Patient Access Research Articles

Specific Recommendations for the Evaluation of Orphan Drugs in Spain: The EvalRare project

Introduction and Objective: Rare diseases (RDs) are low-prevalence conditions that significantly impact patients’ lives, often requiring orphan drugs (ODs) for treatment. This study aims to establish a national framework for assessing the quality of clinical evidence for ODs in Spain, to better manage associated uncertainties and support informed decision-making by stakeholders. Materials and Methods: A qualitative study using focus group methodology was carried out. A review was conducted to identify evaluation criteria from Therapeutic Positioning Reports (TPRs) by the Spanish Agency for Medicines and Health Products (AEMPS) and the GENESIS group of the Spanish Society of Hospital Pharmacy (SEFH). A multidisciplinary focus group of experts from national, regional, and hospital levels discussed and refined these criteria, proposing new variables and reaching consensus on final recommendations. Results: Key evaluation criteria were identified, including study design, comparison type, primary clinical endpoints, study population, and study duration. Recommendations were categorized by acceptability, addressing the specific challenges in OD development. While randomized clinical trials remain the gold standard, alternative designs, such as uncontrolled non-randomized cohort studies, may be acceptable under certain conditions, particularly when there is a significant unmet medical need and no alternative therapies. Both clinical endpoints and validated quality of life metrics are crucial in assessing ODs. Smaller sample sizes and shorter study durations may be acceptable if justified by the disease’s characteristics and the clinical relevance of the outcomes. Conclusion: The proposed framework offers a tailored guide for evaluating the clinical evidence of ODs, providing recommendations that may enhance decision-making by stakeholders for determining patient access to OD treatments. Key words: orphan drugs, evaluation, research methodology, health decision-making, recommendations, rare diseases.

Surgeon-Led Point-Of-Care UltraSound-guided THORacic (POCUS-THOR) Biopsy: A New Paradigm in Efficient Diagnosis and Resource-Sparing Care

Tissue diagnosis through a variety of interventional approaches guides thoracic cancer management, but often introduces delay to definitive treatment and can be resource intensive. We introduced a thoracic surgeon-led, point-of-care ultrasound-guided biopsy program to provide rapid diagnosis for patients with thoracic cancers. We assessed the diagnostic yield and adverse events with this approach. A prospective cohort study was performed of consecutive patients undergoing ultrasound-guided biopsies performed by 5 thoracic surgeons from June 2021 to April 2024 at a tertiary Canadian thoracic surgery institution. Using a bedside ultrasound, 20-gauge tissue cores were obtained using multiple passes with a standard spinal needle. Descriptive univariable statistics were used. 160 patients underwent bedside biopsy for lung (n=101), liver (n=20), chest wall/pleural (n=20), mediastinal (n=18), or other (n=1) lesions. Tissue diagnosis was obtained in 86.3% of patients (n=138), and diagnostic yield was similar for high and low-volume providers and over time. All liver biopsies were diagnostic. Non-diagnostic biopsies were more likely to occur with benign pathology, chest wall/pleural lesions, or extensive necrosis; diagnosis was achieved with other modalities in most cases. There was 1 post-procedure pneumothorax (adverse event rate 0.6%). Thoracic surgeon-led ultrasound-guided biopsies are safe in an outpatient clinic setting, and have high diagnostic accuracy. This results in reduced time to diagnosis by an estimated 28-35 days, and frees up endoscopic and radiology resources for other patients. This low-cost procedure can be adopted as part of comprehensive thoracic malignancy assessment, and can accelerate patient access to cancer treatment.

HPR55 Analysis of Leadtimes and Factors Impacting Leadtimes in Reimbursement Processes in the Netherlands to Estimate and Optimize Time Till Patient Access

HPR55 Analysis of Leadtimes and Factors Impacting Leadtimes in Reimbursement Processes in the Netherlands to Estimate and Optimize Time Till Patient Access

Developing a toolkit for building a community hospital clinical research program.

