"But when one looks at a potentially lifesaving treatment, admittedly expensive, and we have data to support efficacy, cuts like this are shortsighted and sad".
Precision Medicine will hopefully lead to scientifically selected, well-defined populations enriched for responders, enabling smaller databases to support efficacy.
The EU approval was subject to annual reviews and the provision of additional data to support efficacy and safety [11].
However, other classes have data supporting efficacy in migraine prevention, including antihistamines, hormonal agents, dopamine antagonists, nonsteroidal anti-inflammatory agents, DHE, and corticosteroids [12, 39, 47].
A clinical phase I trial supporting efficacy of this strategy in MM patients is currently ongoing.
Response rates were higher after 30 mg and 10 mg than placebo, with secondary endpoints also supporting efficacy.
A well-conducted evaluation can add to the weight of evidence supporting efficacy where mechanism of action is well understood.
In the absence of objective clinical endpoints to support efficacy, PRO measures are often used as primary endpoints.
The EU approval was subject to annual reviews and the provision of additional data to support efficacy and safety [ 11].
