Should regulatory authorities approve drugs based on surrogate endpoints?

: Safety, efficacy and quality of a therapeutic product is the major concern for the pharmaceutical companies. FDA and PMDA are the main regulatory authorities in USA & JAPAN respectively that ensures the maintenance of these required parameters by forming standard guidelines and process for drug approval. These regulatory authorities’ reviews each step of a pharmaceutical drug product from its discovery phase to marketed product. Dossier plays an important role during the approval process of a drug product, as it allows both applicant and review team members to evaluate the data in an effective manner. A dossier consists of five modules containing informative data of various stages of a drug product but in a brief pattern with folders and subfolders. In the present paper, the authors focus on in-depth review of approval process for new and generic drugs in USA and Japan.

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COVID-19 vaccines: progress and understanding on quality control and evaluation

Signal Transduction and Targeted Therapy ◽

10.1038/s41392-021-00621-4 ◽

2021 ◽

Vol 6 (1) ◽

Author(s):

Qunying Mao ◽

Miao Xu ◽

Qian He ◽

Changgui Li ◽

Shufang Meng ◽

...

Keyword(s):

Quality Control ◽

Global Health ◽

Research And Development ◽

Drug Evaluation ◽

Control Laboratory ◽

Vaccine Candidates ◽

Regulatory Authorities ◽

Quality Control Laboratory ◽

Leading Role ◽

National Quality

AbstractThe outbreak of COVID-19 has posed a huge threat to global health and economy. Countermeasures have revolutionized norms for working, socializing, learning, and travel. Importantly, vaccines have been considered as most effective tools to combat with COVID-19. As of the beginning of 2021, >200 COVID-19 vaccine candidates, covering nearly all existing technologies and platforms, are being research and development (R&D) by multiple manufacturers worldwide. This has posed a huge obstacle to the quality control and evaluation of those candidate vaccines, especially in China, where five vaccine platforms are deployed in parallel. To accelerate the R&D progress of COVID-19 vaccines, the guidances on R&D of COVID-19 vaccine have been issued by National Regulatory Authorities or organizations worldwide. The Center for Drug Evaluation and national quality control laboratory in China have played a leading role in launching the research on quality control and evaluation in collaboration with relevant laboratories involved in the vaccine R&D, which greatly supported the progression of vaccines R&D, and accelerated the approval for emergency use and conditional marketing of currently vaccine candidates. In this paper, the progress and experience gained in quality control and evaluation of COVID-19 vaccines developed in China are summarized, which might provide references for the R&D of current and next generation of COVID-19 vaccines worldwide.

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Validity of Surrogate Endpoints and Their Impact on Coverage Recommendations: A Retrospective Analysis across International Health Technology Assessment Agencies

Medical Decision Making ◽

10.1177/0272989x21994553 ◽

2021 ◽

pp. 0272989X2199455

Author(s):

Oriana Ciani ◽

Bogdan Grigore ◽

Hedwig Blommestein ◽

Saskia de Groot ◽

Meilin Möllenkamp ◽

...

Keyword(s):

Health Technology Assessment ◽

Technology Assessment ◽

International Health ◽

Surrogate Endpoint ◽

Health Technology ◽

Surrogate Endpoints ◽

Patient Centered ◽

Statistical Validation ◽

Level Of Evidence ◽

The Impact

Background Surrogate endpoints (i.e., intermediate endpoints intended to predict for patient-centered outcomes) are increasingly common. However, little is known about how surrogate evidence is handled in the context of health technology assessment (HTA). Objectives 1) To map methodologies for the validation of surrogate endpoints and 2) to determine their impact on acceptability of surrogates and coverage decisions made by HTA agencies. Methods We sought HTA reports where evaluation relied on a surrogate from 8 HTA agencies. We extracted data on the methods applied for surrogate validation. We assessed the level of agreement between agencies and fitted mixed-effects logistic regression models to test the impact of validation approaches on the agency’s acceptability of the surrogate endpoint and their coverage recommendation. Results Of the 124 included reports, 61 (49%) discussed the level of evidence to support the relationship between the surrogate and the patient-centered endpoint, 27 (22%) reported a correlation coefficient/association measure, and 40 (32%) quantified the expected effect on the patient-centered outcome. Overall, the surrogate endpoint was deemed acceptable in 49 (40%) reports ( k-coefficient 0.10, P = 0.004). Any consideration of the level of evidence was associated with accepting the surrogate endpoint as valid (odds ratio [OR], 4.60; 95% confidence interval [CI], 1.60–13.18, P = 0.005). However, we did not find strong evidence of an association between accepting the surrogate endpoint and agency coverage recommendation (OR, 0.71; 95% CI, 0.23–2.20; P = 0.55). Conclusions Handling of surrogate endpoint evidence in reports varied greatly across HTA agencies, with inconsistent consideration of the level of evidence and statistical validation. Our findings call for careful reconsideration of the issue of surrogacy and the need for harmonization of practices across international HTA agencies.

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In and out of revolving doors in European Union financial regulatory authorities

Regulation & Governance ◽

10.1111/rego.12424 ◽

2021 ◽

Author(s):

Adam William Chalmers ◽

Robyn Klingler‐Vidra ◽

Alfio Puglisi ◽

Lisa Remke

Keyword(s):

European Union ◽

Regulatory Authorities ◽

Revolving Doors ◽

Financial Regulatory

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