Although health research in Canada is primarily conducted in academic hospitals, most patients receive their care in community hospitals. The benefits of increasing research capacity in community hospitals include improved study recruitment, increased generalizability of results, broader patient access to novel therapies, better patient outcomes, enhanced staff satisfaction, and improved organizational efficiency. Nevertheless, building research programs in community hospitals remains challenging because of a lack of support and expertise. To address this gap, we developed a toolkit to help community hospital professionals build and sustain their community hospital research programs. The toolkit was developed by the Canadian Community Intensive Care Unit Research Network (CCIRNet), a group of clinician-researchers and research staff from community hospitals across Canada who have experience building community hospital research programs. Feedback from a concurrent qualitative study of Canadian community critical care professionals informed the toolkit's design. The CCIRNet toolkit outlines five stages of community hospital clinical research program development: 1) building a research team and gaining support, 2) developing a new research program, 3) choosing a first research study, 4) getting the study up and running, and 5) sustaining a research program. Feedback from qualitative interviews emphasized the need for a step-by-step approach, frequently asked questions, and essential resources. Accordingly, each stage is structured in a question-and-answer format and includes relevant resources for each section. The CCIRNet toolkit is a practical resource for establishing research programs in community hospitals. The toolkit may increase research participation and support clinical research capacity building in community hospitals.

PCR111 European Atlas on Clinical Trials in Cancer and Hematology (EuroACT): Patient Access Disparities and Underuse of Patient Reported Outcome Measures

PCR111 European Atlas on Clinical Trials in Cancer and Hematology (EuroACT): Patient Access Disparities and Underuse of Patient Reported Outcome Measures

HTA344 An Analysis of Excuses: Achieving Patient Access on Day One Following European Commission Authorization

HTA344 An Analysis of Excuses: Achieving Patient Access on Day One Following European Commission Authorization

Healthcare Monitoring-based Internet of Things (IOT)

Remote health care is needed when direct medical monitoring is unavailable. Modern technology provides several ways to make patient access simpler. In particular, cloud, IoT, and data mining technologies have been successful in health care and medicine. This method identifies illnesses using data collected from two groups of patients, a male group and a female group where data are based on the heart rate, systolic blood pressure, and body temperature parameters. The three classifier types use the collected data: the naïve Bayes, support vector machine (SVM), and the J48. These three classification methods were considered to measure the classification accuracy using the proposed healthcare IOT-based monitoring system. The Precision, Recall, and F-measure evaluation measurements were used to quantify the obtained accuracy results of the two groups. The obtained results showed excellent classification results of 0.96, 0.99, and 0.98 for the naïve Bayes classifier to the male group that outperformed the results of the other two classifiers for the precision, recall, and f-measure, respectively. As for the female group, similar results have been obtained. In conclusion: The use of the Internet of Things in the proposed system makes it accurate and easy to use remotely by patients.

Fluid–structure interaction in a follow-up study of arterio-venous fistula maturation

Arteriovenous fistula (AVF) is the preferred vascular access for hemodialysed patients. AVF is created surgically using the patient’s artery and vein. Once the connection (anastomosis) is made, the maturation process begins. Studies have shown that most AVFs do not survive beyond one year. This study presents fluid–structure interaction (FSI) modelling of non-Newtonian blood flow through an end-to-side radio-cephalic AVF, investigated weekly during a 15-week follow-up period and 1.5 years postoperatively using ultrasound methods. The aim was to collect qualitative and quantitative data regarding changes in hemodynamics and alterations in the walls of AVF vasculature. Different material properties were assigned to the artery, suture zone (anastomosis), and vein, while the stiffening of the venous arm over time was also modelled. The proposed FSI methodology can be implemented in future follow-up studies involving groups of patients. The main findings revealed: a) counter-rotating vortices in the anastomosis cross-section affecting local pressure conditions; b) different temporal progression of vorticity, shear strain rate, and turbulent kinetic energy and similarity of the temporal progression of WSS obtained under the assumptions of the rigid-walled and FSI; c) a negligible low-WSS zone in the presented thrombosis-free AVF; d) migration of the zone of maximal temporal wall deformation over time.

Geospatial Mapping of Head and Neck Cancer Research: Assessing Access, Disparities, and Characteristics of Head and Neck Cancer Clinical Trials Across the United States.

To report geographic distribution and characteristics of head and neck cancer (HNC) clinical trials in the United States. We conducted a retrospective analysis of U.S. HNC clinical trials searching ClinicalTrials.gov from January 1, 2017 to December 31, 2021 using the terms "head and neck cancer" or "head and neck neoplasms." A total of 381 clinical trials met inclusion criteria with 2181 trial opportunities, which were correlated with population density. Of the U.S. population, 72% live within a 25-mile radius of trials. California, Pennsylvania, and New York had the greatest number of clinical trial entries. The majority of patients living more than 25 miles from an HNC clinical trial site are located in rural areas. One hundred sixty-five (43.3%) trials were about systemic therapy, of which 138 (83.6%) involved targeted immunotherapy. There were 286 unique principal investigators. One hundred six (37.1%) were females and 180 (62.9%) were males. We demonstrate disparity in the geographic distribution of HNC trials favoring densely populated urban areas, which may limit patient access due to travel burden. Studies are skewed towards immunotherapy drug trials, with fewer radiation and surgery investigations. Level III.

