The impact of comparability on orphan drug development

In Market Access by Simon ReidLeave a Comment

As we’ve been hearing from pharma and biotech executives in the rare disease space in recent years, market access is a key element in supporting the expansion and development of orphan drugs around the globe. This issue is really dynamic as it is impacted by challenges from the very early drug discovery stage, to clinical trials, manufacturing and consequently the access to rare diseases patients. During research with pharma, it was mentioned that companies are struggling to get the FDA to consider that even though comparable drugs are different from a biochemical standpoint, they behave the same in animals, during pre-clinical studies, and in human, during clinical trials.Companies fear that if things remain the same, they would be delivering a drug that isn’t as good as it could be from its efficacy point of view.  Elements of this will be presented and discussed at the 2013 edition of the World Orphan Drug Congress USA 2013. As we’ve been hearing from pharma and biotech executives in the rare disease space in recent years, market access is a key element in supporting the expansion and development of orphan drugs around the globe.

This issue is really dynamic as it is impacted by challenges from the very early drug discovery stage, to clinical trials, manufacturing and consequently the access to rare diseases patients.

During research with pharma, it was mentioned that companies are struggling to get the FDA to consider that even though comparable drugs are different from a biochemical standpoint, they behave the same in animals, during pre-clinical studies, and in human, during clinical trials.

Companies fear that if things remain the same, they would be delivering a drug that isn’t as good as it could be from its efficacy point of view.

Elements of this will be presented and discussed at the 2013 edition of the World Orphan Drug Congress USA 2013.

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