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The United States (US) Expanded Access Program (EAP) to coronavirus disease 2019 (COVID-19) convalescent plasma was initiated in response to the rapid spread of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the causative agent of COVID-19. While randomized clinical trials were in various stages of development and enrollment, there was an urgent need for widespread access to potential therapeutic agents. The objective of this study is to report on the demographic, geographical, and chronological characteristics of patients in the EAP, and key safety metrics following transfusion of COVID-19 convalescent plasma. Mayo Clinic served as the central institutional review board for all participating facilities, and any US physician could participate as a local physician-principal investigator. Eligible patients were hospitalized, were aged 18 years or older, and had-or were at risk of progression to-severe or life-threatening COVID-19; eligible patients were enrolled through the EAP central website. Blood collection facilities rapidly implemented programs to collect convalescent plasma for hospitalized patients with COVID-19. Demographic and clinical characteristics of all enrolled patients in the EAP were summarized. Temporal patterns in access to COVID-19 convalescent plasma were investigated by comparing daily and weekly changes in EAP enrollment in response to changes in infection rate at the state level. Geographical analyses on access to convalescent plasma included assessing EAP enrollment in all national hospital referral regions, as well as assessing enrollment in metropolitan areas and less populated areas that did not have access to COVID-19 clinical trials. From April 3 to August 23, 2020, 105,717 hospitalized patients with severe or life-threatening COVID-19 were enrolled in the EAP. The majority of patients were 60 years of age or older (57.8%), were male (58.4%), and had overweight or obesity (83.8%). There was substantial inclusion of minorities and underserved populations: 46.4% of patients were of a race other than white, and 37.2% of patients were of Hispanic ethnicity. Chronologically and geographically, increases in the number of both enrollments and transfusions in the EAP closely followed confirmed infections across all 50 states. Nearly all national hospital referral regions enrolled and transfused patients in the EAP, including both in metropolitan and in less populated areas. The incidence of serious adverse events was objectively low (<1%), and the overall crude 30-day mortality rate was 25.2% (95% CI, 25.0% to 25.5%). This registry study was limited by the observational and pragmatic study design that did not include a control or comparator group; thus, the data should not be used to infer definitive treatment effects. These results suggest that the EAP provided widespread access to COVID-19 convalescent plasma in all 50 states, including for underserved racial and ethnic minority populations. The study design of the EAP may serve as a model for future efforts when broad access to a treatment is needed in response to an emerging infectious disease. ClinicalTrials.gov NCT#: NCT04338360.

Social media is fundamentally altering how we access health information and make decisions about medical treatment, including for terminally ill patients. This specifically includes the growing phenomenon of patients who use online petitions and social media campaigns in an attempt to gain access to experimental drugs through expanded access pathways. Importantly, controversy surrounding expanded access and “compassionate use” involves several disparate stakeholders, including patients, manufacturers, policymakers, and regulatory agencies—all with competing interests and priorities, leading to confusion, frustration, and ultimately advocacy. In order to explore this issue in detail, this correspondence article first conducts a literature review to describe how the expanded access policy and regulatory environment in the United States has evolved over time and how it currently impacts access to experimental drugs. We then conducted structured web searches to identify patient use of online petitions and social media campaigns aimed at compelling access to experimental drugs. This was carried out in order to characterize the types of communication strategies utilized, the diseases and drugs subject to expanded access petitions, and the prevalent themes associated with this form of “digital” patient advocacy. We find that patients and their families experience mixed results, but still gravitate towards the use of online campaigns out of desperation, lack of reliable information about treatment access options, and in direct response to limitations of the current fragmented structure of expanded access regulation and policy currently in place. In response, we discuss potential policy reforms to improve expanded access processes, including advocating greater transparency for expanded access programs, exploring use of targeted economic incentives for manufacturers, and developing systems to facilitate patient information about existing treatment options. This includes leveraging recent legislative attention to reform expanded access through the CURE Act Provisions contained in the proposed U.S. 21st Century Cures Act. While expanded access may not be the best option for the majority of individuals, terminally ill patients and their families nevertheless deserve better processes, policies, and availability to potentially life-changing information, before they decide to pursue an online campaign in the desperate hope of gaining access to experimental drugs.

Background Compassionate use programs allow patients with life-threating and serious conditions to access investigational therapies when standard treatments are inadequate. This study aims to analyze the trends and outcomes of the compassionate use of medicinal products in Israel. Methods Data from the Israeli Ministry of Health’s compassionate use database (2020–2024) were anonymized and analyzed. Duplicates were removed, and a pivot table was used to assess factors such as active ingredients, indications, treatment counts, and patient demographics. Results were presented as counts and percentages, with treatments over 0.5% of the total classified as common. Statistical analyses included Student’s t-test and chi-squared test for subgroup differences ( p  < 0.05 significant). Commonly used medicinal products were cross-referenced with the MOH drug database for registration and reimbursement status. Results A total of 3,284 compassionate treatments were administered, employing 596 distinct medicinal products to address 1,361 conditions in 2020–2024. Temporal analysis identified a peak in 2020, which accounted for 24% of total treatments, followed by a decrease thereafter. Patient age stratification indicated that those aged 65 to 80 received the highest treatment proportion (26%), while the 45 to 65 age group accounted for 19%. Treatments were mainly concentrated in large central hospitals (77%) and the central district (49%), with the southern district showing the least usage. The authorization process was primarily for the continuation of study drug in 63% of cases. Additionally, Belantamab mafodotin and Trametinib were the most frequently utilized medicinal products, accounting for 9% and 8.6% of treatments, respectively. Disease category analysis revealed that relapsed refractory multiple myeloma, central nervous system tumors, and inflammatory bowel disease were among the top conditions treated, varying by age group. Notably, 60% of the most common technologies (13 out of 22) were subsequently included into the national health basket, typically following an extended period of compassionate use that exceeded two years. Conclusion The study suggests that Israel’s compassionate use programs have accelerated early access to novel therapies for complex conditions and provided a bridge to the inclusion of novel medicinal products in the national health basket. Nonetheless, the study identifies a concerning downward trend in utilization alongside potential access disparities, thereby underscoring the necessity for further targeted investigations.