Evolving Landscape of Sickle Cell Anemia Management in Africa: A Critical Review.

Sickle cell disease (SCD) is a prevalent inherited blood disorder, particularly affecting populations in Africa. This review examined the disease's burden, its diverse clinical presentations, and the challenges associated with its management in African settings. Africa bears a significant burden of SCD, with prevalence varying across countries and age groups. Newborn screening programs have highlighted the high prevalence of SCD at birth, emphasizing the need for early diagnosis and intervention. The clinical manifestations of SCD in Africa are multifaceted, encompassing acute complications like vaso-occlusive crises, acute chest syndrome, and stroke, as well as chronic complications such as organ damage and leg ulcers. Biological factors, including fetal hemoglobin levels, and demographic factors, like age and sex, influence disease severity and outcomes. The management of SCD in Africa faces numerous challenges. Limited access to resources, including diagnostic tools, medications, and trained healthcare professionals, hinders optimal care. The high cost of advanced therapies further restricts patient access. Cultural stigma and a lack of awareness create additional barriers to effective management. To address these challenges, early diagnosis through newborn screening programs and point-of-care testing is crucial. Comprehensive care models, including hydroxyurea therapy, pain management, and patient education, are essential for improving outcomes. Collaboration with international networks and leveraging local resources can enhance the sustainability of SCD programs. In conclusion, SCD significantly impacts African populations. Overcoming the challenges associated with its management requires addressing resource limitations, affordability issues, and cultural barriers. Early diagnosis, comprehensive care models, and ongoing research focused on affordability and accessibility are crucial for improving the lives of individuals living with SCD in Africa.

A qualitative study of attitudes and perceptions of smoking cessation medication among patients with cancer.

Among patients with cancer, tobacco use remains high. Nearly half who report smoking at diagnosis continue to smoke following diagnosis, and use of smoking cessation medication remains low. This studyexplores attitudes, barriers, and preferences for smoking cessation medication among patients enrolled in a tobacco treatment trial in the context of cancer care. This is a secondary qualitative analysis of the Smokefree Support Study. Of 221 participants who completed the 6-month follow-up survey, n = 72 participants were randomly selected for an exit interview. Interviews were conducted using a semi-structured interview guide, recorded, transcribed, and individually coded using NVivo 11 until high interrater reliability was reached (Kappa > 0.86). Participants (55.6% female) were predominantly White (83.3%) and on average 60years old. Many participants held negative beliefs about smoking cessation medications, primarily concerns about safety and side effects in the context of cancer treatment. Lack of understanding interactions with ongoing chemotherapy and access were the most common barriers reported by those who did not use cessation medication during the study. For those who did use medication during the study, positive outcomes included craving reduction, ease of use and access, and psychological benefits. The findings suggest a knowledge gap in the safety and effectiveness of smoking cessation medications, specifically in relation to cancer care. Addressing this knowledge gap may increase medication uptake, adherence, and quit rates. Cancer care providers are seen as instrumental in emphasizing the importance and safety of smoking cessation medications as well as patient access concerns. Tobacco use among patients with cancer remains high and has serious clinical implications. Tobacco cessation is associated with decreased treatment-related toxicity, decreased risk of second primary cancers, and increased survival rates. Smoking cessation medications are effective, yet patients with cancer have low usage rates. Elucidating reasons associated with smoking cessation medication nonadherence will assist cancer care providers in better addressing factors that increase adherence rates.

Current gaps in acute reperfusion therapies.

Advances in intravenous thrombolysis and endovascular thrombectomy have significantly reduced disability and improved outcomes associated with acute ischemic stroke. An expansion of indications for reperfusion therapies in select groups of patients to permit treatment in an extended time window, with large ischemic core, and with simplified imaging protocols have enabled a broader group of patients access to disability-sparing therapy. Cerebroprotection has had renewed development in the era of acute reperfusion. In this review, we highlight recent developments in stroke reperfusion research and related questions that are under study or remain unanswered.

