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How we used a hybrid qual-quant methodology to assess the market opportunity for a new rare disease treatment

How we used a hybrid qual-quant methodology to assess the market opportunity for a new rare disease treatment

The challenge:
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.

The solution:
We recommended using a two-phase qual-quant methodology to capture current insights and validate the hypotheses from previous research. Due to the dispersed nature of the respondents and a limited universe of treaters, we began by using virtual online trios to qualitatively explore physicians’ current perceptions and treatment approach. We then conducted a more robust quantitative phase using online surveys to explore unmet needs and size the market opportunity for a novel, oral compound treatment.

The outputs:
The initial qualitative phase enabled interaction and idea generation. It provided an opportunity for collaborative enquiry with physicians to test and discuss hypotheses and understand changing needs and markets dynamics. We were able to assess relevance of pre-determined patient profiles, discuss endpoint data for the new product, and explore the relevance of a future paediatric license for a new product.

By conducting the quantitative phase, we were able to help our client to understand more about patient volumes, subtypes, and flow, including referral pathways, time to diagnosis and treatment.  We explored current treatment approaches and factors that influence treatment decisions including price sensitivity. In addition, we were able to gauge reactions to the product TPP and assess the anticipated level of adoption across patient subtypes, including the likelihood to switch patients from the standard treatment. Using insights from the research, our client was able to develop an effective launch strategy for their product.

Find out more about our experience in rare diseases »

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