Sometimes tiny target patient and clinician groups – do we need to challenge basic principles or just conduct standard research on a smaller scale?
Parallels in orphan diseases & oncology
Orphan diseases have become an important part of pharma strategy, with significant acquisitions including the deal between Sanofi and Genzyme and key collaborations such as Pfizer/Protalix and GSK/Angiochem. As Orphanet states: “Rare diseases are rare, but rare disease patients are numerous.”
And with approximately 8,000 recognised orphan diseases and up to 10 new diseases being identified each month, the potential for orphan drugs is growing, not only in terms of sales within the indication, but also increasingly as an instrument of market access for later larger indications.
In a similar vein, oncology is a therapy area where patient pools are also becoming rapidly fragmented, almost to an ‘orphan status’ level. Oncology leads the change in the industry trend towards personalised medicine, with most of its R&D in targeted therapies that seek out individual mutations. This means crossover learning from researching other orphan diseases is increasingly helpful in oncology.
Quantitative testing versus cumulative thinking
We found we had to develop new approaches to fit the needs of these small target groups. It results in very small, ‘almost KOL-level’ prescriber groups, with geographic dispersal, who are often over researched. Therefore, the pressure is on to innovate in order to conduct high-quality research within industry compliance guidelines.
Asynchronous web-based approaches have proved very successful and we will focus on a couple of examples that can maximise insights.
When researching within this group, adopting ‘consulting’ rather than ‘testing’ tone gains much better insights. Clinicians want to interact and see what their peers are thinking, yet coordinating groups is a problem.
Online crowdsourcing to solve specific business issues, even if the crowd is small, can be really valuable in this respect, particularly if used like a modern day Delphi technique where clinicians have a direct hand in guiding the solution – as with our HealthBrain® tool – rather than merely giving feedback on a pre-developed solution.
As very few physicians drive market changes, sample sizes that are normally regarded as ‘qualitative level’ can be used effectively for conjoint studies and forecasting. Simalto-like exercises and other trade-off techniques take little time, but bring deep understanding when we supplement with rationale behind the trade-offs.
We now see a less rigorous divide between quantitative and qualitative research; rather, we see a broader tapestry of techniques for each facilitated by the web.
The wide geographic dispersal of these patients can actually be turned into an advantage, as patients often look to online communities for information – making social media research more attractive in these groups. Specially constructed, moderated social media sites can give a light touch to research, where respondents can log on at their leisure within agreed time intervals, maximising participation while minimising respondent disruption.
These sites, if managed well, gain their own momentum, with insight-yielding conversations continuing between research sessions.
One significant change is the treatment of our respondents as consultants, recruiting them for longer term relationships and asking their advice, rather than just testing our thoughts on them. In quantitative research in particular, this challenge needs to lead us to even more High Tech Light Touch approaches and as personalised medicine progresses, we believe that these approaches will become much more widespread.
The Authors
Caroline Mathie is head of orphan diseases of The Planning Shop international
caroline.mathie@planningshopintl.com
Kelly Price is senior vice president, oncology business unit,
of The Planning Shop international
kelly.price@planningshopintl.com