scholarly journals 4381 Examination of FDA Pediatric Regulations: Inclusion of Pediatric Populations in Clinical Trials, 2016 - 2018

2020 ◽  
Vol 4 (s1) ◽  
pp. 131-131
Author(s):  
Annie Ly ◽  
Apurva Uniyal ◽  
Terry Church
Keyword(s):  
Clinical Trials ◽  
Pharmaceutical Industry ◽  
Drug Administration ◽  
Pediatric Population ◽  
Eligibility Criteria ◽  
Data Presentation ◽  
Study Population ◽  
Pediatric Populations ◽  
Approved Drugs ◽  
The U.S

OBJECTIVES/GOALS: To assess whether FDA regulations aimed at the pediatric population following the Best Pharmaceuticals for Children Act (BPCA) of 2002 are effective, this study examines the inclusion of the pediatric population in recent clinical trials for drugs used by both adult and pediatric groups. METHODS/STUDY POPULATION: From the U.S. Food and Drug Administration (FDA) a list of drugs approved between 2016 and 2018 was compiled. A search of clinicaltrials.gov provided corresponding clinical trials for the approved drugs. Study information such as eligibility criteria and demographics was gathered from each trial. From studies that included both adult and pediatric populations, the percentage of pediatric and adult subjects was calculated, resulting in values expressing exclusively pediatric subjects or the pediatric subjects as part of a category that included both populations (i.e. 18 years old). RESULTS/ANTICIPATED RESULTS: Between 2016 and 2018, 26 drugs were approved under the BPCA. From an assessment of 220 total studies, a lack of standardization is evident in terms of which ages constitute a particular pediatric sub-population even though guidelines for these sub-populations already exist under the BPCA. This lack of standardization resulted in the separate examination of each drug for pediatric inclusion. For the majority of the trials evaluated, 1% of the pediatric population was represented in trials that were open to both adult and pediatric populations. DISCUSSION/SIGNIFICANCE OF IMPACT: There is a need for more effective regulations and incentives for the pharmaceutical industry to standardize data presentation and better incorporate the pediatric population in clinical trials, especially for drugs targeted for this group.

Comments on the Draft Guidance on “Adaptive Design Clinical Trials for Drugs and Biologics” of the U.S. Food and Drug Administration

2010 ◽  
Vol 20 (6) ◽  
pp. 1125-1131 ◽  
Author(s):  
Werner Brannath ◽  
Hans Ulrich Burger ◽  
Ekkehard Glimm ◽  
Nigel Stallard ◽  
Marc Vandemeulebroecke ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Administration ◽  
Adaptive Design ◽  
Food And Drug Administration ◽  
Draft Guidance ◽  
The U.S

The impact of newly approved drugs on clinical and operational strategies for multiple myeloma clinical trials.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e19511-e19511
Author(s):  
Bhavani Krishnan ◽  
Bernadette Collins ◽  
Michael Jeffrey Cho ◽  
Tanya Partridge ◽  
Durga Vighnay ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Multiple Myeloma ◽  
Clinical Trials ◽  
Real World ◽  
Operational Strategies ◽  
Clinical Trial Enrollment ◽  
Near Term ◽  
Approved Drugs ◽  
The Impact ◽  
The U.S

e19511 Background: The global burden of multiple myeloma (MM) has increased steadily in last 3 decades. The IARC reports there were ~160,000 new cases worldwide in 2018. There has been an increase in the development and approval of more effective targeted therapy options (new immunomodulatory agents, proteasome inhibitors, and monoclonal antibodies); this, coupled with high adoption of these therapies presents a major challenge in enrollment of current ongoing clinical trials. We assessed the impact of near-term regulatory approvals on clinical trial enrollment. Methods: Public domain clinical trial enrollment data from MM studies (Phase I, I/II, II) closed/completed between 2011-2018 were used to determine enrollment trends pre and post 2014. We leveraged real-world medical claims data to project/model adoption of recently approved drugs in the U.S.; additionally, drug sales volume data was used to evaluate ex-US national adoption trends. The utilization rates of 5 recently approved drugs by regimen and line of treatment was determined. Results: In the U.S., there is a 13% increase in median enrollment duration with a corresponding 25% decrease in median p/s/m enrollment in MM studies, irrespective of phase, where enrollment was completed between 2015 -2018 compared to 2011-2014. We hypothesize that one of the factors for this increase in enrollment timeline is the approval and adoption of newly approved therapies post 2014. Two of the five recently approved drugs show a steady increase in the number of patients treated over 2016, 2017 and 2018, while one of the drugs plateaus over the same period. Outside of the U.S., our analysis confirms the existence of gaps in time to approval /adoption of recently approved drugs; for example, we observe a two-year delay in approval/adoption for one of the drugs in France compared to the other countries in Western Europe. Conclusions: There are over 10 investigational MM drugs currently in development which are anticipated to be granted approval between 2019-2021. Factoring the real-world adoption of near-term drug approval into global clinical and operational strategies offers insights into mitigating potential future enrollment challenges.

