Recruitment In Clinical Studies Research Articles

Disease characteristics and medications use in idiopathic inflammatory myopathy: a multi-center prospective observational study of decentralized remote vs. traditional clinic enrollment.

Idiopathic Inflammatory Myopathies (IIM) are rare and characterized by heterogeneous manifestations and clinical trajectories. Utilizing tele-research methods has the potential to improve participant recruitment and advance the understanding of the disease. We aimed to evaluate disease characteristics in IIM patients throughout the U.S. and compare these parameters between patients recruited remotely through mobile application or website vs those recruited locally in myositis clinics. "Myositis Patient Centered Tele-Research" (My PACER) is a multicentre prospective observational study of U.S. IIM subjects, competitively recruited through traditional in-person clinic visits (Center-Based Cohort [CBC]), and remotely using mobile application or website and social media (Tele-Research Cohort [TRC]). Data collection comprised baseline demographic and clinical variables, encompassing symptoms, organ involvement, diagnostic tests results and medication use. The study included 120 IIM patients, 82 in the TRC and 38 in the CBC. The average age was 55 ± 13.4, 75% females and 81% Caucasians. Both cohorts exhibited similar demographic characteristics. Overall, 41% dermatomyositis, 27% polymyositis, 23% anti-synthetase syndrome, and 9% necrotizing myositis patients were enrolled, with comparable subtypes prevalence among cohorts (p= 0.85). The groups demonstrated similarities in multiple clinical factors, including muscle enzymes, diagnostic delay, employment status, various patient and physician-reported outcomes, functional tests, and the frequency of abnormal findings in chest CT, pulmonary function tests, and electromyography. TRC patients received biologics and csDMARDs more frequently (p< 0.001 and p= 0.013, respectively). Tele-research recruitment yielded a patient cohort resembling traditionally recruited patients demographically and clinically, indicating its effectiveness for robust and diverse patient recruitment in clinical studies.

Relevance of GLP-1 receptor agonists or SGLT-2 inhibitors on the recruitment for clinical studies in patients with NAFLD.

Guidelines increasingly recommend the use of glucagon-like peptide-1 receptor agonists (GLP-1 RA) or sodium-glucose co-transporter-2 inhibitors (SGLT2i) to prevent cardiovascular and cardiorenal endpoints. Both drugs also show beneficial effects in nonalcoholic fatty liver disease (NAFLD). Preexisting GLP-1 RA and SGLT2i therapies are frequently defined as exclusion criterion in clinical studies to avoid confounding effects. We therefore investigated how this might limit recruitment and design of NAFLD studies. GLP-1 RA and SGLT2i prescriptions were analyzed in NAFLD patients with diabetes mellitus recruited at a tertiary referral center and from the population-based LIFE-Adult-Study. Individuals were stratified according to noninvasive parameters of liver fibrosis based on vibration-controlled transient elastography (VCTE). 97 individuals were recruited at tertiary care and 473 from the LIFE-Adult-Study. VCTE was available in 97/97 and 147/473 cases.GLP-1 RA or SGLT2i were used in 11.9% of the population-based cohort (LSM < 8 kPa), but in 32.0% with LSM ≥ 8 kPa. In the tertiary clinic, it was 30.9% overall, independent of LSM, and 36.8% in patients with medium and high risk for fibrotic NASH (FAST score > 0.35). At baseline, 3.1% of the patients in tertiary care were taking GLP-1 RA and 4.1% SGLT2i. Four years later, the numbers had increased to 15.5% and 21.6%. GLP-1 RA and SGLT2i are frequently and increasingly prescribed. In candidates for liver biopsy for NASH studies (VCTE ≥ 8 kPa) the use of them exceeds 30%, which needs careful consideration when designing NASH trials.