Use of advanced systemic therapy in patients with moderate-to-severe atopic dermatitis in the TARGET-DERM AD Registry

Background Moderate-to-severe atopic dermatitis (AD) significantly impacts quality of life. Advanced systemic therapeutics (AST) represent a new generation of medications targeting AD pathogenesis, but many who may benefit from these medications are AST-naïve. We compared patients in the United States who had started AST with those who had not started AST to evaluate associated characteristics. Methods TARGET-DERM AD, (NCT03661866, “A Longitudinal Observational Study of Patients Undergoing Therapy for IMISC (TARGET-DERM)” launched in 2019, is an ongoing, longitudinal, observational study of patients managed at 37 United States sites. Patients were aged 12 years and above, had moderate-to-severe AD based on validated Investigator Global Assessment (vIGA) at enrollment, at least one follow-up visit post-enrollment, and treatment with any of the following: a topical/systemic corticosteroid, immunomodulator, or phototherapy. AST included dupilumab and upadacitinib. Variables of interest gathered at enrollment included demographics, vIGA and Body Surface Area (BSA), patient-reported outcomes, and all recorded therapeutics. Results Of 3,076 patients, 436 qualified for inclusion, 52 were AST-treated adolescents and 141 AST-treated adults. Both groups had increased likelihood of AST initiation if they had private insurance and higher BSA, vIGAxBSA, or Patient-Oriented SCORing Atopic Dermatitis scores. Adults were more likely to start AST based on minority/ethnicity, more severe vIGA, higher patient-reported outcomes, or if treated at a community clinic. Substantial numbers of adolescent and adult patients (47 and 58%, respectively) with severe disease were AST-naïve. Conclusions Disease severity and patient access to AST are major factors driving AST initiation. However, some patients are undertreated. This analysis supports AD patient advocacy for those inadequately managed with conventional therapies. Further investigations are necessary to delineate AST initiation barriers and relevant outcomes.

Telehealth in Acute Care: Pay Parity and Patient Access

Telehealth in Acute Care: Pay Parity and Patient Access

The experience of implementing a social health insurance programme at health facilities in Khyber-Pakhtunkhwa, Pakistan

Objective:To explore the experiences of key stakeholders, including hospital managers, clinicians, and hospital-based StateLife Insurance Corporation (SLIC) representatives, SCP's implementation challenges and successes.Methods: A qualitative descriptive exploratory design was employed across 38 empanelled public and private facilities inKP. Key informant interviews (n=103) were conducted until data saturation was reached, exploring barriers and facilitatorswithin the SCP's operational framework. Data were analyzed using NVivo software, applying a mix of deductive and inductiveapproaches.Results: Strengths and enablers included a comprehensive benefits package design, a robust third-party administrativesystem, and the digitization of health management information systems for efficiency. Implementation barriers includedlow reimbursement rates affecting service quality at health facilities, documentation issues hindering patient access, unevenavailability of services causing patient frustration, resource-intensive claim preparation with a slow reimbursement process,and a lack of quality assurance mechanisms.Conclusion: This study provides a nuanced understanding of the SCP implementation, highlighting the need for revisitingreimbursement rates, addressing documentation challenges, ensuring service availability, streamlining reimbursementprocesses, and strengthening quality assurance mechanisms. The SCP's comprehensive benefits package, effectivepurchaser-provider relationships, and digitization efforts emerge as key strengths that contribute to its success.Keywords: Sehat Card Program (SCP), Social Health Insurance, Implementation Challenges. Stakeholder Perspectives,Reimbursement Processes, Khyber Pakhtunkhwa

SMS Medicine: Revolutionizing Healthcare Delivery through Mobile Technology

In the contemporary era, digital technologies have significantly transformed the medical sector, enhancing accessibility and efficiency through advanced connectivity. Telemedicine, a pivotal advancement in medical science, facilitates remote diagnosis and treatment via telephones and other digital devices. Among various telemedicine modalities, Short Message Service (SMS) medicine emerges as a particularly effective method for delivering healthcare services over long distances using mobile technologies. This paper explores the transformative potential of SMS medicine in healthcare delivery, particularly in low-resource settings and during emergencies such as the COVID-19 pandemic, where it served as a critical tool in maintaining patient care while minimizing physical contact. Through a comprehensive literature review and analysis of case studies, the study highlights the benefits of SMS medicine, including cost-effectiveness, scalability, improved medication adherence, chronic disease management, and enhanced patient-provider communication. Additionally, the integration of SMS with web and mobile applications, such as MDLIVE and Updox, demonstrates increased flexibility and accessibility for patients, especially in rural areas. However, the implementation of SMS medicine is not without challenges, including data privacy concerns, technological limitations, disparities in mobile phone access, and the need for effective integration with existing healthcare systems. The paper also discusses future directions, emphasizing the potential integration of artificial intelligence (AI) and the Internet of Things (IoT) to further enhance personalized medicine and real-time health monitoring. By addressing these challenges and leveraging SMS’s strengths, SMS medicine can bridge critical gaps in healthcare access and delivery, offering sustainable and inclusive solutions for diverse populations. This research underscores the significance of SMS medicine in revolutionizing healthcare systems, particularly in resource-limited environments.

