MARKET ACCESS AND OUTCOMES MODELLING FOR PHASE II ASSET

On July 16, 2014, In Case Studies

Brief

Myocardial revascularisation is a life-saving intervention for patients suffering myocardial infarction (MI). However, tissue damage associated with revascularisation of the heart muscle represents a significant challenge for clinicians as it can lead to an acute inflammatory response; capillary leaks with major complications including atrial fibrillation; myocardial stunning; and an increased infarct size. A development-stage bio-pharmaceutical company has a novel peptide in development designed to limit the extent of tissue damage. The compound is entering Phase II clinical trials and has the potential to prevent or delay negative cardiac outcomes following revascularisation for a MI event. It therefore represents a potentially significant cost-saving treatment, in addition to providing clinical benefits by reducing mortality and improving patient quality of life. The company would like to assess the cost-effectiveness and budgetary impact of their new treatment.

Challenge

The ultimate objective was to explore the potential value of the asset to attract investment, and structure negotiations with potential partner companies. Furthermore, as for any small bio-tech, it was imperative that there be clear direction for the clinical trials with regard to both end-points and efficacy. Such measures are the foundation of health economic valuations that are now a critical component of any new drug application. With limited outcomes data available, due to the early development stage of the product, the company required an ‘interventional model’ to explore product efficacy (by the reduction of events downstream of the MI event) in relation to both product pricing and budget impact for healthcare providers.

Big idea

A model presenting a theoretical patient cohort representative of the target population was constructed. This was achieved by taking MI epidemiology data and extracting the population receiving revascularisation procedures and sub-dividing this cohort into patients suffering their first MI or a subsequent MI. This allowed for differential intervention points with the new medication. Rates for mortality, major adverse cardiac event and heart failure were then applied to each cohort over time. This created an expected patient flow baseline that could be coupled to any number of factors including downstream clinical events and their related costs, as well as a patient health utility score. The addition of an interventional treatment ‘arm’ enabled exploration of different efficacy thresholds at different intervention points. With these ‘simulated clinical outcomes’, cost and utility calculations could be compared against the baseline using a range of price points for different efficacy profiles.

Results & feedback

Delivered in six weeks, the model conveyed cost and efficacy levels in relation to the potential outcomes of the clinical trial. Through mapping patients by disease state, co-morbidity, and the propagation of risk, this enabled the client to assess the associated impacts of the compound’s required clinical effectiveness in relation to its potential price point. With this robust insight the client was able to adjust their Phase III clinical trial plan and attract investment from a top 10 pharmaceutical company.