Patterns of Patient Recruitment in Clinical Studies on the Example of International Multicenter Clinical Studies (IMCTs)

Introduction. Patient recruitment is one of the main quantitative and qualitative characteristics of a clinical trial. As a quantitative characteristic, the recruitment of patients is, first of all, the final figure necessary to answer the researcher's question; as a qualitative characteristic, the recruitment of patients is a process subject to influence. The recruitment of patients in the center is determined by the rate per month and allows you to gain the statistical power of the protocol, and if the recruitment of patients does not reach the planned capacity, then the capacity may not be gained and this leads to the failure of the clinical trial. Failures in patient recruitment reported in the literature lead to more than 80 % of failed clinical trials of all phases. Patient recruitment as a process is influenced by various factors, and the authors identify recruitment predictors, recruitment facilitators, and barriers to patient recruitment.Aim. To consider the general features and patterns of recruitment of patients in clinical trials on the example of international multicenter clinical trials.Materials and methods. We analyzed the quantitative characteristics of patient recruitment in 4 clinical trials with successful recruitment.Results and discussion. Despite the uniqueness of each protocol, the set of patients has its own patterns common to all clinical studies. It was studied, in particular, that the integrative assessment of the preliminary recruitment of patients by the researcher of the clinical center is not associated with an objective pool of patients and affects the rate of patient recruitment and targeted patient recruitment. We found that the number of clinical centers not recruiting patients in each study is at least 30 % of all centers involved. Three patient recruitment rates have also been identified that need to be considered when planning a clinical trial.Conclusion. We have identified patterns in patient recruitment that will help in planning clinical trials.

Breaking Barriers in Dietary Research: Strategies to Diversify Recruitment in Clinical Studies and Develop Culturally Tailored Diets for Hispanic Communities Living With Inflammatory Bowel Disease

Breaking Barriers in Dietary Research: Strategies to Diversify Recruitment in Clinical Studies and Develop Culturally Tailored Diets for Hispanic Communities Living With Inflammatory Bowel Disease

Educational intervention for providers addressing the diversity gap in cervical cancer clinical study participation (579)

Objectives: Every year, over 570,000 women worldwide are diagnosed with cervical cancer. Cervical cancer accounts for 7.5% of female cancer deaths, and 85% of those deaths occur in low- to moderate-income countries. In the US, it is estimated that Black women have a 30% higher incidence of cervical cancer than White women. Additionally, Hispanic women are 40% more likely to be diagnosed with cervical cancer than non-Hispanic women. In 2020, the US Food and Drug Administration issued industry guidance for enhancing the diversity of clinical study participants, highlighting the critical need for representative studies that reflect the current US population demographics. This study aims to conduct an educational intervention aimed at cervical cancer investigators and patients to increase ethnocultural diversity in clinical study recruitment in a cervical cancer research program. Methods: This educational intervention implemented a pre-post study design of clinical investigators participating in both global Phase 2 and Phase 3 cervical cancer studies. The Phase 3 clinical study serves as the parent foundational study to the Phase 2 study (ongoing study, NCT04646005), as 77% of the clinical investigators participating in Phase 2 clinical study overlap with the Phase 3 study. The ongoing Phase 2 study will educate investigators on ethnocultural diversity, and the inclusion of the two studies will be compared from pre-intervention (Phase 3) to post-intervention (Phase 2). Education will be provided as a one-time virtual training and a supplementary pamphlet and will cover the incidence and mortality differences across races, current enrollment gaps, barriers to including diverse populations, and guidance for the broader inclusion of women in our sponsored studies. Race and ethnicity demographic data for the Phase 2 trial (post-intervention) will be compared with the Phase 3 trial (pre-intervention) using the chi-square statistical test. Assuming significant results, a Z-test will then be used to assess the proportions of each race and ethnicity group. Objectives: Every year, over 570,000 women worldwide are diagnosed with cervical cancer. Cervical cancer accounts for 7.5% of female cancer deaths, and 85% of those deaths occur in low- to moderate-income countries. In the US, it is estimated that Black women have a 30% higher incidence of cervical cancer than White women. Additionally, Hispanic women are 40% more likely to be diagnosed with cervical cancer than non-Hispanic women. In 2020, the US Food and Drug Administration issued industry guidance for enhancing the diversity of clinical study participants, highlighting the critical need for representative studies that reflect the current US population demographics. This study aims to conduct an educational intervention aimed at cervical cancer investigators and patients to increase ethnocultural diversity in clinical study recruitment in a cervical cancer research program. Methods: This educational intervention implemented a pre-post study design of clinical investigators participating in both global Phase 2 and Phase 3 cervical cancer studies. The Phase 3 clinical study serves as the parent foundational study to the Phase 2 study (ongoing study, NCT04646005), as 77% of the clinical investigators participating in Phase 2 clinical study overlap with the Phase 3 study. The ongoing Phase 2 study will educate investigators on ethnocultural diversity, and the inclusion of the two studies will be compared from pre-intervention (Phase 3) to post-intervention (Phase 2). Education will be provided as a one-time virtual training and a supplementary pamphlet and will cover the incidence and mortality differences across races, current enrollment gaps, barriers to including diverse populations, and guidance for the broader inclusion of women in our sponsored studies. Race and ethnicity demographic data for the Phase 2 trial (post-intervention) will be compared with the Phase 3 trial (pre-intervention) using the chi-square statistical test. Assuming significant results, a Z-test will then be used to assess the proportions of each race and ethnicity group.

