scholarly journals Challenges and approaches to implementing master/basket trials in oncology

2019 ◽  
Vol 3 (14) ◽  
pp. 2237-2243 ◽  
Author(s):  
Amy Burd ◽  
Richard L. Schilsky ◽  
John C. Byrd ◽  
Ross L. Levine ◽  
Vassiliki A. Papadimitrakopoulou ◽  
...  

Abstract The appetite for cutting-edge cancer research, across medical institutions, scientific researchers, and health care providers, is increasing based on the promise of true breakthroughs and cures with new therapeutics available for investigation. At the same time, the barriers for advancing clinical research are impacting how quickly drug development efforts are conducted. For example, we know now that under a microscope, patients with the same type of cancer and histology might look the same; however, the reality is that most cancers are driven by genomic, transcriptional, and epigenetic changes that make each patient unique. Additionally, the immunologic reaction to different tumor types is distinct among patients. The challenge for researchers developing new therapies today is vastly different than it was in the era of cytotoxics. Today, we must identify a sufficient number of patients harboring a rare mutation or other characteristic and match this to the right therapeutic option. This summary provides a guide to help inform the scientific cancer community about the benefits and challenges of conducting umbrella or basket trials (master trials), and to create a roadmap to help make this new and evolving form of clinical trial design as effective as possible.

SLEEP ◽  
2020 ◽  
Vol 43 (Supplement_1) ◽  
pp. A345-A345
Author(s):  
S Gehring ◽  
L Auricchio ◽  
S Kidwell ◽  
K Oppy ◽  
S Smallwood ◽  
...  

Abstract Introduction Obstructive Sleep Apnea (OSA) is associated with neuro-cognitive, cardiovascular and metabolic morbidity in children. Adeno-tonsillectomy is the first line of treatment for OSA with PAP therapy and Oxygen supplementation being alternative therapeutic options in select cases. Severe Obstructive Sleep Apnea is a known risk factor for postoperative respiratory complications after adenotonsillectomy. Therefore, inpatient adenotonsillectomy with close monitoring is recommended for this group of children. Challenges to safe and timely care for this high risk group of children can be overcome with effective coordination of care between different locations and health care providers. Methods All children seeking treatment at Dayton Children’s Division of Sleep Medicine were managed through a pathway developed by a multi-disciplinary team involving sleep medicine, otolaryngology and clinical logistics. Severe OSA was defined as AHI ≥15 events/hr (children <2 year old), AHI ≥15 events/hr with three or more Oxygen desaturations <80% (children ≥2 to <6 years old), AHI ≥ 30 events/hr with three or more Oxygen desaturations <80% (Children ≥6 to 18 years old). Results A total of 78 children were diagnosed with severe OSA in 2019. All children were successfully triaged to appropriate therapeutic option (Adenonotonsillectomy, PAP, O2) within 24 hours of diagnosis. Urgent adenotonsillectomy was performed on the same day in 4 children and within 2 weeks on 12 children. There was no postoperative respiratory complication after urgent adenotonsillectomy. Thirteen children had adenotonsillectomy after 2 weeks. PAP therapy was started in 28 children (34%). Therapy was initiated on the same day in 10 children and the next day on one child. Oxygen supplementation was started in 21 children (27%). Conclusion A multidisciplinary collaborative approach can result in delivery of timely and safe care for severe OSA in children. Support NA


2021 ◽  
Vol 162 (44) ◽  
pp. 1769-1775
Author(s):  
Orsolya Horváth ◽  
Enikő Földesi ◽  
Katalin Hegedűs

