Therapeutics case studies

Some therapeutic areas, especially rare pediatric diseases, lack clear development guidelines. ICON’s Regulatory Consulting Solutions helped a pharma client gain global regulatory and payer alignment on novel endpoints for a rare neurodevelopmental disorder, boosting confidence ahead of pivotal trials.

A large pharma company submitted a New Drug Application for a targeted therapy treating a rare tumour subtype. To support safe use, a companion diagnostic PMA was filed in parallel with the FDA’s CDRH.

A leading multinational pharmaceutical company partnered with ICON to conduct a phase 1 obesity study assessing the safety and tolerability of a new investigational oral amycretin therapy.

ICON was once again selected as a partner to conduct a phase 1 study in individuals who are overweight or have obesity, evaluating the safety and tolerability of a new subcutaneous GLP-1 and amylin peptide formulation.

A large pharmaceutical company was conducting a Phase 2 study for adults with a BMI of 18-32. They contracted 60 US sites, including 9 Accellacare sites to recruit for two cohorts and conduct the study.

A pharmaceutical sponsor launched a global trial for elderly UTI patients, opening 125 sites across 15 countries. Enrolment lagged at 1 patient per site per month, with 70% non-enrolling. ICON was brought in to rescue the study.

A virtual biotech start-up acquired an immunomodulatory mechanism targeting inflammatory pathways. It had undergone phase 1 and small phase 2a studies. With urgency to advance development and limited resources, the client proposed two separate studies, posing challenges in complexity, timeline, and budget. The client turned to ICON to streamline development.

Over 19 days, ICON conducted a focused site engagement programme to increase patient screening and accelerate enrolment of a late phase multinational, multicentre study to evaluate a treatment for pulmonary disease.

The FDA advised the trial to re-adjudicate so that the study accounted for the effects of COVID-19 on the trial outcomes. To ensure that the trial data would be submitted to the FDA as scheduled, ICON needed to coordinate re-adjudication of 700 endpoints within a compressed three-month timeline.

ICON had he objective to develop a smartwatch mobile app combination that can be used to alert patients, and physicians and drug development teams to physical behaviours that may exacerbate patients’ pain.

A Biotech client contracted ICON to conduct a Phase III study that was a double-blinded, randomised, multinational, multicentre, placebo-controlled trial.

A large, multinational pharmaceutical company needed to better understand patient perspectives to optimize protocol and study design and operationalise a study for TNBC.