Should informed consent and information related to patient recruitment in clinical trials be available to the reader of scientific articles? A case study in dentistry

ABSTRACT Ethical aspects in research should be transparently reported. This study aimed to investigate whether informed consent and information related to patient recruitment in clinical studies are well reported in the scientific literature. Randomized clinical trials (RCTs) on root coverage procedures published between November 2016 and November 2021 were selected from the PubMed database. Items/questions were used to guide the extraction of data related to patient recruitment, with a focus on the detailed report of informed consent used to clarify the research to the patient. Data were extracted from the published article and the respective research protocol published in a public registry. Information related to potential selective outcome reporting (SOR) was also extracted. In total, 187 documents were initially screened and 74 reports of RCTs were included. No informed consent was published in the article. Only one research protocol provided a link to the informed consent. Deviations from reporting in the research protocol and published article were found, suggesting SOR. Informed consent and information related to patient recruitment in RCTs on root covering procedures are severely underreported. The present findings may stimulate further discussion and debate on the need for making this information publicly available.

Ethical Issues in Social Media Recruitment for Clinical Studies: Ethical Analysis and Framework.

BackgroundSocial media recruitment for clinical studies holds the promise of being a cost-effective way of attracting traditionally marginalized populations and promoting patient engagement with researchers and a particular study. However, using social media for recruiting clinical study participants also poses a range of ethical issues.ObjectiveThis study aims to provide a comprehensive overview of the ethical benefits and risks to be considered for social media recruitment in clinical studies and develop practical recommendations on how to implement these considerations.MethodsOn the basis of established principles of clinical ethics and research ethics, we reviewed the conceptual and empirical literature for ethical benefits and challenges related to social media recruitment. From these, we derived a conceptual framework to evaluate the eligibility of social media use for recruitment for a specific clinical study.ResultsWe identified three eligibility criteria for social media recruitment for clinical studies: information and consent, risks for target groups, and recruitment effectiveness. These criteria can be used to evaluate the implementation of a social media recruitment strategy at its planning stage. We have discussed the practical implications of these criteria for researchers.ConclusionsThe ethical challenges related to social media recruitment are context sensitive. Therefore, social media recruitment should be planned rigorously, taking into account the target group, the appropriateness of social media as a recruitment channel, and the resources available to execute the strategy.