Evaluation of factors associated with recruitment in hematological clinical trials: A retrospective study

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 6567-6567
Author(s):  
C. Amireault ◽  
S. Camden ◽  
J. Poulin ◽  
J. Lemieux
Keyword(s):  
Clinical Trials ◽  
Stage Iv ◽  
Quebec City ◽  
Cancer Clinical Trials ◽  
First Line ◽  
Exclusion Criteria ◽  
Eligibility Criteria ◽  
Factors Associated ◽  
Study Population ◽  
Few Data

6567 Background: Recruitment of patients in cancer clinical trials has been reported to be between 3%–5%. Very few data come from Canada. Methods: The objective was to measure the recruitment and its associated characteristics in hematology clinical trials for malignant diseases. This was a retrospective cohort study using charts review in a tertiary hematology centre in Québec City, Canada. Clinical trials opened between 2002 and 2008 were selected. For each protocol, main criteria were used to define the population under study (e.g., stage IV Hodgkin lymphomas first-line). If the patient filled the main criteria, all eligibility criteria (inclusion and exclusion criteria) of the protocol were assessed. Results: Among all charts reviewed, 697 patients were identified in 17 protocols. However, as a patient could be assessed for more than one protocol if applicable, this population reached 1,394 observations. The study population filling the main criteria of a protocol included 195 observations in 17 protocols (186 different patients). Only 9.7% (8.2–11.4) filled all the eligibility criteria and 3.3% (2.5–4.4) were recruited among all charts reviewed (1394 observations). However, theses rates reached 68.2 % (61.2–75.1) and 23.1 % (17.9–29.8), respectively, among patients meeting the main criteria of a protocol (195 observations). Recruitment in the population who filled all eligibility criteria (inclusion and exclusion criteria) was 33.8% (26.7–42.5). Patient's sex, age, comorbidities, doctor's sex, doctor's age and protocol characteristics were not associated with recruitment in the population filling the main criteria, but having a note in the chart about the protocol appears to be associated with higher recruitment (p < 0.0001). The most common reasons for not being recruited were as follow (could have more than one reason): 40.7% not fulfilling all eligibility criteria, 31.3% protocol not being proposed and 22.7% patients’ refusal. Patients reasons for refusals were (could have more than one reason): 50% unknown, 26.5% fear of side effects, 20.6% too many visits, 14.7 % already enough anxious about disease, other. Conclusions: The largest barriers about recruitment were protocol ineligibility and protocol not being proposed by the medical team. No significant financial relationships to disclose.

scholarly journals The Clinical Trials Transformation Initiative: Methodology supporting the mission

2018 ◽  
Vol 15 (1_suppl) ◽  
pp. 13-18 ◽  
Author(s):  
Amy Corneli ◽  
Zachary Hallinan ◽  
Gerrit Hamre ◽  
Brian Perry ◽  
Jennifer C Goldsack ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Public Private Partnership ◽  
Private Partnership ◽  
Key Stakeholders ◽  
Duke University ◽  
Fit For Purpose ◽  
Evidence Gathering ◽  
The U.S

