☆ 4.3 Review

How therapeutic advances have transformed the medical landscape: a primer for clinicians

INTERNAL MEDICINE JOURNAL (2023)

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Article Ethics

Do doctors have a responsibility to help patients import medicines from abroad?

Narcyz Ghinea

Summary: Many people have the opportunity to purchase medicines online, but there are also risks involved, such as purchasing poor-quality products. People from low socioeconomic backgrounds, in particular, hope to buy more affordable medicines, and doctors have a responsibility to assist patients in purchasing medicines online.

JOURNAL OF MEDICAL ETHICS (2023)

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Editorial Material Pharmacology & Pharmacy

Real-World Evidence in EU Medicines Regulation: Enabling Use and Establishing Value

Peter Arlett et al.

Summary: The vision is to enable and establish the use of real-world evidence by 2025 across various regulatory use cases, through collaboration with different stakeholders to support the development and use of better medicines for patients.

CLINICAL PHARMACOLOGY & THERAPEUTICS (2022)

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Review Public, Environmental & Occupational Health

New Drug Postmarketing Requirements and Commitments in the US: A Systematic Review of the Evidence

Osman Moneer et al.

Summary: This study aimed to assess the track record of postmarketing requirements (PMRs) and postmarketing commitments (PMCs) by synthesizing information about completion rates, timeliness, study types, and results reporting. The findings indicate that although PMCs are completed at higher rates than PMRs, delays and incomplete studies are common.

DRUG SAFETY (2022)

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Article Health Care Sciences & Services

Understanding innovation of health technology assessment methods: the IHTAM framework

Li Jiu et al.

Summary: This study aimed to construct a conceptual framework to help understand the innovation of health technology assessment (HTA) methods. The framework, developed through analyzing existing HTA frameworks and methods from other disciplines, includes an innovation process and defined roles that can assist HTA stakeholders in dynamic engagement and effective collaboration throughout the innovation process.

INTERNATIONAL JOURNAL OF TECHNOLOGY ASSESSMENT IN HEALTH CARE (2022)

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Review Medicine, General & Internal

Study designs for clinical trials applied to personalised medicine: a scoping review

Cecilia Superchi et al.

Summary: This study aimed to provide an overview of the study designs used in the field of personalized medicine (PM) and proposed a new classification of trial designs for PM to assist readers in navigating the complex field of PM clinical trials.

BMJ OPEN (2022)

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Article Health Care Sciences & Services

How do HTA agencies perceive conditional approval of medicines? Evidence from England, Scotland, France and Canada

Mackenzie Mills et al.

Summary: There is a disconnect between regulatory agencies and HTA agencies on the approval and evaluation of medicines, leading to heterogeneity and uncertainty in the conditional marketing authorisation process.

HEALTH POLICY (2022)

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News Item Medicine, General & Internal

FDA approves $3.5m gene therapy for adults with haemophilia B

Janice Hopkins Tanne

BMJ-BRITISH MEDICAL JOURNAL (2022)

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Editorial Material Oncology

You're Cured Till You're Not: Should Disease-Free Survival Be Used as a Regulatory or Clinical End Point for Adjuvant Therapy of Cancer?

Alberto. F. F. Sobrero et al.

JOURNAL OF CLINICAL ONCOLOGY (2022)

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Article Health Policy & Services

Consumer perspectives of accelerated access to medicines: a qualitative study

Jessica Pace et al.

Summary: Consumers in this study expressed a desire for timely access to new medicines, but not at the expense of safety, efficacy, equity, and sustainability. While accelerated access programs may be welcomed by consumers, it is essential to ensure that they are fully informed of the conditions and limitations, and robust post-market data surveillance is implemented and enforced to protect the interests of individual patients and the broader community.

JOURNAL OF HEALTH ORGANIZATION AND MANAGEMENT (2021)

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Editorial Material Health Care Sciences & Services

Not cost-effective at zero price: valuing and paying for combination therapies in cancer

Nicholas R. Latimer et al.

EXPERT REVIEW OF PHARMACOECONOMICS & OUTCOMES RESEARCH (2021)

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Article Oncology

Basket clinical trial design for targeted therapies for cancer: a French National Authority for Health statement for health technology assessment

Etienne Lengliné et al.

LANCET ONCOLOGY (2021)

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Article Health Care Sciences & Services

Potential approaches for the pricing of cancer medicines across Europe to enhance the sustainability of healthcare systems and the implications

Brian Godman et al.

Summary: European health authorities are concerned about the rising prices of new cancer medicines, discussing various pricing methods and the need for price transparency, as new oncology medicines continue to be developed, there will be stricter regulation over the prices of patented medicines.

EXPERT REVIEW OF PHARMACOECONOMICS & OUTCOMES RESEARCH (2021)

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Article Mathematics, Interdisciplinary Applications

Adaptive Enrichment Designs in Clinical Trials

Peter F. Thall

Summary: Adaptive enrichment designs for clinical trials have various functions such as identifying patient subgroups, selecting or comparing treatments based on interim data, or changing entry criteria; the structure of these designs depends on goals, patient heterogeneity, treatment effects, and practical constraints; this article covers basic concepts and different types of enrichment, with numerical examples for qualitative different cases involving treatment-biomarker interactions.

ANNUAL REVIEW OF STATISTICS AND ITS APPLICATION, VOL 8, 2021 (2021)

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Article Medicine, Research & Experimental

US Food and Drug Administration utilization of postmarketing requirements and postmarketing commitments, 2009-2018

Joshua J. Skydel et al.