Összefoglaló. A palliatív ellátások korai bevonása az onkológiai betegek ellátásába előnnyel jár mind a beteg életminősége, mind a kezelés színvonala, mind a költséghatékonyság szempontjából. Szükség van egy olyan modellre, mely alapján a megfelelő időben, a megfelelő beteg a megfelelő minőségű palliatív ellátásban részesül. Ebben a közleményben a palliatív ellátás korai integrációjának előnyei, szintjei és a speciális palliatív ellátás fogalmának ismertetése után a leginkább elterjedt beutalási modellek előnyeit és hátrányait mutatjuk be a nemzetközi szakirodalom alapján. A speciális palliatív ellátást igénylő betegek kiszűrésére szolgáló, prognózisalapú modellek hátránya, hogy nincs elég kapacitás az ilyen módon beutalt nagyszámú beteg ellátására, ezért széles körben nem terjedtek el. A tüneteken alapuló modellek sokszor bonyolultak és a mindennapi klinikai gyakorlatban nem használatosak. Az új kutatások alapján egyszerű, gyakorlatias kérdéssorokat alkalmaznak, melyekkel könnyen kiemelhetők, akik profitálnak a speciális palliatív intervencióból. Ezek közül a Yale egyetemi és a PALLIA -10 modellt ismertetjük részletesen. Amennyiben az aktív onkológiai ellátást végzők a megfelelő palliatív beutalási kritériumokat ismerik és alkalmazzák, a betegek időben jutnak a megfelelő komplex kezeléshez anélkül, hogy a palliatív ellátórendszer túlterhelődne. Orv Hetil. 2021; 162(44): 1769–1775. Summary. Early integration of palliative care into the trajectory of cancer care brings advantages into the patients’ quality of life, the level of care and cost-efficiency, too. On the basis of a predefined model, the right patient may receive the right level of palliative care at the right time. Having defined the advantages, the levels of early integration of palliative care and the concept of special palliative care, we also aim to describe the advantages and disadvantages of the most common referral models on the basis of international literature in this article. The drawback of prognosis-based models to identify patients needing special palliative care is the lack of capacity to provide care for the large number of patients so recognised; therefore they have not become widespread. Needs-based models tend to be complicated and thus rarely applied in everyday clinical practice. On the basis of new researches, simple, pragmatic questionnaires are utilised through which the patients who could benefit from special palliative care interventions are easy to identify. Here we give a detailed report of the Yale University and PALLIA-10 models. On condition that appropriate palliative referral criteria are known and applied by active oncology care providers, patients may receive adequate complex care without the palliative care system being overloaded. Orv Hetil. 2021; 162(44): 1769–1775.


2007 ◽  
Vol 20 (3) ◽  
pp. 345-350 ◽  
Author(s):  
Daniele Alcalá Pompeo ◽  
Maria Helena Pinto ◽  
Claudia Bernardi Cesarino ◽  
Renilda Rosa Dias Ferreira de Araújo ◽  
Nadia Antonia Aparecida Poletti

OBJECTIVE: To know the hospital discharge process in place and the nurses' performance in preparing patients for discharge. METHODS: A descriptive study using semi-structured interviews was used to collect data from 43 patients of medical-surgical units of a major teaching hospital in the state of São Paulo, Brazil. RESULTS: The majority of patients (83.72%) received tailored discharge instructions. However, a great number of patients (72.08%) reported discharge instructions were not given by nurses. Almost a half of patients (48.84%) reported that discharge instructions were given by their physicians. CONCLUSION: The findings of this study provide insights to improve the educational process of new nurses and their preparation to provide effective discharge instructions. There is also a need to design and implement a hospital discharge process that promotes the participation of interdisciplinary health care providers who are involved in patient clinical care. This discharge process might be an effective way to change health care providers' attitude toward discharge instructions.


1996 ◽  
Vol 1 (1) ◽  
pp. 28-34 ◽  
Author(s):  
Peter Littlejohns ◽  
Carol Dumelow ◽  
Sian Griffiths

Objectives: To help develop a means, based on the views of purchasers and providers of health care, of incorporating national research on clinical effectiveness into local professional advisory mechanisms in order to inform health care purchasing and contracting. Methods: Three geographically based multidisciplinary workshops attended by National Health Service (NHS) staff drawn from the principal purchaser and provider groups in one English region were organized around the discussion of three health care purchasing case studies: Coronary artery disease, diabetes and management of clinical depression in general practice. The proceedings were transcribed and analyzed using content analysis methods. Results: 95 people took part. There were major differences between the purchasers' and health care providers' views on the right balance between local and national information and advisory sources for purchasing. In general, providers wanted the provision of advice to purchasers to be local, in which their opinion was sought, either individually or collectively, acted on and the results fed back to them. In contrast, health authority purchasers considered that local professionals were only one source of professional advice, albeit an important one, to be utilized in coming to decisions. General practitioner fundholders as purchasers, however, preferred to rely on their own experiences and contacts with local providers in making purchasing decisions. Conclusions: Professional specialist advisory groups are necessary to inform the purchasing of health care, but should extend beyond advising on the placement of individual contracts. Involving health care providers in all short-term contracting is unlikely to be cost-effective given the time commitment required. The emphasis at purchaser/provider meetings should be on education: Providing an opportunity for purchasers and providers to develop closer relationships to discuss political imperatives and financial constraints; increasing communication and understanding of providers' and purchasers' roles; and providing an environment for professionals and purchasers to share their views on purchasing. As currently presented, elements of the national policies in the NHS advocating the use of both national evidence on clinical effectiveness and local professional advice are contradictory and should be clarified.


