical trials when appropriate. Recognizing the significance of identifying patient-reported outcome measures (PROMs) that capture contemporary patient priorities, the Kidney Health Initiative, a public–private partnership between the American Society of Nephrology and US Food and Drug Administration, convened a workgroup to (1) develop a conceptual framework for a health-related quality of life PROM; (2) identify and map existing PROMs to the conceptual framework, prioritizing them on the basis of their supporting evidence for use in the regulatory environment; and (3) describe next steps for identifying PROMs for use in regulatory clinical trials of transformative KRT devices. This paper summarizes the proposed health-related quality-of-life PROM conceptual framework, maps and prioritizes PROMs, and identifies gaps and future needs to advance the development of rigorous, meaningful PROMS for use in clinical trials of transformative KRT devices.

Download Full-text

Illustrating the relationship between the number of hypoglycaemia events, event rate reduction and the impact on estimates of quality of life improvement in health economic studies

Value in Health ◽

10.1016/j.jval.2014.03.1466 ◽

2014 ◽

Vol 17 (3) ◽

pp. A251-A252

Author(s):

V. Foos ◽

P. McEwan ◽

D. Grant

Keyword(s):

Quality Of Life ◽

Health Economic ◽

Event Rate ◽

Rate Reduction ◽

Life Improvement ◽

The Impact ◽

The Relationship ◽

Economic Studies

Download Full-text

Assessment of Food and Drug Administration– and European Medicines Agency–Approved Systemic Oncology Therapies and Clinically Meaningful Improvements in Quality of Life

JAMA Network Open ◽

10.1001/jamanetworkopen.2020.33004 ◽

2021 ◽

Vol 4 (2) ◽

pp. e2033004

Author(s):

Vanessa Arciero ◽

Seanthel Delos Santos ◽

Liza Koshy ◽

Amanda Rahmadian ◽

Ronak Saluja ◽

...

Keyword(s):

Quality Of Life ◽

Drug Administration ◽

Food And Drug Administration ◽

European Medicines Agency

Download Full-text

Medical Economics: End the FDA (Food and Drug Administration)

The Winners ◽

10.21512/tw.v18i1.4052 ◽

2017 ◽

Vol 18 (1) ◽

pp. 43

Author(s):

John Romanach ◽

Walter E. Block

Keyword(s):

Literature Review ◽

Drug Administration ◽

Food And Drug Administration ◽

Medical Economics ◽

Free Enterprise ◽

Freedom Of Choice ◽

Calculated Risk ◽

The Difference ◽

Financial Losses

The aim of this research is to know what FDA was done to get pure food and drug. Was it to put all the eggs in one basket and entrust the objective to a monopolistic agency which suffers no financial losses when it errors or would the authors be better off relying on a private, competitive certification industry, the firms of which can earn profits for accurate assessments and losses for erroneous ones? Ensuring the quality of pharmaceuticals was concerned, the best and most efficient means toward that end was reliance on free enterprise. The method used was the literature review by applying what the authors knew about the difference between competition and monopoly to an arena where all too seldom was it applied. It finds that the FDA cannot eliminate risk; only deny people from taking the calculated risk in the hope of curing disease. Legislation such as the Compassionate Freedom of Choice Act has been introduced with the intention of empowering patients to make informed decisions and allow them to take drugs not approved by the FDA.

Download Full-text

Can polygenic risk scores contribute to cost-effective cancer screening? A systematic review

10.1101/2021.11.26.21266911 ◽

2021 ◽

Author(s):

Padraig Dixon ◽

Edna Keeney ◽

Jenny C Taylor ◽

Sarah Wordsworth ◽

Richard Martin

Keyword(s):

