Direct-to-Patient Mobile Teledermoscopy: Prospective Observational Study.

Direct-to-patient mobile teledermoscopy is a feasible and useful adjunct to smartphone imaging for monitoring patient-identified lesions of concern, achieving comparable diagnostic and management accuracy as in-office dermatology.

Direct-to-participant investigational medicinal product supply in clinical trials in Europe: Exploring the experiences of sponsors, site staff and couriers.

AIMS: Insights into the current practice of direct-to-participant (DtP) supply of investigational medicinal product (IMP) in the context of clinical trials conducted in Europe are needed, as regulations are unharmonized. This study is set out to explore how DtP IMP supply has been employed in Europe and what the advantages and disadvantages and barriers and facilitators of its implementation are. METHODS: We conducted semi-structured interviews with representatives from sponsor companies, courier services and site study staff involved in the IMP dispensing and delivery process in Europe. Interviews were conducted between May and November 2021, and data were analysed following thematic analysis. RESULTS: Sixteen respondents participated in one of the 12 interviews. Respondents had experience with different models of DtP IMP supply including shipment from the investigative site, a central pharmacy (a depot under the control of a pharmacist) and a local pharmacy-aiming to reduce trial participation burden. The respondents indicated that investigative site-to-participant shipment is not affected by regulatory barriers, but could burden site staff. Shipment from central locations was considered most efficient, but possible regulatory barriers related to maintaining participants' privacy and investigator oversight were identified. The respondents indicated that the involvement of local pharmacies to dispense IMP can be considered when the IMP is authorized. CONCLUSIONS: Several DtP IMP supply models are implemented in clinical trials conducted in Europe. In this study, three main DtP IMP models were identified, which can be referenced when describing these approaches for regulatory approval.

Decentralised, patient-centric, site-less, virtual, and digital clinical trials? From confusion to consensus.

There is increasing interest in clinical trials that use technologies and other innovative operational approaches to organise trial activities around trial participants instead of investigator sites. A range of terms has been introduced to refer to this operational clinical trial model, including virtual, digital, remote, and decentralised clinical trials (DCTs). However, this lack of standardised terminology can cause confusion over what a particular trial model entails and for what purposes it can be used, hampering discussions by stakeholders on its acceptability and suitability. Here, we review the different terms described in the scientific literature, advocate the consistent use of a unified term, 'decentralised clinical trial,' and provide a detailed definition of this term.

Patient and Clinician Satisfaction in Teledermatology: Key Factors for Successful Implementation.

Purpose of review: Teledermatology has emerged as a promising solution for remote dermatologic care, especially during COVID-19 pandemic. It improves access to care through information and communication technologies. This paper explores patient and clinician satisfaction in teledermatology. Recent findings: Patient satisfaction encompasses various aspects, including future willingness, the quality of care, technical quality, and access to care. Clinician satisfaction is influenced by quality of care, implementation, technical aspects, clinician-patient rapport, and financial considerations. It is important to evaluate patient and clinician satisfaction in different teledermatology models, including store-and-forward, live interactive, and hybrid interactive approaches. Summary: By evaluating satisfaction within different teledermatology models and addressing the factors that impact satisfaction, teledermatology can ensure high-quality care and promote positive outcomes. With proper implementation and ongoing evaluation, teledermatology has the potential to become a widely accepted and valuable component of dermatologic care, offering enhanced access to specialized services.

Accuracy Assessment of Outpatient Telemedicine Encounters at an Academic Ophthalmology Department.