A month ago, an investigatory television programme, The Hyena, reported that children with incurable diseases such as spinal muscular atrophy were being denied supposedly important treatment, and Italian show-business personalities joined the call to relax rules on stem-cell treatment. Last May, a delegation including representatives of the Italian Medicines Agency (AIFA) and the ISS, the health ministry's national institute, visited the Brescia lab and reported chaotic conditions: ethics-committee approvals had been based on inadequate information, and there were no detailed protocols or patient follow-up, for example.

The first-line phase 3 study with tremelimumab did not show an improvement in its primary endpoint, overall survival, and the drug was never licensed.2,7 At least a third of patients randomly assigned to the control group who were alive at the end of the study or censored at any point had received ipilimumab at some stage, which might well have contributed to the negative outcome of the study.3 This conclusion was supported by the finding that outcomes for patients in US and non-US sites were different, with treatment with tremelimumab resulting in significantly better survival in patients treated in non-US centres, where the expanded access programme for ipilimumab was less readily available, and no difference in US centres, where ipilimumab was readily available via the expanded access programme. [...]patient information and consent forms should explicitly state that involvement in a clinical trial might limit access to future treatments that are made available as part of an expanded access programme.

Background. Nowadays one of the most critical aspects of innovative cell-based therapies is the unregulated industry, as it is becoming a competitor of the regulated system. Many private clinics, worldwide, advertise and offer cell-based interventions treatments directly to the consumer and this poses a risk to both vulnerable patients and health systems. Several countries have implemented Compassionate Use Programmes (CUP) that provide patients with medicines that have not yet completed the approval pathway, in the event that no reasonable alternative exists. Recently, in the public discourse, compassionate use has been increasingly associated with a patient’s right to try. Thus, the aim of this study was to assess public knowledge of the clinical trials process with specific reference to innovative stem cell treatments, and trust in the institutions responsible for regulatory activities. We also asked people about their “right” to use unregulated therapies. Methods. We developed an ad hoc questionnaire on three main areas of concern and administered it to 300 people in the patient waiting room at an Italian university hospital. Results. Our findings suggest that people have a good knowledge of the clinical trials process and trust in healthcare institutions. Nonetheless, one person in two believes it is a right to use unregulated therapies. Conclusions.We stress the need, in the age of cellular therapies, for a commitment to support vulnerable patients and to strengthen awareness among the public about the substantial boundary that differentiates experimental therapies from unproven therapies. There should not be a “right to try” something that is unsafe but rather approved treatments and in line with good clinical practice. The trend, which emerged on this issue from our study, is quite different, confirming the urgent need to improve health information so that it is as complete as possible.

Arthur Caplan, a medical ethicist at the New York University School of Medicine, discusses how to fairly distribute experimental drugs outside of clinical trials. Last year, the FDA received 1,262 requests from drug companies, on behalf of desperate patients, to use unapproved drugs outside of clinical trial programmes. The number of 'compassionate use' requests has risen by nearly 25% since 2010, with no sign of abating. Yet, while companies carry most of the onus of deciding which cases to support and which to deny, they typically make their decisions on an ad hoc basis. Social media campaigns and personal connections can sway the outcomes. Arthur Caplan, a medical ethicist at New York University School of Medicine, believes that a fairer decision-making process is needed. He told Asher Mullard about a pilot project that he ran with Johnson & Johnson's pharmaceutical company Janssen to equitably and transparently distribute an experimental drug outside of its clinical trial programme.

With drug companies’ policies hard to decipher, frustrated patients often resort to social-media campaigns and other public appeals.

The President of the United States has repeatedly touted hydroxychlororquine as a likely cure for COVID-19 and urged Americans to try it, stating at one of his media briefings, \"What do you have to lose? What do you have to lose? Take it\" (1). A few others around the world have chimed in to promote one drug or another, this drug in combination with others, or their own favorite untested nostrums. This has led to drug hoarding, the inability of patients who actually need and benefit from certain drugs to access them, and serious side effects and even deaths from self-medication. As it was unclear at the time whether any benefit would come from these interventions, a mechanism called monitored emergency use of unregistered and investigational interventions (MEURI) was created as a bridge to clinical trials in order to curtail unconstrained use of medications.