Engaging patient advocacy groups: The key to conducting effective rare disease research
This article, published by Pharma Times September 2019, outlines why market researchers should reach out to patient advocacy groups when conducting rare disease research.
Until recently, the size of the market for specific rare diseases had made it difficult for pharmaceutical companies to justify research and development costs. The introduction of regulatory benefits such as longer market exclusivity, breakthrough designations, reduced fees and tax incentives have encouraged more investment. Nevertheless, where funding has been allocated for rare diseases, the scarcity and geographical dispersal of patients can make it difficult to conduct effective market research. Identifying and recruiting respondents is one of the core challenges when conducting research in this space.
How to conduct an effective epidemiological data review
Clients often ask us if we can triangulate various data sources to fully understand the opportunity for an asset. There are clear benefits - combining primary market research findings with existing data sets provides validity and depth, whilst eliminating many inconsistencies, which helps guide the direction for future marketing strategy and initiatives.
How patient insights were brought to life with graphic novel outputs
Our client was developing a novel therapy for the treatment of a rare disease in which patients suffer acute symptomatic attacks. Their new product promised greater freedom from these acute attacks and therefore from the associated burden. The challenge was to understand why many patients remain on older therapy types and how the pathway to more modern therapy options, with better efficacy and side effect profiles, could be mapped out for physicians and patients. Our client further requested that insights from the research were brought to life in the insights workshop meeting in a new and impactful way.
How we used a hybrid qual-quant methodology to assess the market opportunity for a new rare disease treatment
Our client is a recently established independent biopharmaceutical company specialising in small molecule development. They were developing a new oral compound for the treatment of a rare genetic disease for which the standard treatment is administered intravenously, however a large proportion of patients receive monitoring only or remain undiagnosed. Their objective was to successfully launch their product in order to provide clinically meaningful benefits to patients and contribute to the overall growth of their business.
How we used behavioural economic theory to reveal fresh insights into patient compliance
Our client currently has a product for a chronic rare disease which has seen an initial successful uptake. HCPs appear to support the product and are actively prescribing it to suitable patients.
However, sales data suggests that repeat prescriptions drastically reduce after the first 3-6 months. Although the product has a known SE profile, our client believes that this can be well managed and controlled.