India’s clinical-trial rules to speed up drug approvals

Nature ◽  
2019 ◽  
Author(s):  
Gayathri Vaidyanathan
Keyword(s):  
Clinical Trial ◽  
Speed Up ◽  
Drug Approvals

Timing of US Food and Drug Administration (FDA) cancer drug approvals relative to publication of clinical trial results.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 2071-2071 ◽  
Author(s):  
Ali Raza Khaki ◽  
Aakash Desai ◽  
Martin W. Schoen ◽  
Bishal Gyawali ◽  
Eddy J. Chen ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Cancer Drug ◽  
Cancer Drugs ◽  
Cancer Treatments ◽  
Fda Approval ◽  
Anti Cancer ◽  
Accelerated Approval ◽  
Drug Approvals ◽  
Clinical Trial Results ◽  
Careful Assessment

2071 Background: Publication of clinical trial results in peer reviewed literature is essential to inform clinicians regarding the use of new anti-cancer treatments, which often have a low therapeutic ratio and require careful assessment of risks and benefits. Publication of registration trials should precede FDA approval to facilitate evaluation and implementation of new therapies. The timing of trial publication relative to FDA drug approvals has not been systematically investigated. Methods: We collected all FDA drug approvals for a cancer indication between 2000-19. Trials were identified using FDA labels as well as drugs and publications indexed on HemOnc.org. Approvals for generics/biosimilars, non-oncology indications and label revisions without supportive evidence were excluded. Dates of approval, the approval pathway, approval type (new vs expansion), and the first full publication related to the registration were recorded. Trials and approvals were matched using available metadata. We calculated the proportion of drugs approved prior to publication overall and for those receiving accelerated approval (AA). We used logistic regression to compare rates of pre-publication approval by approval pathway and by new vs expanded approval. Results: Among a total of 378 drug approvals, 139 (37%) had pre-publication approval. Of these, the median overall time from approval to publication was 140 days (IQR 64-281 days). For those with approval after publication, median time from publication to approval was 157 days (IQR 72-359 days). The number of drugs approved pre-publication rose by 27% between the first and last quarters of the study period, though, the proportion decreased as more anti-cancer drugs have been approved in recent years (Table). More drugs were approved pre-publication through AA than regular approval (46% vs 34%, OR 1.66 [95% CI 1.03-2.70], p=0.04) and as new approvals vs. expanded approvals (45% vs 32%, OR 1.76 [95% CI 1.15-2.70], p=0.01). Conclusions: A substantial minority of FDA approvals occur before trial results are published, with the odds being higher for drugs receiving AA and for new approvals. Since clinicians rely upon published results to inform risk/benefit decisions, efforts are needed to ensure trial results are published by the time of FDA approval of new cancer drugs and indications. [Table: see text]

scholarly journals Appendicostomy Antegrade Colonic Irrigation by Electromechanical Pump, a Novel Method for Managing Fecal Incontinence, Randomized Clinical Trial

2019 ◽  
Author(s):  
Seyed Hamzeh Mousavie ◽  
Rezvan Mirzaei ◽  
Bahar Mahjoubi ◽  
Amir Nahavandi
Keyword(s):  
Clinical Trial ◽  
Fecal Incontinence ◽  
Traditional Method ◽  
Cleveland Clinic ◽  
Colonic Irrigation ◽  
Incontinence Score ◽  
Cord Injury ◽  
Speed Up ◽  
Novel Method ◽  
Colon Irrigation

