A bill passed by the Senate would sharply curtail the FDA’s oversight of access to investigational drugs. Although its impact would probably be limited, the motivation behind it threatens to weaken ...the FDA’s ability to pursue its public health mission.
IMPORTANCE: Patient advocacy organizations (PAOs) are influential health care stakeholders that provide direct counseling and education for patients, engage in policy advocacy, and shape research ...agendas. Many PAOs report having financial relationships with for-profit industry, yet little is known about the nature of these relationships. OBJECTIVE: To describe the nature of industry funding and partnerships between PAOs and for-profit companies in the United States. DESIGN, SETTING, AND PARTICIPANTS: A survey was conducted from September 1, 2013, to June 30, 2014, of a nationally representative random sample of 439 PAO leaders, representing 5.6% of 7865 PAOs identified in the United States. Survey questions addressed the nature of their activities, their financial relationships with industry, and the perceived effectiveness of their conflict of interest policies. MAIN OUTCOMES AND MEASURES: Amount and sources of revenue as well as organizational experiences with and policies regarding financial conflict of interest. RESULTS: Of the 439 surveys mailed to PAO leaders, 289 (65.8%) were returned with at least 80% of the questions answered. The PAOs varied widely in terms of size, funding, activities, and disease focus. The median total revenue among responding organizations was $299 140 (interquartile range, $70 000-$1 200 000). A total of 165 of 245 PAOs (67.3%) reported receiving industry funding, with 19 of 160 PAOs (11.9%) receiving more than half of their funding from industry. Among the subset of PAOs that received industry funding, the median amount was $50 000 (interquartile range, $15 000-$200 000); the median proportion of industry support derived from the pharmaceutical, device, and/or biotechnology sectors was 45% (interquartile range, 0%-100%). A total of 220 of 269 respondents (81.8%) indicated that conflicts of interest are very or moderately relevant to PAOs, and 94 of 171 (55.0%) believed that their organizations’ conflict of interest policies were very good. A total of 22 of 285 PAO leaders (7.7%) perceived pressure to conform their positions to the interests of corporate donors. CONCLUSIONS AND RELEVANCE: Patient advocacy organizations engage in wide-ranging health activities. Although most PAOs receive modest funding from industry, a minority receive substantial industry support, raising added concerns about independence. Many respondents report a need to improve their conflict of interest policies to help maintain public trust.
In two phase 3 placebo-controlled, randomized trials in 1012 and 1040 patients with mild-to-moderate Alzheimer's disease, solanezumab, a humanized monoclonal antibody that preferentially binds ...soluble forms of amyloid, did not improve cognition or functional status.
Alzheimer's disease is associated with the accumulation of aggregated amyloid-beta (Aβ) peptide in the cerebral cortex and hippocampus. One approach to reducing brain amyloid involves increasing the clearance of Aβ by means of prolonged treatment with monoclonal antibodies directed against this peptide. In preclinical studies, a murine antibody that targeted the central domain of Aβ and was selective for soluble forms slowed Aβ deposition in a transgenic mouse model
1
; in another transgenic murine model, Aβ–antibody complexes were present in the cerebrospinal fluid (CSF) and plasma, and behavioral deficits were reversed without a decrease in amyloid plaques, as assessed by . . .
In this placebo-controlled trial, the γ-secretase inhibitor semagacestat did not improve cognitive status in patients with Alzheimer's disease and was associated with more adverse events than ...placebo, including skin cancers and infections.
Alzheimer's disease begins decades before the appearance of clinical symptoms, with the deposition of aggregated amyloid-beta (Aβ) peptide plaques in the cortex and hippocampus. This protein is cleaved from the amyloid precursor protein (APP) by the sequential action of β- and γ-secretases, producing fragments that include Aβ1-40 and Aβ1-42. Since the accumulation of aggregated Aβ is associated with disease progression, both β-secretase and γ-secretase represent potential therapeutic targets. Multiple small molecules can inhibit γ-secretase in vitro,
1
–
4
but Notch and other transmembrane proteins are also substrates for γ-secretase,
1
–
4
and studies have raised concern that the inhibition of γ-secretase could . . .
