PATIENT-BASED MODEL TO CHALLENGE PRE-EXISTING EVALUATION OF A NOVEL THERAPEUTIC FOR A POPULATION WITH HIGH UNMET MEDICAL NEED
A global biotechnology company had previously assessed the potential acquisition of a product with a novel mechanism of action, for use within a niche population with high unmet medical need. While the acquisition candidate was evaluated and found to fall short of the necessary threshold criteria in terms of forecast revenue potential, a similar asset was subsequently acquired by a top-ten multi-national pharmaceutical company. Our client now wishes to re-explore, in a robust and flexible way, the true potential of the agent for in-licensing. A patient-based model needs to be built, consisting of robust patient populations on which to evaluate the product. In addition, the model architecture must facilitate:
- The exploration of product potential in a number of patient populations
- The identification of key areas of unmet need across the USA and top-five European markets
- Incorporation of new drivers of product use, to be populated with market research outputs that may be generated closer to launch
The disease condition is poorly understood in terms of diagnosis, survival, risk factors and identifying patients who are likely to progress from a related pre-existing disease. Clinical groups active in this area are developing guidelines regarding diagnostic criteria, and clinical markers of disease progression and severity. These guidelines are driven by recent scientific advances and the rapidly increasing body of knowledge of the disease, as to the pathological pathways and mechanisms involved. A considerable challenge exists in trying to estimate realistic patient populations and include relevant criteria which are likely to be used to identify treatment candidates when the product eventually launches.
Peer-reviewed research publications were used to understand both the therapeutic target’s pathway and to identify and understand potential patient populations. These populations included those predisposed to the disease through specific genetic mutation to those diagnosed at a particular severity level. Flexibility was incorporated to facilitate the selection of clinically relevant variables. This will allow the client the ability to select patients most likely to respond positively to their target therapeutic and to modify their patient inclusion criteria as insight on disease progression evolves. Such profiling parameters will become more robust over time as more disease and clinical trial information becomes available.
Results & feedback
Black Swan Analysis identified and quantified different patient populations using literature resources. A patient-based model was constructed to allow valuation of the novel therapeutic against product and clinical developments in the emerging marketplace. The model deepened the clients’ working knowledge of the disease area and has assisted their understanding of key areas of risk and uncertainty. The client is currently completing their due diligence assessment in order to establish if the addition of this product to its portfolio is of significant commercial benefit.