Antegrade colonic irrigation enema (ACIE) is established as an old treatment for child fecal incontinence and recently is approved as a treatment for different causes of adult fecal incontinence (cancer surgery, neurogenic bowel, spinal cord injury, sphincter trauma, etc.). Despite the benefits of emptying the large bowel and prevention of fecal incontinence or constipation, this method is time-consuming and requires thorough instruction and training. The purpose of this study was to innovate and assess novel technique to decrease time-consuming and improve performance of irrigation. We designed and electromechanical pump for colon irrigation and assessed by a randomized crossover clinical trial study, involving two-four weeks treatment phases. 30 patients have included that suffered fecal incontinence and had been managed with appendicostomy antegrade colon irrigation. The results showed that the pump decrease time and volume of irrigation compare with the traditional method. All patients had a reduction in toileting times. Traditional mean toileting time was 67 minutes, versus pump-toileting time that was 24.6 minutes (P:0.00). The volume of water was reduced in 13 patients. Mean of the volume was 1712 ml in the traditional method and 1128 ml in pump method (P:0.279). Mean Cleveland Clinic Florida Fecal Incontinence score (CCF_FIS) for the pump was 5.84 compare 6.24 for traditional method (P:0.000). Our study provides evidence for the first time that our novel method can facilitate and speed-up colon irrigation without any adverse effect on the outcome. © 2018 Tehran University of Medical Sciences. All rights reserved. Acta Med Iran 2018;57(4):216-223.

Effects of FDA drug approvals on a thoracic oncology program’s clinical trial enrollment in 2015.

2016 ◽  
Vol 34 (15_suppl) ◽  
pp. e20662-e20662
Author(s):  
Alice Huai-Yu Li ◽  
Jonathan Wade Goldman ◽  
Marshall L. Spiegel ◽  
Carlos R. Adame ◽  
Courtney L. Wells ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trial Enrollment ◽  
Drug Approvals

The impact of NCI-sponsored network group clinical trials on guideline care and new drug indications.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 6604-6604
Author(s):  
Joseph M. Unger ◽  
Van T. Nghiem ◽  
Dawn L. Hershman ◽  
Riha Vaidya ◽  
Michael Leo LeBlanc ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Guidelines ◽  
Research Network ◽  
Phase Iii ◽  
Cancer Clinical Trials ◽  
New Drug ◽  
Public Data ◽  
Drug Approvals ◽  
The Impact

6604 Background: National Cancer Institute Clinical Trial Network (NCTN) groups serve a vital role in identifying new antineoplastic regimens. However, the clinical impact of their trials has not been systematically examined. We analyzed the influence of network group cancer clinical trials on clinical guidelines and new drug approvals. Methods: We evaluated Phase III cancer clinical trials which the SWOG Cancer Research Network coordinated or participated in (1980-2017). Included trials were completed and its results published. A documented practice influential (DPI) trial was one with verified influence on National Comprehensive Cancer Network (NCCN) clinical guidelines (available starting in 1996) or on U.S. Food and Drug Administration (FDA)-approved package inserts. We estimated the rate of DPI trials overall and over time. The total federal investment supporting the set of trials was also determined based on public data. Results: In total, 182 trials comprising 148,028 patients were studied. We identified 79 DPI studies (43.4%); 73 influenced NCCN guidelines, 12 influenced new drug approvals, and 6 influenced both. The rate of DPI trials was 72.3% (47/65) among formally positive trials (i.e., achieved their protocol specified endpoint) and 27.4% (32/117) among negative trials. Thus 40.5% (32/79) of DPI trials were based on negative studies, half of which (16/32 = 50.0%) reaffirmed standard of care over experimental therapy. There were no differences between DPI and non-DPI trials in key study design characteristics. Total federal investment for the programs conducting the trials was $1.36 billion (USD2017), a rate of $7.5 million per trial, or $17.2 million per DPI trial. Conclusions: Nearly half of all phase III trials by one of the NCTN’s largest groups had documented practice influence on clinical care guidelines or new drug approvals. Even many negative trials impacted guideline recommendations. Compared to the costs of a new drug approval in pharmaceutical companies – typically estimated at > $1 billion – the amount invested by federal funders to provide this valuable evidence was modest. These findings highlight the major role of the NCTN’s clinical trial program in advancing oncology practice.

scholarly journals Landmark Cancer Clinical Trials and Real-World Patient Populations: Examining Race and Age Reporting

