Comparative Assessment of Non-trial Access to Investigational Medical Products in the U.S. and Japan.

The U.S. and Japan have preapproval access systems for patients who have exhausted approved treatment options.

Patient advocacy organizations' information for patients on pre-approval access to investigational treatments.

Objective: To evaluate the availability of information regarding patient access to investigational treatments through clinical trials and non-trial pre-approval access pathways from a sample of patient advocacy organization (PAO) websites in the United States.

Results: We systematically analyzed the content of 118 randomly selected PAO websites to assess whether they contained information on clinical trials and non-trial pathways-e.g.

Single-Patient Expanded Access Requests: IRB Professionals' Experiences and Perspectives.

Background: U.S. physicians may treat a patient with an investigational drug outside of a clinical trial by using the expanded access (EA) pathway or the recently created federal right to try (RTT) pathway.

Ethical Issues in Gender-Affirming Care for Youth.

Transgender and gender-nonconforming (TGNC) youth who suffer from gender dysphoria are at a substantially elevated risk of numerous adverse physical and psychosocial outcomes compared with their cisgender peers. Innovative treatment options used to support and affirm an individual's preferred gender identity can help resolve gender dysphoria and avoid many negative sequelae of nontreatment. Yet, despite advances in these relatively novel treatment options, which appear to be highly effective in addressing gender dysphoria and mitigating associated adverse outcomes, ethical challenges abound in ensuring that young patients receive appropriate, safe, affordable treatment and that access to this treatment is fair and equitable.

Medical Crowdfunding for Scientifically Unsupported or Potentially Dangerous Treatments.

This study identified fundraising campaigns for unsubstantiated treatments, such as homeopathic remedies for cancer and antibiotic therapy for chronic Lyme disease, on GoFundMe and other crowdfunding platforms.

Improving Expanded Access in the United States: The Role of the Institutional Review Board.

Background: The FDA allows patients with a serious or immediately life-threatening illness to use investigational medical products outside of clinical trials through its "expanded access" program. In response to criticism that the process to apply for expanded access is too onerous, numerous changes have been made over the last few years. These have been largely focused on the FDA and the pharmaceutical industry, while institutional review boards (IRBs)-which must approve expanded access protocols, except in emergencies when there is not time to do so-have remained relatively unstudied.

Meaningful Use of Electronic Health Records for Quality Assessment and Review of Clinical Ethics Consultation.

Evolving practice requires peer review of clinical ethics (CE) consultation for quality assessment and improvement. Many institutions have identified the chart note as the basis for this process, but to our knowledge, electronic health record (EHR) systems are not necessarily designed to easily include CE consultation notes. This article provides a framework for the inclusion of CE consultation notes into the formal EHR, describing a developed system in the Epic EHR that allows for the elaborated electronic notation of the CE chart note.

Charlie Gard and the Limits of Parental Authority.

The parents of Charlie Gard, who was born August 4, 2016, with an exceedingly rare and incurable disease called mitochondrial DNA depletion syndrome, fought a prolonged and heated legal battle to allow him access to experimental treatment that they hoped would prolong his life and to prevent his doctors from withdrawing life-sustaining care. Charlie's clinicians at the Great Ormond Street Hospital in London believed that the brain damage Charlie had suffered as a result of frequent epileptic seizures, along with many other severe disabilities, would render any innovative therapy futile, and they disagreed with his parents' wishes to use an experimental therapy. They felt it in Charlie's best interest that he be allowed to die.

Effects of Vacuolar H-ATPase Inhibition on Activation of Cathepsin B and Cathepsin L Secreted from MDA-MB231 Breast Cancer Cells.

Studies indicate secreted cathepsins are involved in metastasis. V-ATPases, which are necessary for activating intracellular cathepsins, also play a role in metastasis and are targeted to the plasma membrane of metastatic breast cancer cells. We are interested in a connection between cell surface V-ATPases, activation of secreted cathepsins and the metastatic phenotype of MDA-MB231 cells.

Multinuclear NMR and MRI Reveal an Early Metabolic Response to mTOR Inhibition in Sarcoma.

Biomarkers predicting rapalog responses in sarcomas where PI3K and mTOR are often hyperactivated could improve the suitable recruitment of responsive patients to clinical trials. PI3K/mTOR pathway activation drives energy production by regulating anaerobic glycolysis in cancer cells, suggesting a route toward a monitoring strategy. In this study, we took a multimodality approach to evaluate the phenotypic effects and metabolic changes that occur with inhibition of the PI3K/mTOR pathway.