Systematic Review ◽

Health Economic ◽

Cost Effectiveness ◽

Cancer Screening ◽

Cost Effective ◽

Risk Scores ◽

Polygenic Risk ◽

The Cost ◽

Economic Studies

Polygenic risk is known to influence susceptibility to cancer. The use of data on polygenic risk, in conjunction with other predictors of future disease status, may offer significant potential for preventative care through risk-stratified screening programmes. An important element in the evaluation of screening programmes is their cost-effectiveness. We undertook a systematic review of papers evaluating the cost-effectiveness of screening interventions informed by polygenic risk scores compared to more conventional screening modalities. We included papers reporting cost-effectiveness outcomes in the English language published as articles or uploaded onto preprint servers with no restriction on date, type of cancer or form of polygenic risk modelled. We excluded papers evaluating screening interventions that did not report cost-effectiveness outcomes or which had a focus on monogenic risk. We evaluated studies using the Quality of Health Economic Studies checklist. Ten studies were included in the review, which investigated three cancers: prostate (n=5), colorectal (n=3) and breast (n=2). All study designs were cost-utility papers implemented as Markov models (n=6) or microsimulations (n=4). Nine of ten papers scored highly (score >75 on a 0-100) scale) when assessed using the Quality of Health Economic Studies checklist. Eight of ten studies concluded that polygenic risk informed cancer screening was likely to be more cost-effective than alternatives. However, the included studies lacked robust external data on the cost of polygenic risk stratification, did not account for how very large volumes of polygenic risk data on individuals would be collected and used, did not consider ancestry-related differences in polygenic risk, and did not fully account for downstream economic sequalae stemming from the use of polygenic risk data in these ways. These topics merit attention in future research on how polygenic risk data might contribute to cost-effective cancer screening.

Download Full-text

Cost-Utility Analyses in Foot & Ankle Surgery

Foot & Ankle Orthopaedics ◽

10.1177/2473011418s00283 ◽

2018 ◽

Vol 3 (3) ◽

pp. 2473011418S0028

Author(s):

Aditya Karhade ◽

John Kwon

Keyword(s):

Health Economic ◽

Cost Effective ◽

Cost Utility ◽

Ankle Sprains ◽

Foot And Ankle ◽

Percentile Score ◽

Ankle Surgery ◽

Value Based Care ◽

Economic Studies

Category: Other Introduction/Purpose: Orthopedic surgery lies at the forefront of the initiative to deliver value-based care. As a result, cost utility analyses have gained increased importance in the orthopedic literature. While other authors have previously reviewed the quality of US-based cost-utility analyses in orthopedic trauma, spine, sports, and arthroplasty, a similar analysis specific to foot and ankle surgery has not been performed. As such, the practicing foot and ankle surgeon does not yet have a comprehensive analysis of the available cost-utility analyses or the quality of the available evidence. The purpose of this study was to perform a systematic review of cost-utility analyses in foot and ankle surgery. Methods: A systematic review of foot and ankle studies was conducted to identify cost-utility based analyses published between 2000 and 2017. Of 687 studies screened by abstract, eight cost-utility foot and ankle studies were identified that met inclusion criteria and were scored by the Quality of Health Economic Studies instrument for overall quality. Results: Of the eight studies that met inclusion criteria, four were published in the U.S. Regarding topic, three examined end-stage arthritis, two examined diabetic foot ulcers, two examined ankle fractures and one examined ankle sprains. Cost-effective interventions identified by these studies included total ankle arthroplasty over ankle fusion or nonoperative treatment, adjunctive hyperbaric oxygen therapy for diabetic foot ulcers, suture button fixation over syndesmotic screws for unstable supination-external rotation type IV ankle fractures, and below knee cast and fracture boot immobilization over tubular bandages for acute severe ankle sprains respectively. The mean Quality of Health Economic Studies scores for these studies was 81.9 with interquartile range, 25th percentile score of 74.8 to 75th percentile score of 86. Conclusion: Despite the increasing focus on value-based care delivery, there are few foot and ankle cost-utility analyses in the current literature. Nonetheless, the quality of existing analyses is high as rated by the Quality of Health Economic Studies and certain interventions have been identified as cost-effective as highlighted above. The findings of this review can be used to help design future analyses in order to best demonstrate the cost-effectiveness of foot and ankle interventions.

Download Full-text

New Drug Approvals by FDA: An Update

Research and Reports of Medicine ◽

10.31829/2637-367x/rrm2018-1(1)-e101 ◽

2018 ◽

pp. 1-6

Keyword(s):

Clinical Trials ◽

Drug Administration ◽

Food And Drug Administration ◽

New Products ◽

New Therapies ◽

New Drug ◽

Arduous Task ◽

Drug Approvals ◽

Preclinical Safety

Bringing a drug into the market is an arduous task, and involved testing from preclinical safety to clinical trials. With its understanding of the science used to create new products, testing and manufacturing procedures, and the diseases and conditions that new products are designed to treat, FDA (U.S. Food and Drug Administration) provides scientific and regulatory advice needed to bring new therapies to market. This article provides an overview of the steps involved during development of a drug and its launch into the market.

Download Full-text