Objective To explore how characteristics of medical decisions influence parents' preferences for control over decisions for their seriously ill infants. Study design In qualitative interviews, ...parents of infants in the neonatal intensive care unit (NICU) were asked to consider all medical decisions they could recall, and were prompted with decisions commonly encountered in the NICU. For each decision, parents were asked detailed questions about who made each decision, whom they would have preferred to make the decision, and why. Using standard qualitative methods, responses were coded and organized such that decision-level characteristics could be analyzed according to preferred decision-making role. Results Parents identified 2 factors that were associated with a preference to delegate decisions to the medical team (high degree of urgency, high level of required medical expertise) and 4 factors associated with a preference to retain parental control (high perceived risk, high personal experience with the decision, involvement of foreign bodily fluids, and similarity to decisions that they perceived as part of the normal parental role). Conclusions Characteristics of decisions influence preferences for control over medical decisions among parents of patients in the NICU. These insights may guide improvements in physician-parent communication and consent.
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GEOZS, IJS, IMTLJ, KILJ, KISLJ, NUK, OILJ, PNG, SAZU, SBCE, SBJE, UL, UM, UPCLJ, UPUK, ZRSKP
Translating whole-exome sequencing (WES) for prospective clinical use may have an impact on the care of patients with cancer; however, multiple innovations are necessary for clinical implementation. ...These include rapid and robust WES of DNA derived from formalin-fixed, paraffin-embedded tumor tissue, analytical output similar to data from frozen samples and clinical interpretation of WES data for prospective use. Here, we describe a prospective clinical WES platform for archival formalin-fixed, paraffin-embedded tumor samples. The platform employs computational methods for effective clinical analysis and interpretation of WES data. When applied retrospectively to 511 exomes, the interpretative framework revealed a 'long tail' of somatic alterations in clinically important genes. Prospective application of this approach identified clinically relevant alterations in 15 out of 16 patients. In one patient, previously undetected findings guided clinical trial enrollment, leading to an objective clinical response. Overall, this methodology may inform the widespread implementation of precision cancer medicine.
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DOBA, IJS, IZUM, KILJ, NUK, PILJ, PNG, SAZU, UILJ, UKNU, UL, UM, UPUK
Background Revelations that some members of Congress, including members of key health care committees, hold substantial personal investments in the health care industry have raised concerns about ...lawmakers' financial conflicts of interest (COI) and their potential impact on health care legislation and oversight. Aims 1) To assess historical trends in both the number of legislators holding health care-related assets and the value and composition of those assets. 2) To compare the financial holdings of members of health care-focused committees and subcommittees to those of other members of the House and Senate. Methods We analyzed 11 years of personal financial disclosures by all members of the House and Senate. For each year, we calculated the percentage of members holding a health care-related asset (overall, by party, and by committee); the total value of all assets and health care-related assets held; the mean and median values of assets held per member; and the share of asset values attributable to 9 health asset categories. Findings During the study period, over a third of all members of Congress held health care-related assets. These assets were often substantial, with a median total value per member of over $43,000. Members of health care-focused committees and subcommittees in the House and Senate did not hold health care-related assets at a higher rate than other members of their respective chambers. Conclusions These findings suggest that lawmakers' health care-related COI warrant the same level of attention that has been paid to the COI of other actors in the health care system.
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DOBA, IZUM, KILJ, NUK, PILJ, PNG, SAZU, SIK, UILJ, UKNU, UL, UM, UPUK
Ethics and Genomic Incidental Findings McGuire, Amy L.; Joffe, Steven; Koenig, Barbara A. ...
Science (American Association for the Advancement of Science),
05/2013, Volume:
340, Issue:
6136
Journal Article
Peer reviewed
Open access
The American College of Medical Genetics and Genomics (ACMG) recently issued a statement (1) recommending that all laboratories conducting clinical sequencing seek and report pathogenic and expected ...pathogenic mutations for a short list of carefully chosen genes and conditions. The recommendations establish a baseline for reporting clinically relevant incidental findings and articulate ethical principles relevant to their disclosure. The ACMG acknowledged that the list will evolve over time and is developing a mechanism for community input (2). This paper focuses on the ethical framework for the recommendations, rather than on the choice of which genes to include on the list.
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BFBNIB, NMLJ, NUK, PNG, SAZU, UL, UM, UPUK
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