Summary: The US Food and Drug Administration requires most new therapeutics to conduct at least one postmarketing clinical study, focusing on safety and efficacy for both approved and unapproved indications. The median number of clinical studies outlined per therapeutic has remained constant over the past decade. Increasing emphasis on faster approval and lifecycle evaluation may necessitate more postmarketing requirements and commitments to address gaps in clinical evidence for therapeutics at approval.

CLINICAL TRIALS (2021)

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Review Cell & Tissue Engineering

The use of innovative payment mechanisms for gene therapies in Europe and the USA

Jesper Jorgensen et al.

Summary: Innovative reimbursement mechanisms are increasingly used for gene therapies in Europe, while the usage is more limited in the USA. Decision makers in different countries face varying incentives and challenges in managing uncertainties around the long-term therapeutic potential of gene therapies.

REGENERATIVE MEDICINE (2021)

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Article Mathematical & Computational Biology

Optimizing subgroup selection in two-stage adaptive enrichment and umbrella designs

Nicolas M. Ballarini et al.

Summary: We propose a two-stage confirmatory clinical trial design that uses adaptation to identify the subgroup of patients benefiting from a new treatment. The study implements adaptations using the conditional error rate approach while optimizing design parameters based on a utility function considering the population prevalence of the subgroups. Results are shown for both traditional trials with familywise error rate control and umbrella trials with control only over the per-comparison type 1 error rate.

STATISTICS IN MEDICINE (2021)

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Review Pharmacology & Pharmacy

Key Characteristics of Database Studies on Drug Effectiveness in the Postmarketing Stage: A Systematic Review

Chihaya Shiragasawa et al.

Summary: This systematic review summarized the current status of cross-disease research articles on database studies focusing on drug effectiveness. The review aimed to provide information to enhance appropriate database studies on drug effectiveness during the postmarketing stage.

PHARMACEUTICAL MEDICINE (2021)

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Article Pharmacology & Pharmacy

Bridging the gap: Can International Consortium of Health Outcomes Measurement standard sets align outcomes accepted for regulatory and health technology assessment decision-making of oncology medicines

Rachel R. J. Kalf et al.

Summary: The study indicates that regulators and HTA agencies place a strong emphasis on disease-specific outcomes in oncology, while HTA agencies focus more on generic outcomes for cross-disease comparisons.

PHARMACOLOGY RESEARCH & PERSPECTIVES (2021)

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Editorial Material Medicine, General & Internal

Approval of Aducanumab for Alzheimer Disease-the FDA's Perspective

Billy Dunn et al.

JAMA INTERNAL MEDICINE (2021)

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Review Oncology

Recent advances and discoveries in the mechanisms and functions of CAR T cells

Rebecca C. Larson et al.

Summary: This review summarizes the major advances that have been made in the efficacy and safety of CAR T cell therapies over the past 3 years and looks ahead to new findings that will impact the future of this immunotherapy.

NATURE REVIEWS CANCER (2021)

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Article Ethics

Formulating an Ethics of Pharmaceutical Disinvestment

Jessica Pace et al.

JOURNAL OF BIOETHICAL INQUIRY (2020)

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Article Rheumatology

Methodological considerations when analysing and interpreting real-world data

Til Sturmer et al.

RHEUMATOLOGY (2020)

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Article Health Care Sciences & Services

Challenges in the value assessment, pricing and funding of targeted combination therapies in oncology

D. Danko et al.

HEALTH POLICY (2019)

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Editorial Material Oncology

Treatment sequencing in oncology: balancing clinical trial and real-world evidence Foreword

Orazio Caffo

FUTURE ONCOLOGY (2019)

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Letter Medicine, General & Internal

Use of an Online Crowdfunding Platform for Unmet Financial Obligations in Cancer Care

Andrew J. Cohen et al.

JAMA INTERNAL MEDICINE (2019)

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Editorial Material Critical Care Medicine

The Challenge of Rare Diseases

James K. Stoller

CHEST (2018)

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Article Medicine, Research & Experimental

Outcome pre-specification requires sufficient detail to guard against outcome switching in clinical trials: a case study

Brennan C. Kahan et al.

TRIALS (2018)

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Editorial Material Medicine, General & Internal

Demands for access to new therapies: are there alternatives to accelerated access?

Jessica Pace et al.

BMJ-BRITISH MEDICAL JOURNAL (2017)

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Editorial Material Medicine, General & Internal

Demands for access to new therapies: are there alternatives to accelerated access?

Jessica Pace et al.

BMJ-BRITISH MEDICAL JOURNAL (2017)

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Article Oncology

Importance of patient reported outcome measures versus clinical outcomes for breast cancer patients evaluation on quality of care

Melissa Kool et al.

BREAST (2016)

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Article Pharmacology & Pharmacy

Characteristics and follow-up of postmarketing studies of conditionally authorized medicines in the EU

Jarno Hoekman et al.

BRITISH JOURNAL OF CLINICAL PHARMACOLOGY (2016)

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Article Cell & Tissue Engineering

Gene therapies: the challenge of super-high-cost treatments and how to pay for them

David R. Carr et al.

REGENERATIVE MEDICINE (2016)

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Article Oncology

Survivorship Programs and Care Planning

Mary S. McCabe et al.

CANCER (2013)

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Article Urology & Nephrology

Systemic therapy for advancing or metastatic prostate cancer (STAMPEDE): a multi-arm, multistage randomized controlled trial

Nicholas D. James et al.

BJU INTERNATIONAL (2009)

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Editorial Material Biotechnology & Applied Microbiology

Off-label or off-limits?

Mark Ratner et al.

NATURE BIOTECHNOLOGY (2008)

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Article Public, Environmental & Occupational Health

Therapeutic misconception and the appreciation of risks in clinical trials

CW Lidz et al.

SOCIAL SCIENCE & MEDICINE (2004)

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