The mission of the Clinical Trials Transformation Initiative, a public–private partnership co-founded by the U.S. Food and Drug Administration and Duke University, is to develop and drive adoption of practices that will increase the quality and efficiency of clinical trials. The Clinical Trials Transformation Initiative works collaboratively with key stakeholders, implements “fit-for-purpose” evidence-gathering projects, and develops actionable recommendations and tools to address the challenges faced by the clinical trials enterprise. In pursuit of its mission, The Clinical Trials Transformation Initiative follows an innovative and collaborative, five-step methodology: (1) state the problem and identify impediments to research, (2) gather evidence to identify gaps and barriers, (3) explore results by analyzing and interpreting findings, (4) finalize solutions by developing recommendations and tools, and (5) drive adoption through disseminating and implementing recommendations and tools. This article describes each step of the Clinical Trials Transformation Initiative's methodology, with a specific focus on describing the evidence-gathering activities.

Strategies to overcome barriers to accrual (BtA) to NCI-sponsored clinical trials: A project of the NCI-Myeloma Steering Committee Accrual Working Group (NCI-MYSC AWG).

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 8592-8592
Author(s):  
Matthias Weiss ◽  
Morie A. Gertz ◽  
Richard F. Little ◽  
Vincent Rajkumar ◽  
Susanna J. Jacobus ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Administration ◽  
Phase Ii ◽  
Working Group ◽  
Treatment Options ◽  
Insurance Coverage ◽  
Scientific Progress ◽  
Phase Iii ◽  
Eligibility Criteria ◽  
Ct Protocol

8592 Background: Accrual to NCI clinical trials(CT)is often slower than planned at times mandating premature closure resulting in loss of valuable resources and delay of scientific progress. Methods: The NCI-MYSC AWG identified 10 potential BtA. SWOG, ECOG and Alliance investigators were queried and agreed that these barriers impede accrual (results stratified for academic and community sites). The MYSC AWG developed in collaboration with NCI and FDA strategies to overcome these barriers. Results: Strategies listed for the 3 most often cited BtA: 1. Reimbursement for CT related expenses:increase awareness of improved reimbursement for phase II CT; tailor reimbursement according to CT complexity; request funds from industry and other sources ( http://biqsfp.cancer.gov ) for qualifying ancillary CT components. 2. Spectrum of available treatment options influences CT participation: educate patients and providers about the significance of a new CT using social media, presentations at national meetings and by adding educational material to CT protocol; encourage opinion leaders and advocacy groups not to promote a new therapy as “standard” in the absence of phase III data. 3. Requirement of CT specific therapy at NCI designated sites only: “MYSC AWG Drug Administration Table” describes NCI/FDA approved rules for CT specific drug administration; CT protocol will outline which standard treatment components of a CT can be administered at any site as long as protocol specific guidelines are followed and conduct is supervised by enrolling investigator. Examples of additional strategies to overcome identified BtA: determine feasibility, indication and insurance coverage of CT specific tests during protocol development; discourage narrow eligibility criteria; avoid competing CT; allow up to 1 cycle of commercially available therapy prior to enrollment; CIRB support for phase II CT. Conclusions: The MYSC Accrual Working Group developed in collaboration with NCI and FDA strategies to overcome barriers to myeloma clinical trial accrual. These strategies may be applicable to NCI-sponsored clinical trials evaluating interventions in other diseases.

scholarly journals THE STATE OF THE NATION: A 50-STATE COVID-19 SURVEY REPORT #9: WILL AMERICANS VACCINATE THEMSELVES AND THEIR CHILDREN AGAINST COVID-19?

2020 ◽  
Author(s):  
Roy H. Perlis ◽  
David Lazer ◽  
Katherine Ognyanova ◽  
Matthew Baum ◽  
Mauricio Santillana ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
District Of Columbia ◽  
The State ◽  
Phase Iii ◽  
Phase Iii Clinical Trials ◽  
Survey Report ◽  
The U.S

At least 5 companies have launched Phase III clinical trials of COVID-19 vaccines, the final step before seeking approval from the U.S. Food and Drug Administration (FDA). According to NIAID director Anthony Fauci, vaccines may be widely available in the U.S. by spring 2021 if these trials are successful.But should these vaccines become available, will Americans accept them? Between July 10 and July 26, we surveyed 19,058 adults in all 50 U.S. states and the District of Columbia. We asked about the likelihood that they would seek vaccination for themselves, and for their children. We also asked about the factors that would influence their decision making.We find that, overall, 66% of adults would be somewhat or extremely likely to vaccinate themselves; 66% would be somewhat or extremely likely to vaccinate their children. These rates vary markedly between states, as shown on the figure below.