Purpose We assess the clinical accuracy of direct-to-patient real-time outpatient video visit encounters at our eye center. Design This was a retrospective longitudinal study. Subjects and Methods Patients who completed a video visit over a 3-week period between March and April 2020 were included. Accuracy assessment was determined by comparing diagnosis and management from the video visit with subsequent in-person follow-up over the next year. Results A total of 210 patients (mean age 55±18 years) were included, of whom 172 (82%) were recommended a scheduled in-person follow-up encounter after their video visit. Among the 141 total patients who completed in-person follow-up, 137 (97%) had a diagnostic agreement between telemedicine and in-person evaluation. Management plan agreed for 116 (82%), with the remainder of visits either escalating or deescalating treatment upon in-person follow-up with little substantive change. Compared with established patients, new patients had higher diagnostic disagreement following video visits (12 vs. 1%, p =0.014). Acute visits trended toward more diagnostic disagreement compared with routine visits (6 vs. 1%, p =0.28) but had a similar rate of management change on follow-up (21 vs. 16%, p =0.48). New patients were more likely to have early unplanned follow-up than established patients (17 vs. 5%, p =0.029), and acute video visits were associated with unplanned early in-person assessments compared with routine video visits (13 vs. 3%, p =0.027). There were no serious adverse events associated with the use of our telemedicine program in the outpatient setting. Conclusions Video visits had high diagnostic and management agreement with subsequent in-person follow-up encounters.

An industry survey of implementation strategies for clinical supply chain management of cell and gene therapies.

BACKGROUND AIMS: The novelty of cell and gene therapies (CGTs) has introduced unique supply chain challenges and considerations not seen by chemically synthesized (small-molecule) drugs. These complexities increase during the clinical phases, where drug safety and efficacy milestones are still underdeveloped. For example, for autologous therapies such as chimeric antigen receptor T-cell therapies, in which the treatment is developed from the patient's own cells, supply chain management plays an integral role in chemistry, manufacturing and control processes. Supply chain management requires proactive planning because of the strict cold chain requirements and time sensitivity of CGTs. This research examines strategies and responses to challenges experienced by industry stakeholders (e.g., sponsors and manufacturers) during the implementation phases of clinical supply chain management. This research further evaluates the adequacy of the current regulatory framework for distribution and supply chain management of CGTs in the US. METHODS: A survey methodology was used to query subject matter experts from the biopharmaceutical industry who were familiar with the clinical supply management of CGTs in the US. The survey instrument was developed using an implementation framework and disseminated electronically to mid- and senior-level subject matter experts who had experience with clinical trials, supply chain management and CGTs. RESULTS: A total of 128 respondents accessed the survey, and 105 respondents answered at least one question. Seventy-five respondents completed the survey. Results showed that a lack of harmonization in regulations across the supply chain, limited resources, challenges with vendor management, high costs and complexities in the supply chain due to product specificity and customization proved to be impediments for the industry. In addition, the coronavirus disease 2019 pandemic had a significant impact on supply chain implementation. The results revealed that less than half of the respondents had business continuity plans in place. These challenges increased for smaller and mid-size organizations. Thirty percent of small and mid-size organizations were less prepared to scale up than larger companies. CONCLUSIONS: Suggestions from industry stakeholders were to adopt and enforce Good Distribution Practices in the US (81%), pre-plan distribution strategies with internal and external stakeholders along the supply chain and develop agile systems and robust processes end to end. Hurdles in scaling up and scaling out from the clinical to commercial phases for time- and temperature-sensitive CGT products make it difficult to predict the supply chain's long-term feasibility. Although there are initiatives to improve these impediments, such as improving industry partnerships and creating global CGT transportation standards, there are still regulatory knowledge gaps present for CGTs. Therefore, it is essential to establish a baseline and foundation for CGT supply chains extending beyond the loading dock.

A Framework for Extension Studies Using Real-World Data to Examine Long-Term Safety and Effectiveness.

Understanding the long-term benefits and risks of treatments, devices, and vaccines is critically important for individual- and population-level healthcare decision-making. Extension studies, or 'roll-over studies,' are studies that allow for patients participating in a parent clinical trial to 'roll-over' into a subsequent related study to continue to observe and measure long-term safety, tolerability, and/or effectiveness. These designs are not new and are often used as an approach to satisfy regulatory post-approval safety requirements. However, designs using traditional clinical trial infrastructure can be expensive and burdensome to conduct, particularly, when following patients for many years post trial completion. Given the increasing availability and access of real-world data (RWD) sources, direct-to-patient technologies, and novel real-world study designs, there are more cost-efficient approaches to conducting extension studies while assessing important long-term outcomes. Here, we describe various fit-for-purpose design options for extension studies, discuss related methodological considerations, and provide scientific and operational guidance on practices when planning to conduct an extension study using RWD. This manuscript is endorsed by the International Society for Pharmacoepidemiology (ISPE).

