Recent Advances in Systems and Network Medicine: Meeting Report from the First International Conference in Systems and Network Medicine.

The gathered together 200 global thought leaders, scientists, clinicians, academicians, industry and government experts, medical and graduate students, postdoctoral scholars and policymakers. Held at Georgetown University Conference Center in Washington D.C.

The Case for the Use of Patient and Caregiver Perception of Change Assessments in Rare Disease Clinical Trials: A Methodologic Overview.

Introduction: The traditional model of evaluating treatments based primarily on primary outcome measures has stumbled in its application to rare disease. Rare disease clinical trials face the methodological challenges of small, heterogeneous patient populations and relatively few validated, disease-specific outcome measures. Incorporating qualitative research into rare disease clinical trials may help sponsors, regulators, payers, and prescribers to better understand the real-world and patient-specific impact of a potential therapy.

The Rules of Engagement: CTTI Recommendations for Successful Collaborations Between Sponsors and Patient Groups Around Clinical Trials.

Objective: To identify the elements necessary for successful collaboration between patient groups and academic and industry sponsors of clinical trials, in order to develop recommendations for best practices for effective patient group engagement.

Methods: In-depth interviews, informed by a previously reported survey, were conducted to identify the fundamentals of successful patient group engagement. Thirty-two respondents from 3 sectors participated: patient groups, academic researchers, and industry.

Quantum of Effectiveness Evidence in FDA's Approval of Orphan Drugs: Update, July 2010 to June 2014.

Background: This study serves as a follow-up to a March 2012 analysis conducted by Frank Sasinowski that reviewed the quantum of effectiveness evidence that is required to secure FDA approval of therapies for rare diseases, or orphan drugs, from the 1983 enactment of the Orphan Drug Act through June 30, 2010. The current study was designed to determine, over the 4 years since the original study, how frequently FDA has required marketing applications of drugs for rare diseases to provide the conventional level of proof of effectiveness that is ordinarily expected for most drugs for prevalent diseases.

Methods: This study employed methods similar to the original analysis, identifying the noncancer orphan drugs approved as new chemical entities by relying on FDA's publicly available documents for drugs approved by FDA from July 1, 2010, to June 30, 2014.