Op 14 december 2017 heeft de 'Committee for Medicinal Products for Human Use" (CHMP) een richtlijn vastgesteld betreffende het klinisch onderzoek van geneesmiddelen voor de behandeling van reumatoïde artritis. Dir document en wel tweede revisie daarvan is van kracht geworden op 1 juli 2018.
Van belang is met name de paragraaf die handelt over primaire eindpunten. De letterlijke Engelse tekst die voor zich spreekt, luidt:
"An endpoint reflecting a target disease state (ideally remission, or Low Disease Activity (LDA) at the minimum) should be selected as the primary one, since these are established treatment targets in the field. Moreover, sustained remission, and to lesser extent LDA, are correlated with the prevention of structural damage and functional loss.
For studies on the treatment of naïve patients, remission at 3 to 6 months as established by a combined measure as listed above in Section 6 , Assessment of efficacy, is the preferred endpoint. For studies in patients who have inadequately responded to previous synthetic or biologic DMARD treatment, LDA at 3 or 6 months could be considered as the primary endpoint.
The primary endpoint of choice (either remission or LDA as defined by either DAS28-CRP, DAS28-ESR, SDAI or CDAI), should be corroborated by the other outcomes as secondary endpoints, such as the more stringent outcome SDAI remission, or by CDAI, which is the only outcome independent of biomarkers CRP or ESR.
Given that endpoints based on change of response criteria, such as ACR20, represent a relative change from baseline, these do not necessarily reflect treatment targets of remission or LDA. As their clinical relevance may not be immediately clear, improvement of response outcomes are in general not considered as primary endpoints. An exception may be a specific group of difficult to treat patients with an inadequate response to multiple DMARDs (synthetic and biologic) from different classes. A relative small improvement in disease activity without achieving LDA or remission may still be considered as a benefit for this specific group, and ACR20 at Month 6 is considered an acceptable primary endpoint."