Evaluation of pathological complete response as surrogate endpoint in neoadjuvant randomised clinical trials of early stage breast cancer: systematic review and …

F Conforti, L Pala, I Sala, C Oriecuia, T De Pas… - Bmj, 2021 - bmj.com
Objective To evaluate pathological complete response as a surrogate endpoint for disease-
free survival and overall survival in regulatory neoadjuvant trials of early stage breast …

Systemic therapy for early-stage breast cancer: learning from the past to build the future

E Agostinetto, J Gligorov, M Piccart - Nature Reviews Clinical Oncology, 2022 - nature.com
The treatment of breast cancer has improved dramatically over the past century, from a
strictly surgical approach to a coordinated one, including local and systemic therapies …

Reporting of surrogate endpoints in randomised controlled trial reports (CONSORT-Surrogate): extension checklist with explanation and elaboration

AM Manyara, P Davies, D Stewart, CJ Weir, AE Young… - bmj, 2024 - bmj.com
Randomised controlled trials commonly use surrogate endpoints to substitute for a target
outcome (outcome of direct interest and relevance to trial participants, clinicians, and other …

Surrogacy of pathologic complete response in trials of neoadjuvant therapy for early breast cancer: critical analysis of strengths, weaknesses, and misinterpretations

F Conforti, L Pala, V Bagnardi, T De Pas… - JAMA …, 2022 - jamanetwork.com
Importance The pathologic complete response (pCR) is supported by regulatory agencies
as a surrogate end point for long-term patients' clinical outcomes in the accelerated approval …

Reporting of surrogate endpoints in randomised controlled trial protocols (SPIRIT-Surrogate): extension checklist with explanation and elaboration

AM Manyara, P Davies, D Stewart, CJ Weir, AE Young… - bmj, 2024 - bmj.com
Randomised controlled trials often use surrogate endpoints to substitute for a target outcome
(an outcome of direct interest and relevance to trial participants, clinicians, and other …

Overall survival benefits of cancer drugs approved in China from 2005 to 2020

Y Zhang, H Naci, AK Wagner, Z Xu, Y Yang… - JAMA network …, 2022 - jamanetwork.com
Importance Of approximately 9 million patients with cancer in China in 2020, more than half
were diagnosed with late-stage cancers. Recent regulatory reforms in China have focused …

Implications of oncology trial design and uncertainties in efficacy-safety data on health technology assessments

D Trapani, K Tay-Teo, ME Tesch, F Roitberg… - Current …, 2022 - mdpi.com
Background: Advances in cancer medicines have resulted in tangible health impacts, but the
magnitude of benefits of approved cancer medicines could vary greatly. Health Technology …

Evaluating covid-19 vaccine efficacy and safety in the post-authorisation phase

C Prugger, A Spelsberg, U Keil, J Erviti, P Doshi - bmj, 2021 - bmj.com
When covid-19 vaccines were first authorised, regulators required post-authorisation studies
to tackle important uncertainties about efficacy and safety. But these studies may have little …

Aspiring to reasonableness in accelerated approval: anticipating and avoiding the next aducanumab

EA Largent, A Peterson, J Karlawish, HF Lynch - Drugs & aging, 2022 - Springer
Abstract The US Food and Drug Administration's decisions about drug approval—though
guided by science, as well as relevant statutes, regulations, and guidance documents …

For whom the price escalates: high price and uncertain value of cancer drugs

G Shin, HY Kwon, SJ Bae - … of Environmental Research and Public Health, 2022 - mdpi.com
The price of cancer drugs has skyrocketed, yet it is not clear whether their value is
commensurate with their price. More cancer drugs are approved under expedited review …