Do regulating authorities such as the EMEA and FDA recognise advanced statistical analyses and simulation techniques?
Neither the EMEA or FDA seem to impose closed lists of authorised methods and non authorised methods. These authorities usually privilege discussion and method transparency, even if the dialogue is sometimes not very easy from the point of view of the pharmaceutical industry. Our recommendation would be not to substitute classical statistical analysis with sophisticated techniques, but to complete it. This would give regulatory authorities more complete information with which to enrich the decision-making process. On the other hand, FDA experts are perfectly aware of the limits of classical clinical assessments, particularly in rare diseases. Internal working groups in orphan drugs exist and very probably new guidelines for statistics and modelling will be issued very soon. Preliminary recommendations in pharmacogenetics were already published in 2005 (Pharmacogenetics – Toward improving treatment with medicines, CIOMS, Geneva 2005) by the Council for International Organizations of Med