LOW RISK OF SPONTANEOUS MEDICATION-RELATED OSTEONECROSIS OF THE JAW IN FIBROUS DYSPLASIA PATIENTS AFTER ANTIRESORPTIVE THERAPIES: A SYSTEMATIC REVIEW

<h3>Objectives</h3> The objective of this systematic review was to evaluate the risk for spontaneous occurrence of medicated-related osteonecrosis of the jaw (MRONJ) in patients with fibrous dysplasia (FD) and McCune-Albright syndrome (MAS). FD is a rare genetic disorder caused by somatic activating mutations of the GNAS locus encoding the Gsα signaling protein, resulting in reduction of GTPase activity and prolonged activation of Gsα, leading to excessive production of Cyclic adenosine monophosphate (cAMP), which disrupts the ability of bone mesenchymal stem cells to differentiate to mature osteogenic cells. With the increased use of bisphosphonates for the antiresorptive properties, the role of antiresorptives in controlling activity within FD lesions and improvement of bone quality is still inconclusive. <h3>Study Design</h3> A systematic review of the literature was performed by searching PubMed and EMBASE databases using controlled search vocabulary, namely, fibrous dysplasia, McCune-Albright syndrome, jaw, osteonecrosis, antiresorptives, antiresorptive therapy, bisphosphonate, denosumab, teriparatide, estrogen, estrogen therapy, hormone therapy, raloxifene, bazedoxifene, calcitonin, and cathepsin K inhibitor. Articles were limited to human studies, in the English language, in which patients were on antiresorptives for at least 2 years. PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) statement guidelines were used to eliminate nonrelevant studies. The patient, intervention, comparison, outcome, and time (PICOT) question asked was, "Do antiresorptives cause spontaneous occurrence of MRONJ in patients with FD/MAS monitored for at least 2 years of therapy?" <h3>Results</h3> Among the 65 records screened, only 3 articles were eligible for qualitative synthesis based on PRISMA screening criteria. There were no reported cases of spontaneous MRONJ among the patients with FD/MAS across these studies. This is a significant finding among this cohort of patients. <h3>Conclusion</h3> Patients with FD demonstrated low susceptibility to spontaneous occurrence of MRONJ, but more confirmatory studies are still needed. This systematic review was limited by the low number of FD/MAS eligible studies. This could be because of the rare genetic nature of FD/MAS, with low patient recruitment in clinical studies among factors. These pose significant challenges to implementing well-designed randomized placebo-controlled clinical trials that focus on antiresorptive-induced osteonecrosis of the jaw in FD/MAS. As more clinical studies become available, a future follow-up systematic review may provide additional insights into MRONJ complications in patients with FD/MAS treated with antiresorptives.

A Study on the Application and Use of Artificial Intelligence to Support Drug Development

PurposeThe Tufts Center for the Study of Drug Development (CSDD) and the Drug Information Association (DIA) in collaboration with 8 pharmaceutical and biotechnology companies conducted a study examining the adoption and effect of artificial intelligence (AI), such as machine learning, on drug development. The study was conducted to clarify and understand AI adoption across the industry and to gather detailed insights into the spectrum of activities included in the definition of AI. The study investigated and identified analytical platforms and innovations across pharmaceutical and biotechnology companies currently being used or planned for in the future. MethodsA 2-part method was used that comprised in-depth interviews with AI industry experts and a global survey conducted across pharmaceutical and biotechnology organizations. Eleven in-depth interviews focused on use and implementation of AI across drug development. The survey assessed use of AI and included perceptions about current and future use. The survey also examined technology definitions, assessment of organizational and personal AI expertise, and use of partnerships. A total of 402 responses, including data from 217 unique organizations, were analyzed. FindingsAlthough 7 in 10 respondents reported using AI in some capacity, a wide range of use was reported by AI type. Patient selection and recruitment for clinical studies was the most commonly reported AI activity, with 34 respondents currently using AI for this activity. In addition, identification of medicinal products data gathering was the top activity being piloted or in the planning stages, reported by 49 respondents. The study also revealed that the most significant challenges to AI implementation included staff skills (55%), data structure (52%), and budgets (49%). Nearly 60% of respondents noted planned increases in staff within 1–2 years to support AI use or implementation. ImplicationsDespite the challenges to AI implementation, the survey revealed that most organizations use AI in some capacity and that it is important to the success of an organization's workforce. Many organizations reported expectations for increasing staff as implementation of AI expands. Further research should examine the changing development landscape as the role of AI evolves.

