Therapeutics case studies

A small Asia-based biotech engaged ICON to manage its EU orphan drug designation submission for a treatment targeting Dercum’s disease. The designation was key to supporting a planned Phase 3 study and expanding into the EU, where the company had no existing presence.

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.

ICON was contracted to conduct an early-phase clinical trial in Huntington’s disease across 12 countries and 53 sites.

ICON provided full-service support to a large biotech company in two global Phase 3 trials for a gene therapy product treating spinal muscular atrophy (SMA), a rare but serious condition causing muscle wasting and weakness.

Clinical trial participation exposes rare disease participants to significant pressure financially, logistically and psychologically, and these incremental pressures can negatively impact a patient’s ability or willingness to enrol or remain in a clinical trial.

ICON developed the first PKU-specific Quality of Life questionnaire available and validated in four age groups and seven languages.

A pharmaceutical client needed to better understand patient perspectives, concerns and awareness of that could impact the development of a vaccine for Human Cytomegalovirus (HCMV).

The client was conducting a a pharmaco-epidemiologic study to characterise the impacts and burden of Achondroplasia across the lifespan.

A mid-sized biopharmaceutical sponsor was conducting a phase 3 study on CDKL5 deficiency disorder, a rare
genetic seizure disorder in children.

A sponsor needed to recruit nearly 500 patients for a pivotal study in an ultra rare disease. The study's demanding protocol posed significant difficulties for patients. ICON was able to revise the protocol, reducing patient burden, and implemented a global recruitment strategy and additional support that allowed recruitment to be completed ahead of schedule, saving the sponsor an estimated $700,000.

A biotech company sought ICON's help to analyse and report on a limited data set to demonstrate cardiovascular safety in a rare disease study.

A large pharma company was attempting to recruit 50 patients, including 15 treatment-naive patients, in a global study for a rare pulmonary disorder.