Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials

Patients have received experimental pharmaceuticals outside of clinical trials for decades. There are no industry-wide best practices, and many companies that have granted compassionate use, or ‘preapproval’, access to their investigational products have done so without fanfare and without divulging the process or grounds on which decisions were made. The number of compassionate use requests has increased over time. Driving the demand are new treatments for serious unmet medical needs; patient advocacy groups pressing for access to emerging treatments; internet platforms enabling broad awareness of compelling cases or novel drugs and a lack of trust among some that the pharmaceutical industry and/or the FDA have patients’ best interests in mind. High-profile cases in the media have highlighted the gap between patient expectations for compassionate use and company utilisation of fair processes to adjudicate requests. With many pharmaceutical manufacturers, patient groups, healthcare providers and policy analysts unhappy with the inequities of the status quo, fairer and more ethical management of compassionate use requests was needed. This paper reports on a novel collaboration between a pharmaceutical company and an academic medical ethics department that led to the formation of the Compassionate Use Advisory Committee (CompAC). Comprising medical experts, bioethicists and patient representatives, CompAC established an ethical framework for the allocation of a scarce investigational oncology agent to single patients requesting non-trial access. This is the first account of how the committee was formed and how it built an ethical framework and put it into practice

Fair, just and compassionate: A pilot for making allocation decisions for patients requesting experimental drugs outside of clinical trials

Patients have received experimental pharmaceuticals outside of clinical trials for decades. There are no industry-wide best practices, and many companies that have granted compassionate use, or ‘preapproval’, access to their investigational products have done so without fanfare and without divulging the process or grounds on which decisions were made. The number of compassionate use requests has increased over time. Driving the demand are new treatments for serious unmet medical needs; patient advocacy groups pressing for access to emerging treatments; internet platforms enabling broad awareness of compelling cases or novel drugs and a lack of trust among some that the pharmaceutical industry and/or the FDA have patients’ best interests in mind. High-profile cases in the media have highlighted the gap between patient expectations for compassionate use and company utilisation of fair processes to adjudicate requests. With many pharmaceutical manufacturers, patient groups, healthcare providers and policy analysts unhappy with the inequities of the status quo, fairer and more ethical management of compassionate use requests was needed. This paper reports on a novel collaboration between a pharmaceutical company and an academic medical ethics department that led to the formation of the Compassionate Use Advisory Committee (CompAC). Comprising medical experts, bioethicists and patient representatives, CompAC established an ethical framework for the allocation of a scarce investigational oncology agent to single patients requesting non-trial access. This is the first account of how the committee was formed and how it built an ethical framework and put it into practice

From adaptive licensing to adaptive pathways: Delivering a flexible life-span approach to bring new drugs to patients

The concept of adaptive licensing (AL) has met with considerable interest. Yet some remain skeptical about its feasibility. Others argue that the focus and name of AL should be broadened. Against this background of ongoing debate, we examine the environmental changes that will likely make adaptive pathways the preferred approach in the future. The key drivers include: growing patient demand for timely access to promising therapies, emerging science leading to fragmentation of treatment populations, rising payer influence on product accessibility, and pressure on pharma/investors to ensure sustainability of drug development. We also discuss a number of environmental changes that will enable an adaptive paradigm. A life-span approach to bringing innovation to patients is expected to help address the perceived access vs. evidence trade-off, help de-risk drug development, and lead to better outcomes for patients

Does engaging in a group-based intervention increase parental sense of competence in parents of pre-school children?:A Systematic Review of the Current Literature

As the preschool years are a formative period for long-term physical and mental health, this period is recognised as an important window for early effective intervention. Parenting behaviour is a key factor to target in order to optimise child development. Group-based interventions for parents are considered efficient and cost effective methods of early intervention and have been found to improve child behaviour and adjustment. Self-efficacy is key to behaviour change and as such parental self-efficacy should be a consideration in interventions aimed at influencing parenting behaviour. Therefore, the purpose of this systematic review was to examine the impact of group-based early interventions for parents of preschool children on parental self-efficacy. Nine databases were searched (ASSIA, CINAHL, EMBASE, Maternity and Infant Care, Ovid Medline, PsycINFO, Pubmed, Science Direct and Web of Science). Studies were included if they were a randomised controlled trial of a group-based intervention for parents of preschool children and measured change in parental self-efficacy. Fifteen studies were identified. Although changes in parental self-efficacy following a group-based intervention were noted in the majority of studies reviewed, the methodological quality of the studies included in the review means these findings have to be interpreted with caution; only seven studies were rated to be methodologically adequate. Further research is needed to understand the mechanisms by which these interventions may improve parental self-efficacy. Studies specifically examining the impact of such interventions on paternal self-efficacy are also warranted