Meeting report: the ALPHA project: a stakeholder meeting on lupus clinical trial outcome measures and the patient perspective.

Drug development in lupus has improved over the past 10 years but still lags behind that of other rheumatic disease areas. Assessment of prospective lupus therapies in clinical trials has proved challenging for reasons that are multifactorial including the heterogeneity of the disease, study design limitations and a lack of validated biomarkers which greatly impacts regulatory decision-making. Moreover, most composite outcome measures currently used in trials do not include patient-reported outcomes.

The ALPHA Project: Establishing consensus and prioritisation of global community recommendations to address major challenges in lupus diagnosis, care, treatment and research.

The Addressing Lupus Pillars for Health Advancement (ALPHA) Project is a global consensus effort to identify, prioritise and address top barriers in lupus impacting diagnosis, care, treatment and research. To conduct this process, the ALPHA Project convened a multistakeholder Global Advisory Committee (GAC) of lupus experts and collected input from global audiences, including patients. In phase I, the ALPHA Project used expert interviews and a global survey of lupus experts to identify and categorise barriers into three overarching pillars: drug development, clinical care and access to care.

Unique Burdens of Pediatric Clinical Trials in Duchenne Muscular Dystrophy, April 20-21, 2017, Bethesda, Maryland, USA.

Recent increases in the number and breadth of clinical trials for patients with Duchenne muscular dystrophy (DMD) have engendered hope for a better future. Despite the overall enthusiasm by the DMD community for these trials, however, the burdens and pressures that they place on children with Duchenne muscular dystrophy and their families have become painfully apparent. In order to identify, and mediate, these challenges, Parent Project Muscular Dystrophy (PPMD) sponsored a meeting to examine some of these issues more closely in Bethesda, Maryland, on April 20-21, 2017.

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.

Among the challenges confronting patients with rare diseases is a dearth of treatment options. The development of safe and effective new therapies is hampered by challenges associated with conducting clinical trials in small populations. In this article, we describe how the Duchenne muscular dystrophy community-led by Parent Project Muscular Dystrophy-created a proposed draft guidance document for industry for submission to the U.

The practicing clinician and regulatory safety concerns.

Pharmaceutical agents are prescribed to produce a therapeutic effect, but safety concerns require constant attention to the benefit:risk relationship inherent in their use and the needs of the individual patient. Such calculations involve assumptions about the likely tolerability of harm, in that greater safety risks may be acceptable for use of a lifesaving drug, compared with those acceptable for an agent providing only improved "quality of life." Making such assumptions is an activity integral to the bedside clinician's role, is done during many (perhaps most) patient encounters, and is often undertaken with inadequate information.