2020 ◽  
Vol 35 (6) ◽  
pp. 669-675
Author(s):  
Mehmet Ali Ceyhan ◽  
Gültekin Günhan Demir

AbstractBackground:Shopping centers (SCs) are social areas with a group of commercial establishments which attract customers of numerous people every day. However, analysis of urgent health conditions and provided health care in SCs has not been performed so far.Objective:The aim of the study was to perform a comparative analysis of clients visiting SCs and demographics, complaints, and health care of patients admitted to Emergency Medical Intervention Units (EMIU) located in grand SCs in Ankara, Turkey.Methods:Customer and health care records of nine grand SCs in Ankara from January 1, 2018 through December 31, 2018 were evaluated retrospectively. Health care services in EMIUs of SCs were provided by employed medical staff. Data including demographic characteristics, complaints, treatment protocols, discharge, and referral to hospital of the patients were retrospectively analyzed from medical registration forms.Results:Medical records of nine grand SCs were analyzed. Number of customers could not be obtained in three SCs due to privacy issues and were not included in patient presentation rate (PPR) and transport-to-hospital rate (TTHR) calculation. Total number of customers in the remaining six SCs were 53,277,239. The total number of patients seeking medical care was 6,749. The number of patients seeking health care in six SCs with known number of customers was 4,498 and PPR ranged from 0.018 to 0.381 patients per 1,000 attendants. The median age of the recorded 4,065 patients (60.2%) was 28 (interquartile range [IQR]: 38-21), and 3,611 (53.5%) of the patients admitted to EMIUs were female. The number of patients treated in the SC was 4,634 (68.6%) and 189 patients (2.8%) were transferred-to-hospital by ambulance for further evaluation and treatment. Transportation to hospital was required in 125 patients who sought medical care in six SCs which provided total number of customers, and TTHR ranged from 0.000 to 0.005 patients per 1,000 attendants. No sudden cardiac death was seen. Medical conditions were the primary reasons for seeking health care. The most frequent causes of presentation were laceration and abrasions (639 patients, 9.4%).Conclusion:The PPR and TTHR in SCs are low. The most common causes of presentation are minor conditions and injuries. Majority of urgent medical conditions in SCs can be managed by health care providers in EMIUs.


2006 ◽  
Vol 24 (21) ◽  
pp. 3490-3496 ◽  
Author(s):  
Robin Matsuyama ◽  
Sashidhar Reddy ◽  
Thomas J. Smith

Purpose The number of patients receiving chemotherapy near the end of life is increasing, as are concerns about goals of treatment, toxicity, and costs. We sought to determine the available sources of knowledge, the choices, and concerns of actual patients, and how patients balanced competing issues. Methods We used a literature search from 1980 to present. Results Available patient sources provide little information about prognosis, choices, alternatives, consequences, or how to choose. Many patients would choose chemotherapy for a small benefit in health outcomes, and for a smaller benefit than perceived by their health care providers for their own treatment. Adverse effects are less a concern for patients than for their well health care providers. There are no decision aids to assist patients with metastatic disease in making their choices, such as there are for adjuvant breast therapy. Conclusion The perspective of the patient is different from that of a well person. Patients are willing to undergo treatments that have small benefits with major toxicity. Receiving realistic information about the different options of care and the likelihood of successful treatment or adverse effects is difficult. These factors may explain some of the increased use of chemotherapy near the end of life. Decision aids and honest, unbiased sources to inform patients of their prognosis, choices, consequences, typical outcomes, and ways to make decisions are needed. More prospective information about how patients make their choices, and what they would consider a good choice, would assist informed decision making.


Author(s):  
Abhinav Gorea

Different situations arise while treating the patients when there are ethical dilemmas to give one or other type of treatment or not to do anything. Sometimes doctors and nurses consider that what is good for the patient must be done because the patient does not understand the situation and consequences. This may lead to complete cure and patient usually goes back to home happily but sometimes a complication may occur and the patient may sue the health care providers. When such situations are analyzed then principles of ethics and law are considered to see if any of these have been violated or not to reach the conclusion. In this study principles of law and ethics of treatment have been discussed to reach the right conclusion; which will be helpful in situations where there are ethical dilemmas during the treatment.