Preserving consent-for-consent with feasibility-assessment and recruitment in clinical studies: FARSITE architecture.

Best practice guidance for clinical studies asks investigators to employ the highest possible standards in privacy and consent. When considering the feasibility of a clinical study, issues of privacy extend not only to actual but also to potential study participants. The consent required to access records to determine whether or not an individual might be eligible to participate in a study is sometimes referred to as consent-for-consent. Some initiatives to enhance the efficiency of study-recruitment could compromise consent-for-consent, for example by inviting a patient to take part in a study without the knowledge of their attending clinician. Through iterative working with experts and examination of protocols we explored a range of scenarios for assessing the feasibility of clinical trials and observational studies, and recruiting participants. The main requirement we identified was to speed up feasibility-assessment and recruitment while preserving the patient-clinician trust relationship that is central to consent-for-consent. We present an appropriate information system architecture, FARSITE (Feasibility Assessment and Recruitment System for Improving Trial Efficiency), and show in principle that faster recruitment into clinical studies need not compromise best practice in privacy or consent. We show that FARSITE is a specific instance of an 'e-Lab' architecture for assembling data, methods and expertise around study protocols and defined populations.

Potential Eligibility for Recombinant Tissue Plasminogen Activator Therapy in Children: A Population-Based Study

Intravenous recombinant tissue plasminogen activator is an established therapy for adults with ischemic stroke. In this Greater Cincinnati/Northern Kentucky population-based study, 8% were eligible. However, no established therapy exists for children with acute ischemic stroke. Accordingly, investigators assessed rates of eligibility for recombinant tissue plasminogen activator therapy among children (<18 years of age) in the same population to aid planning of future clinical trials. The investigators identified 29 pediatric ischemic strokes during 3 separate study periods (1993-1994, 1999, and 2005) and determined potential eligibility for recombinant tissue plasminogen activator therapy based on 2007 American Heart Association guidelines for adults. Depending on how relative contraindications were considered, 1 to 3 cases (3%-10%) met eligibility criteria. On the basis of national pediatric stroke incidence rates extrapolated from our population, it is estimated that up to 178 children might be eligible for intravenous recombinant tissue plasminogen activator therapy annually in the United States. Thus, recruitment for clinical studies is likely to be challenging and requires a concerted multicenter effort.

Die richtige Rekrutierung von Studienteilnehmern

Patient recruitment in clinical studies is an essential step early in a drug trial. The quality of this process determines the safety of the trial and may modulate the validity of the resulting scientific information. While there are good reasons to recruit participants as quickly as possible to accelerate drug development and promote new treatment options, it is even more important to safeguard health and well-being of the participant. Therefore all steps of recruitment from patient identification and patient availability to patient information and enrolment have to be tailored accordingly. This paper describes the numerous challenges in this task, pitfalls and risks that may jeopardize reliable and effective recruitment, and strategies to improve this important process.

Comparison of two screening methods for in-house genotyping in clinical pharmacology units

Two genetic screening methods, the fluorescence resonance energy transfer (FRET) technique on the LightCycler and the real-time pyrophosphate detection technique on the Pyrosequencer have been compared with regard to their usefulness as screening methods for subject recruitment in clinical studies in pharmacology units. Two SNPs of possible clinical relevance were selected, namely the 118A>G SNP of the OPRM1 gene and the 3435C>T SNP of the ABCB1 gene. Genotypes diagnosed using conventional sequencing served as control. The allelic frequency of the mutated 118G allele of the OPRM1 gene was 12.7% and that of the mutated 3435T allele of the ABCB1 gene was 50.7%. All results obtained with the Pyrosequencer were in accord with those obtained using conventional sequencing. With the LightCycler, an incorrect genotype was assigned to 1 of the 130 DNA samples corresponding to an error rate of 0.8%. Although both methods were found suitable for rapid SNP detection, Pyrosequencing was the preferred method since it provides the nucleotide sequence directly thus facilitating interpretation.