Identification of a Vulnerable Group for Post-Acute Sequelae of SARS-CoV-2 (PASC): People with Autoimmune Diseases Recover More Slowly from COVID-19.

PURPOSE: Evidence is emerging that a significant percentage of COVID-19 cases experience symptom persistence beyond 30 days and go on to develop post-acute sequelae. Our objective was to compare the risk for COVID-19 symptom persistence by self-reported use of medications for autoimmune disease among participants of an on-line COVID-19 registry. PATIENTS AND METHODS: A community-based online survey collected weekly data on COVID-19 symptom presentation. Participants who completed informed consent online, reported a positive COVID-19 test result within 14 days prior to enrollment and also reported demographics, underlying illnesses, and medication use were included. Symptom presence and severity were evaluated weekly after enrollment and compared between participants reporting use of medications for autoimmune conditions and all others. Logistic regression was used to evaluate the odds of more severe acute illness and symptom persistence approximately 30 days after enrollment. RESULTS: A total of 1,518 COVID-19-positive participants were included. Participants reporting use of medications for autoimmune disease (n=70) were more likely to have experienced symptoms at all time points over a 30-day time period and were more likely to report more severe presentation of COVID-19 during acute illness (adjusted OR (95% CI)=1.32 (0.76-2.29)) compared to those reporting not taking medications for autoimmune disease. At about 30 days after enrollment, users of medications for autoimmune disease were more than twice as likely to report three or more symptoms (adjusted OR (95% CI)=2.53 (1.21-5.29)). In particular, their risk of persistent shortness of breath and fatigue was elevated (adjusted OR (95% CI)=2.66 (1.15-6.18) and 4.73 (2.17-10.34), respectively). CONCLUSION: Individuals with underlying autoimmune conditions appear to be particularly vulnerable to post-acute sequelae from COVID-19; early intervention might be considered.

Enhancing Patient Centricity and Advancing Innovation in Clinical Research with Virtual Randomized Clinical Trials (vRCTs).

The current public health crisis has highlighted the need to accelerate healthcare innovation. Despite unwavering levels of cooperation among academia, industry, and policy makers, it can still take years to bring a life-saving product to market. There are some obvious limitations, including lack of blinding or masking and small sample size, which render the results less applicable to the real world. Traditional randomized controlled trials (RCTs) are lengthy, expensive, and have a low success rate. There is a growing acknowledgement that the current process no longer fully meets the growing healthcare needs. Advances in technology coupled with proliferation of telehealth modalities, sensors, wearable and connected devices have paved the way for a new paradigm. Virtual randomized controlled trials (vRCTs) have the potential to drastically shorten the clinical trial cycle while maximizing patient-centricity, compliance, and recruitment. This new approach can inform clinical trials in real time and with a holistic view of a patient's health. This paper provides an overview of virtual clinical trials, addressing critical issues, including regulatory compliance, data security, privacy, and ownership.

ACCELERATE: A Patient-Powered Natural History Study Design Enabling Clinical and Therapeutic Discoveries in a Rare Disorder.

Geographically dispersed patients, inconsistent treatment tracking, and limited infrastructure slow research for many orphan diseases. We assess the feasibility of a patient-powered study design to overcome these challenges for Castleman disease, a rare hematologic disorder. Here, we report initial results from the ACCELERATE natural history registry. ACCELERATE includes a traditional physician-reported arm and a patient-powered arm, which enables patients to directly contribute medical data and biospecimens. This study design enables successful enrollment, with the 5-year minimum enrollment goal being met in 2 years. A median of 683 clinical, laboratory, and imaging data elements are captured per patient in the patient-powered arm compared with 37 in the physician-reported arm. These data reveal subgrouping characteristics, identify off-label treatments, support treatment guidelines, and are used in 17 clinical and translational studies. This feasibility study demonstrates that the direct-to-patient design is effective for collecting natural history data and biospecimens, tracking therapies, and providing critical research infrastructure.