Cancers ◽  
2021 ◽  
Vol 13 (22) ◽  
pp. 5770
Author(s):  
Thejus Jayakrishnan ◽  
Sonikpreet Aulakh ◽  
Mizba Baksh ◽  
Kianna Nguyen ◽  
Meghna Ailawadhi ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Real World ◽  
Age Distribution ◽  
Mean Difference ◽  
Cancer Drug ◽  
P Value ◽  
World Population ◽  
Drug Approvals ◽  
Age Disparity

Background: Concern exists that the clinical trial populations differ from respective cancer populations in terms of their age distribution affecting the generalizability of the results, especially in underrepresented minorities. We hypothesized that the clinical trials that do not report race are likely to suffer from a higher degree of age disparity. Methods: Food and Drug Administration (FDA) drug approvals from July 2007 to June 2019 were reviewed to identify oncology approvals, and trials with age details were selected. The outcomes studied were the weighted mean difference in age between the clinical trial population and real-world population for various cancers, the prevalence of race reporting and association of age and race reporting with each other. Results: Of the 261 trials, race was reported in 223 (85.4%) of the trials, while 38 trials (14.6%) had no mention of race. Race reporting improved minimally over time: 29 (85.3%) in 2007–2010 vs. 49 (80.3%) in 2011–2014 vs. 145 (85.4%) during the period 2015–2019 (p-value = 0.41). Age discrepancy between the clinical trial population and the real-world population was higher for studies that did not report race (mean difference −8.8 years (95% CI −12.6 to −5.0 years)) vs. studies that did report it (mean difference −5.1 years, (95% CI −6.4 to −3.7 years), p-value = 0.04). Conclusion: The study demonstrates that a significant number of clinical trials leading to cancer drug approvals suffer from racial and age disparity when compared to real-world populations, and that the two factors may be interrelated. We recommend continued efforts to recruit diverse populations.

Trends in PRO reporting in clinical trials leading to GU cancer drug approvals from 2007 to 2018.

2020 ◽  
Vol 38 (6_suppl) ◽  
pp. 641-641
Author(s):  
Monica Tamil ◽  
Houssein Safa ◽  
Adele Semaan ◽  
Jad Chahoud
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Standards Of Care ◽  
Cancer Drug ◽  
Type I ◽  
Cancer Drugs ◽  
Urological Cancer ◽  
Quality Reporting ◽  
Patient Reported ◽  
Drug Approvals

641 Background: Having standardized and high quality reporting of patient reported outcomes (PRO), especially in clinical trials that establish standards of care in oncology is important for patient centered care. This study assessed the status of reporting and quality of analysis of PROs in FDA approved drugs for genitourinary malignancies. Methods: We conducted a systematic review of the FDA archives to identify urological cancer drugs approved between 2007-2018. We retrieved the clinical trials that led to these drug approvals from ClinicalTrials.gov and PubMed. We systematically screened for PROs and reviewed their analytic tools and interpretation methods reported in their published manuscripts and study protocols. A clinical trial was considered to include PROs if they were reported in either the primary or a subsequent manuscript. Results: We identified 22 clinical trials leading to FDA approval of urological cancer drugs between 2007-2018. Only 63% of trials had published PROs. PROs were reported in the primary clinical trial manuscripts for two drugs (9%), and in a secondary PRO manuscript for 12 drugs (54%). The median time between the primary and secondary papers was 12 months (IQR:7.5-26 months). Among the 14 published PRO papers, the hypothesis was broad in 79%, and not reported in 21%. PROs were never included as a primary endpoint of a study. Instead, PROs were reported as secondary endpoints in 5 (36%) and as exploratory endpoints in 7 (50%) studies, while two papers (14%) did not mention PRO reporting in their endpoints. The most common PRO instruments were EQ-5D (64%) and FACT-P (50%). In 92% of PRO papers, statistical analyses were conducted to account for missing data. Control for type I error was needed but not done in 57% of the trials. Conclusions: Delays in publication of PROs occur regularly in trials leading to drug approval in GU malignancies. Our study highlights the need to enhance standardization of the analysis and interpretation of PROs to maximize the value of this data for drug development and approval.

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