2019 ◽  
Vol 25 (6) ◽  
pp. 613-618 ◽  
Author(s):  
Juan Carlos Martinez-Gutierrez ◽  
Thabele Leslie-Mazwi ◽  
Ronil V Chandra ◽  
Kevin L Ong ◽  
Raul G Nogueira ◽  
...  

Background The number needed to treat is a commonly used statistical term in modern neurointerventional practice. It represents the number of patients that need to be treated for one patient to benefit from an intervention. Given its growing popularity in reflecting study results, understanding the basics behind this statistic is of practical value to the neurointerventionalist. Methods Here, we review the basic theory and calculation of the number needed to treat, its application to stroke interventions, and its limitations. In addition, we demonstrate several simple methods of calculating the number needed to treat utilizing recent thrombectomy trial results. By presenting the number needed to treat as a universal metric, we provide a comprehensive comparative of the number needed to treat for key stroke therapies, including mechanical thrombectomy, tissue plasminogen activator, carotid endarterectomy, and prevention with antiplatelet and statin drugs. Conclusions In comparison with available stroke therapies, mechanical thrombectomy stands out as the most effective acute intervention in patients with emergent large-vessel occlusions. Understanding how the number needed to treat is derived and its implications helps provide perspective to clinical trial data, identify health-care resource priorities, and improve communication with patients, health-care providers, and additional key stakeholders.


2014 ◽  
Author(s):  
◽  
Mirna Becevic

Affordable Care Act (ACA) has allowed more patients that did not previously have health care insurance to have coverage and access to care. This increase in the number of patients seeking medical care will only add additional stress to the existing disproportion of supply and demand for health care providers. In addition, rising health care costs have major effect on how, where, and even if consumers will get needed care. This study examined three different telehealth platforms in three different medical specialties in order to evaluate the perception that they would be appropriate vehicles for increasing access to care. I also wanted to find out what the users' perceptions of these technologies are, as that can be a driving factor in adoption of new technologies. The first study examined the usability and acceptance of new mobile application in teledermatology clinic. The second study focused on usability and acceptance of ICU Robots in a medical ICU. Finally, the third study evaluated if children and youth currently using telepsychiatry as a care delivery method would have other in-person options if telehealth was not available. The results of these three studies point at the complexity and richness of telehealth. The adoption and acceptance of mHealth was very fast and streamlined. In the same fashion, children might not have other appropriate options for care if telepsychiatry was not available in rural Missouri. Interestingly, though, the provider acceptance of ICU Robots was slow, with some provider disengagements observed. This research contributes to the field of health informatics and medical informatics by evaluating adoption and usability of technologies from the provider perspective, vs. the more traditional approach of examining patient satisfaction, or even provider satisfaction without fully understanding the implications of attitudes on the adoption itself. This study has focused purposefully on different groups of providers using different types of telehealth technologies so we could try to see the bigger picture of how telehealth actually contributes to the health care organizational structure.


10.2196/15688 ◽  
2020 ◽  
Vol 3 (1) ◽  
pp. e15688
Author(s):  
Centaine L Snoswell ◽  
John B North ◽  
Liam J Caffery

Background Telehealth is a disruptive modality that challenges the traditional model of having a clinician or patient physically present for an appointment. The benefit is that it offers the opportunity to redesign the way services are offered. For instance, a virtual health practitioner can provide videoconference consultations while being located anywhere in the world that has internet. A virtual health practitioner also obviates the issues of attracting a specialist medical workforce to rural areas, and allows the rural health service to control the specialist services that they offer. Objective The aim of this research was to evaluate the economic effects of 3 different models of care on rural and metropolitan hospital sites. The models of care examined were patient travel, telehealth using videoconferencing, and employment of a virtual health practitioner by a rural site. Methods Using retrospective activity data for 3 years, a return on investment (ROI) analysis was undertaken from the perspective of a rural site and metropolitan partner site using a telehealth orthopedic fracture clinic as an example. Further analysis was conducted to calculate the number of patients that would be required to attend the clinic in each model of care for the sites to break even. Results The only service model that resulted in a positive ROI for the rural site over the 3-year period was the virtual health practitioner model. The breakeven analysis demonstrated that the rural site required the lowest number of patients to recoup costs in the virtual health practitioner model of care. The rural site was unable to recoup its costs within the travel model due to the lack of opportunity for reimbursement for services and the requirement to cover the cost of travel for patients. Conclusions Our model demonstrated that rural health care providers can increase their ROI by employing a virtual health practitioner.


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