Are Orphan Disease Areas Orphaned?

Pharma IQ News

Leading biopharmaceutical research consultancy Medical Marketing Research Group (MMR Group) has successfully completed two major orphan drug Opinion Leader identification projects for two of the world’s top 10 Pharma companies. In the process MMR Group has proven that Orphan Disease areas have wider medical support than commonly thought.

Orphan diseases are rare (often genetic) conditions.  As fewer people are affected, treatments for these diseases are assigned orphan drug status in many countries to incentivise R&D into the therapeutic areas.  

For the MMR Group, these two orphan disease projects represented a new challenge the company relished taking on.  Often there is a lack of knowledge amongst physicians about rare orphan diseases and diagnosis is complicated.  Additionally, related publications and symposia are infrequent, so traditional approaches to identifying Opinion Leaders do not work. However, with its robust research models and pioneering peer nomination process, the MMR Group overcame these hurdles with ease.

The first project focused on a rare genetic disorder with less than 20,000 patients affected globally. The condition has a prominence in certain populations, although spontaneous mutations amongst other populations are known to occur. Applying its proprietary approaches the MMR Group found experts in over 23 countries outside the major markets of Western Europe, USA, Canada, Australia and Japan.  Many of these experts were new to the client; had never been approached before and were not aware of the development of an orphan drug.  In all the MMR Group found more experts in the orphan disease field in six months of research than the company had identified in two years.

The second assignment involved an Opinion Leader study across multiple EU countries. The orphan disease only affects less than 1 individual in every 100,000 population, mostly affecting people over 40 and presents with a variety of symptoms, ultimately leading to anaemia.  Average survival rates being just five years, although some patients may live for decades.  The MMR Group carried out a global peer nomination study to identify OLs in the disease area and received peer nominations from 300 different individuals.  From these nominations, around 50 experts from the targeted EU countries were selected for the study and profiled by MMR Group for its client.

The result of these two projects proved to each client that although orphan disease areas are associated with a low incidence of patients, there are in fact many physicians in practice who are regarded and recognised by their peers as leaders in the treatment and management of these rare conditions. 


For more information on MMR-G and its research projects contact Neil Jones on:

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