Self-reported symptoms from exposure to Covid-19 provide support to clinical diagnosis, triage and prognosis: An exploratory analysis.

BACKGROUND: Symptomatic COVID-19 is prevalent in the community. We identify factors indicating COVID-19 positivity in non-hospitalized patients and prognosticators of moderate-to-severe disease. METHODS: Appeals conducted in April-June 2020 in social media, collaborating medical societies and patient advocacy groups recruited 20,476 participants ≥18 years who believed they had COVID-19 exposure. Volunteers consented on-line and reported height, weight, concomitant illnesses, medication and supplement use, residential, occupational or community COVID-19 exposure, symptoms and symptom severity on a 4-point scale. Of the 12,117 curated analytic population 2279 reported a COVID-19 viral test result: 865 positive (COVID+) and 1414 negative (COVID-). RESULTS: The triad of anosmia, ageusia and fever best distinguished COVID+ from COVID-participants (OR 6.07, 95% CI: 4.39 to 8.47). COVID + subjects with BMI≥30, concomitant respiratory disorders or an organ transplant had increased risk of moderate-to- severe dyspnoea. Race and anti-autoimmunity medication did not affect moderate-to-severe dyspnea risk. CONCLUSIONS: The triad of anosmia, ageusia and fever differentiates COVID-19. Elevated risks of severe symptoms outside the hospital were most evident among the obese and those with pulmonary comorbidity. Race and use of medication for autoimmune disease did not predict severe disease. These findings should facilitate rapid COVID-19 diagnosis and triage in settings without testing.

Challenges and opportunities for sponsors in conducting clinical trials during a pandemic.

The COVID-19 pandemic may impact the conduct of clinical trials of medical products. Challenges have arisen, from country/ state lockdowns, site closures due to hospitals being taken over / sites being taken over for COVID-19 related care, travel limitations to sites for patients, interruptions to the supply chain for the investigational product, or other considerations if site personnel or trial subjects become infected with COVID-19. These challenges may lead to difficulties in meeting protocol-specified procedures, including administering or using the investigational product or adhering to protocol-scheduled visits and laboratory/diagnostic testing. This position paper from the perspective of Indian Society for Clinical Research (ISCR) aims to provide guidance to both frontline Clinical Research Professionals and sponsors on measures that can be taken while continuing ongoing clinical trial activities at site as well as resuming site level activities in the post COVID setting. Broad guidance is also given to sites and sponsors on use of Direct to Patient drug shipments, supplies and cold chain management and use of technologies to support enhanced remote functioning during and post COVID.

Patients Want to Meet With Imaging Experts.

PURPOSE: The evolving health care landscape demands a multidisciplinary approach to care and increased emphasis on patient engagement. Radiologists may play an important role in this respect, by improving patient agency and understanding through direct patient interaction. The purpose of this study is to assess patient preferences and perceptions regarding direct communication with radiologists, focusing on a medically underserved demographic. METHODS: Our institutional review board approved this HIPAA-compliant study. English- and/or Spanish-speaking adults undergoing imaging studies across two tertiary-care hospitals and a busy outpatient center were surveyed over a consecutive two-month period, to assess knowledge of the role of a radiologist, interest in, and perceived benefits of reviewing examination results with imaging experts. RESULTS: A total of 628 surveys were completed: 20% of total respondents accurately identified the role of radiologists; 28% identified radiologists as doctors. A total of 68% of respondents expressed an interest in receiving imaging results immediately, or whenever results became available, regardless of when referring physicians received results. An interest in reviewing imaging results with an expert was indicated by 81% of respondents; 88% anticipated that reviewing imaging results with an expert would be beneficial. Although 58% of respondents indicated that they might be willing to pay out of pocket for such a service, 50% selected $0 when asked to indicate an amount. CONCLUSIONS: Although most patients do not know the role of radiologists, they want immediate results, and perceive value in consulting directly with imaging experts.

Patient centric approach for clinical trials: Current trend and new opportunities.