Implementation strategies for providing optimised tuberculosis and diabetes integrated care in LMICs (POTENTIAL): protocol for a multiphase sequential and concurrent mixed-methods study

IntroductionAlmost a quarter of patients with tuberculosis (TB) in Pakistan may also have diabetes, with an additional quarter in a pre-diabetic state. Diabetes is a risk factor for TB. When...

Safety of ipsilateral intravenous access in breast cancer surgery patients

A clinical decision report appraising: Naranjo J, Portner ER, Jakub JW, Cheville AL, Nuttall GA. Ipsilateral Intravenous Catheter Placement in Breast Cancer Surgery Patients. Anesth Analg. 2021 Sep 1;133(3):707-712. https://doi.org/10.1213/ANE.0000000000005597 for a post-lumpectomy patient requiring ipsilateral peripherally inserted central catheter.

HealthWise: AI-Powered Medical Chatbot with Llama 2

This research paper presents the development of a medical chatbot utilizing cutting- edge technologies, specifically the Langchain framework and the Llama 2 language model, in conjunction with the PineCone database for efficient data storage and retrieval. The primary objective of this project is to create an intelligent conversational agent capable of providing reliable medical information, assisting users in symptom assessment, and guiding them towards appropriate healthcare resources. The integration of Langchain facilitates seamless interaction between the user interface and the underlying LLM, enabling natural language understanding and generation. Leveraging the advanced capabilities of the Llama 2 model ensures that the chatbot can engage in meaningful dialogues while maintaining context and relevance. Additionally, PineCone serves as a robust solution for managing and indexing medical data, enhancing the chatbot's ability to deliver accurate and context-aware responses. This study explores the design, implementation, and potential implications of our medical chatbot, contributing to the emerging field of AI-driven healthcare solutions and underscoring its role in improving patient engagement and access to medical advice. Keywords – Medical Chatbot, Langchain, Llama-2, Pine Cone, Natural Language Processing, Artificial Intelligence, Machine Learning, Generative Artificial Intelligence.

The Impact of Surgical Timing on Outcomes in Patients Undergoing Arteriovenous FistulaCreation for Hemodialysis. A Meta-analysis

Background: Arteriovenous fistula (AVF) creation is the preferred method for vascular access in hemodialysis patients, offering better long-term patency, fewer complications, and lower infection rates compared to other options. However, the timing of AVF creation plays a critical role in determining outcomes. This meta-analysis examines the impact of surgical timing on AVF success, focusing on primary failure rates, maturation time, patency, and postoperative complications. Objectives: We sought to evaluate whether the timing of AVF creation impacts outcomes in hemodialysis patients and to provide evidence-based recommendations for clinical practice. Methods: Following PRISMA guidelines, a comprehensive meta-analysis was conducted from major databases (PubMed, Embase, Cochrane) until 2024 that included data. We selected studies based on defined criteria, and assessed for quality using the Newcastle-Ottawa Scale and Cochrane Risk of Bias tool. Primary failure rates, maturation time, primary and secondary patency and postoperative complications were analyzed as outcomes. Heterogeneity was addressed with random effects models, and subgroup analyses were carried out on the basis of patient characteristics. Results: Early AVF creation (creating an AVF ≥ 6 months prior to dialysis initiation) was shown to reduced primary failure rate by 50%, shortened maturation time by 2 to 3 weeks and increase primary patency rates by up to 35%. Early AVF creation, on the other hand, was also accompanied by fewer postoperative complications, including infection and thrombosis, versus delayed AVF creation. Consistent benefits were seen for different patient profiles in subgroup analyses. Conclusion: Early AVF creation is associated with superior outcomes, including lower failure rates, quicker maturation, and fewer complications. These findings support early AVF as the preferred practice in hemodialysis care, enhancing long-term patient outcomes and quality of care.