How to successfully position an orphan product to investors

In Clinical Development, Market Access, Partnering & Investment by André Singer

It is well known that most biotechs fail to develop an orphan drug even when they have reached advanced stages of clinical trials. However, there’s one more stage where biotechs are stumbling, which is establishing the market potential for investors.

In an rapidly growing market for rare disease therapies, big pharmaceutical companies and other potential investors looking for in-licensing and partnering opportunities in the rare disease space, are looking for particular signs of validation to solidify these opportunities. The prospecting partners are not only looking for the positive clinical data, but also to understand the market potential – number of patients with the disease, how many would benefit from the therapy, market access strategies – the disease epidemiology and natural history and even insurance claims data.

Most of the times the biotechs are so focused on the science and navigating the regulatory hurdles, that the business and strategic components of the company and its compound values’ proposition is left aside.

At the World Orphan Drug Congress USA 2014, David Lapidus from LapidusData will be hosting a Workshop to explain the relevance of establishing a reliable estimate for an orphan product market’s potential and reduce the uncertainty of its revenue forecast.