Thursday, 4 September 2014

EMA: the regulatory challenges in MS drug development

No discussion room for the Big Pharma at the EMA. #MSBlog #MSResearch

"This opinion paper written by the members of the European Medicines Agency (EMA) is refreshing. It deals with several topical issues in relation to developing drugs in MS, for example (1) new outcome measures in MS, (1) new aspects of MS disability, (3) developing MS drugs in the paediatric setting, (4) placebo, patient-reported outcomes and biomarkers in MS and (5) development approach for new MS drugs. They are still discuss the 2-stepped approach, i.e. developing drugs in series, first a trial in MSers with sub-optimal response and if the safety looks okay to then do the second trial in MSers naive to previous treatment. This is in contrast to the 1-stepped approach in the drug is tested in both groups in parallel. The serial approach will take 5-years longer, eat into patent-life and make drug development even more expensive. The one-stepped approach will allow MSers quicker access to drugs. I note they acknowledge that MSers' attitudes to drugs has changed: '... especially the strong position expressed by the patients that they would be ready to accept the more aggressive treatments and their risks, when sufficient data on efficacy are available, has been received by the regulators and is being considered in the update of the guideline'. This would indicate that they are listening. Unfortunately the article does not mention repurposing of existing drugs so there appears to be no space for the BPA (Big Pharma Alternative)."



Improving and facilitating the process of making new drugs available to MSers requires cooperation among the regulators and other stakeholders. This cooperation will also positively contribute towards developing guidelines of the highest quality in medical, regulatory and scientific aspects. This would be beneficial both in areas that require further guideline development, but also in fields where existing guidance should be adapted to take into account evolution in science. Considering the input from all stakeholders, the European Medicines Agency confirmed its intention to update the relevant guideline and apply a flexible approach towards new drug development strategies in MS.

"Please note one of the hot debates at ACTRIMS-ECTRIMS next week will cover the BPA in a different guise. Please tune in if you can."

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