scholarly journals 4552 Pediatrician Readiness to Participate in Clinical Trials: Roles of interest, barriers and interventions

2020 ◽  
Vol 4 (s1) ◽  
pp. 64-65
Author(s):  
Beatrice Boateng ◽  
Jessica Snowden ◽  
Diana Munoz-Mendoza ◽  
Clare Nesmith ◽  
Frederick Barr ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Gold Standard ◽  
Online Survey ◽  
Randomized Clinical Trials ◽  
Evidence Based ◽  
Clinical Educator ◽  
Study Population ◽  
Pediatric Populations ◽  
Prior Experiences ◽  
Based Medicine

OBJECTIVES/GOALS: Clinical trials are the gold standard for developing evidence-based medicine. However, 20% of pediatric randomized clinical trials are discontinued and about 30% of completed trials go unpublished. (Pica and Bourgeois, 2016) Although patient recruitment is the most cited barrier to completing clinical trials, trials funded by academia are more likely discontinued compared to those funded by industry. This study is an attempt to gain additional insights into clinical trials in academic pediatrics. METHODS/STUDY POPULATION: Junior pediatrics faculty (Instructors and Assistant Professors) were recruited to participate in an online survey through RedCAP. The physicians were asked if they had prior experiences with clinical trials and whether they have interest in participating in clinical trials. Those interested were asked three additional questions: what role they were interested in, barriers to participating and interventions they thought would educate them about participating in clinical trials. RESULTS/ANTICIPATED RESULTS: Ninety two (92) out of 119 (77%) junior pediatrics faculty completed the survey. Twenty (20) pediatric subspecialties were represented and respondents were on various academic pathways. A third of the respondents (35%) had previously participated in clinical trials. A majority of the faculty respondents (84; 70%) are on the clinical educator pathway. The 13 respondents who were not interested in clinical trials indicated their preference for patient care, education and quality improvement. Of those interested in clinical trials, the top three preferred roles were site co-investigator (68%), help designing future protocol (47%) and site principal investigator (44%). Other than time, the top barriers to participation were a lack of awareness of what it takes to lead or engage in clinical trials (53%) and a lack of training on clinical trials (45%). Mentoring from an experienced clinical trialist emerged as the top preferred intervention (78%). DISCUSSION/SIGNIFICANCE OF IMPACT: Although limited to one institution, the findings of this study provide insights into pediatric faculty interest in clinical trials. If academic pediatricians are provided with mentoring, there could be an uptick in completed and published clinical trials involving pediatric populations.

scholarly journals Patient advocacy organizations’ information for patients on pre-approval access to investigational treatments

2019 ◽  
Vol 12 (1) ◽  
Author(s):  
Kelly McBride Folkers ◽  
Sarah Leone ◽  
Arthur Caplan
Keyword(s):  
United States ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Drug Administration ◽  
Patient Advocacy ◽  
The United States ◽  
Patient Access ◽  
Advocacy Organizations ◽  
Lack Of Information ◽  
The U.S

Abstract Objective To evaluate the availability of information regarding patient access to investigational treatments through clinical trials and non-trial pre-approval access pathways from a sample of patient advocacy organization (PAO) websites in the United States. Results We systematically analyzed the content of 118 randomly selected PAO websites to assess whether they contained information on clinical trials and non-trial pathways—e.g., the U.S. Food and Drug Administration (FDA) expanded access (EA) program and right to try—over the course of two months from February to March 2019. A majority (81%, n = 96) of PAOs provided a link to ClinicalTrials.gov, and 73% (n = 86) had their own clinical trial finder or list of relevant trials. 23% (n = 27) mentioned EA, with 8% (n = 9) providing specific resources for FDA’s EA program. 8% (n = 10) provided a statement on the passage of the federal right to try law. A majority of PAO websites contained information on clinical trials, but a minority discussed non-trial pre-approval access. The lack of information on the latter highlights an area in need of improvement.

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