The clinical research industry today is undergoing a major facelift. Companies are continuously looking to adopt and implement effective and innovative ways to accelerate drug launches in the market. Companies today are more open and do not view patients as mere "subjects" who generate data, - but as informed collaborators whose participation is "core" to the overall success of trials leading to the emergence of the concept of "patient-centric trials." This paper is intended to highlight the current trends and new opportunities that can be seen in industry -indicative of crucial role patients today play in their own health care using technology, social media and self education.

Direct-to-Patient Research: Piloting a New Approach to Understanding Drug Safety During Pregnancy.

BACKGROUND: Little is known about the effects of human fetal exposure when a new drug is authorized unless it was specifically developed for use in pregnancy. Since many factors may contribute to adverse fetal effects, having comprehensive information about in utero exposures will enhance our ability to make correct determinations about causality. OBJECTIVE: The objective of the study was to assess the extent to which women, recruited without the intervention of health care professionals (HCPs), will provide information, suitable for research purposes, via the Internet or by phone on some potential risk factors in pregnancy. METHODS: To pilot direct-to-patient research for pharmacovigilance, we conducted a prospective, noninterventional study of medication use and lifestyle factors as part of the Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium (PROTECT) Consortium. Consenting women who self-identified as pregnant and residing in the United Kingdom (UK), Denmark (DK), The Netherlands, or Poland were recruited and could then choose to provide data every 2 or 4 weeks via the Internet or a telephonic interactive voice response system (IVRS). Self-reported drug use was compared with pharmacy register data in DK and with electronic health records in the UK. RESULTS: Recruited women were on average older and more highly educated than the general population. Most respondents chose a frequency of every 4 weeks (56.99%, 1177/2065). Only 29.83% (464/1555) of women with due dates occurring during the study provided information on pregnancy outcome. For those responding by Internet, over 90.00% (1915/2065) reported using >1 pregnancy-related medication, 83.34% (1721/2065) reported using >1 other medicine, and 23.53% (486/2065) reported only over-the-counter medications, not counting herbals and dietary supplements. Some respondents (7.16%, 148/2065) reported that they chose not to take a prescribed medication (mostly medicines for pain or inflammation, and for depression) and 1.30% (27/2065) reported using medicines that had been prescribed to a friend or family member (oxycodone, paracetamol, and medications for acid-related problems). Relatively few respondents reported using fish oil (4.60%, 95/2065), other dietary supplements (1.88%, 39/2065), herbal products (7.07%, 146/2065), or homeopathic products (1.16%, 24/2065). Most medications for chronic conditions that were listed in the Danish prescription registry were also self-reported (83.3%, 145/174 agreement), with larger discrepancies for medications indicated for short-term use (54.0%, 153/283 agreement) and pregnancy-related medications (66.1%, 78/118). CONCLUSIONS: Self-reported information on medication use as well as other potential teratogenic factors can be collected via the Internet, although recruitment costs are not insubstantial and maintaining follow-up is challenging. Direct data collection from consumers adds detail, but clinical input may be needed to fully understand patients' medical histories and capture birth outcomes.

Analysis of Direct-to-patient Model in Pharmacy Retail Field / 中国药师

Objective:To analyze the direct-to-patient( DTP)model in the field of pharmacy retail. Methods:The meaning and features of the model were analyzed,the advantages and challenges of the model were summarized,and some countermeasure sugges-tions for the model application in the pharmacy retail field were provided. Results:The DTP model can promote the development of drug retail enterprises. However,there are still some challenges needing continue exploration in practice. Conclusion:In order to pro-mote the application and development of the DTP model,drug retail enterprises demand the policy adjustment by government,increased investment,enhancement of centralized development and implementation of new business forms.

Lab Tests Online and consumer understanding of laboratory testing.

Lab Tests Online is a "peer-reviewed, non-commercial, patient-centered" resource where patients and their relatives and caregivers can learn about the tests used to screen for, diagnose, and manage disease. Consumers are becoming increasingly involved in the management of their own health care and increasingly have access to their laboratory results through electronic health records. Research has shown that consumers have difficulty with health literacy in general and with numerical data in particular. The Lab Tests Online global websites are an important step toward helping consumers understand the complexity of the pathology process, the expertise of the people involved and the meaning of the results provided to them and their healthcare professionals.