Case Study

Management Challenge:

• US based globally-reaching medium sized biotech wished to expand Phase III and subsequent post approval studies for a novel drug in ultra-rare paediatric neurodegeneration associated with iron accumulation
• Medical Affairs required consultant to identify location of patients, potential
  study sites and Principal Investigators in UK, France and Germany

Project Setup

• The project was let by consultant with 20 years of industry experience in Medical Affairs and clinical trial set up through all phases and post approval real world studies in the UK and Europe.
• The project team included a medical writer to prepare study reports and manuscripts and consultant to search literature for epidemiologic distribution of patients in the selected countries

Approach & Solution

• Within the selected countries, patients were located through identifying secondary and tertiary treatment centres and building up a knowledge of the patient treatment pathway. A KOL advisory board was also convened. The Phase III trial (with the consultants working with the nominated CRO) was then initiated.
• A troubleshooting and warning system was adopted in those sites where recruitment was poor and strategies developed to enhance this process via referrals from other national centres not involved in the trial.
• In French centres not involved in the Phase III trial an extended access (named patient) programme was implemented.
• After the successful completion of the Phase III trial and post EMEA approval, a number of real-world data gathering studies were designed by the consultant and implemented including Investigator Initiated Studies and country-specific registry programmes to track disease progression and efficacy of new product
• The new product was presented and included in EU and UK therapeutic guidance and the clinical trial data published and presented to NICE in the UK at a Health Technology Assessment
• Full training was given to the ex-UK MSLs team on the studies and on the healthcare systems in each country particularly pertaining to the uptake of new products in Ultra-rare disease.

Benefits & Outcome

• Recruitment targets were met for the Phase III trial in the selected countries with all patients continuing on the drug to date
• A highly successful post-approval data collection ensured regulatory approval and reimbursement in all selected countries.