It is recommended that the Should the Delegate request This response is sponsor amends the RMP to list the RMP be amended as considered the use of follow up forms as recommended by the unacceptable. routine risk minimisation evaluator, Biogen Idec will The RMP is not the activity. amend the RMP to list the use Delegate’s of follow up forms as routine responsibility but that risk minimisation activity. of the RMP evaluator and the director of the RMP section. Furthermore, in accordance with the relevant EU guideline, the use of specific questionnaires as a follow-up to a reported suspected adverse reaction is considered to be routine pharmacovigilance. Consequently the RMP should be amended accordingly. The RMP in its current version is unacceptable. The RMP is lacking The eligibility criteria for the This response is discussion/provision of Phase III study were selected considered insufficient. information about certain to ensure a representative The sponsor states: A clinical aspects highly relevant study population of PTPs revised RMP will to the use of the product in the with severe haemophilia A. include a discussion on targeted patient population. No inclusion or exclusion the effect of the This includes the increased risk criteria in the Phase III study exclusion criteria of antibody development for restricted the study across the clinical patients with high risk gene population based upon gene program and the mutations (Gouw et al., Gosh et mutations. In addition, implications for al.). It is recommended that the genotype data were collected treatment of the target sponsor revised the RMP to from study participants and population. However, include discussion on this point, was representative of the the sponsor has not and describes how it has been expected mutation submitted the updated considered in the clinical frequencies in the general RMP, and it is unclear development program and in population of patients with when the sponsor is the generation of the RMP. haemophilia A. A revised going to submit the RMP will include a discussion updated RMP. on the effect of the exclusion This recommendation criteria across the clinical remains. program and the implications for treatment of the target population. The consumer medicine Biogen Idec believes that the This response is information (CMI) and risk management described considered directions for use (DFU) are in the CMI and DFU is based unacceptable. In considered part of the risk upon information provided accordance with the
It is recommended that the sponsor amends the RMP to list the use of follow up forms as routine risk minimisation activity. Should the Delegate request the RMP be amended as recommended by the evaluator, Biogen Idec will amend the RMP to list the use of follow up forms as routine risk minimisation activity. This response is considered unacceptable. The RMP is not the Delegate’s responsibility but that of the RMP evaluator and the director of the RMP section. Furthermore, in accordance with the relevant EU guideline, the use of specific questionnaires as a follow-up to a reported suspected adverse reaction is considered to be routine pharmacovigilance. Consequently the RMP should be amended accordingly. The RMP in its current version is unacceptable. The RMP is lacking discussion/provision of information about certain clinical aspects highly relevant to the use of the product in the targeted patient population. This includes the increased risk of antibody development for patients with high risk gene mutations (Gouw et al., Gosh et al.). It is recommended that the sponsor revised the RMP to include discussion on this point, and describes how it has been considered in the clinical development program and in the generation of the RMP. The eligibility criteria for the Phase III study were selected to ensure a representative study population of PTPs with severe haemophilia A. No inclusion or exclusion criteria in the Phase III study restricted the study population based upon gene mutations. In addition, genotype data were collected from study participants and was representative of the expected mutation frequencies in the general population of patients with haemophilia A. A revised RMP will include a discussion on the effect of the exclusion criteria across the clinical program and the implications for treatment of the target population. This response is considered insufficient. The sponsor states: A revised RMP will include a discussion on the effect of the exclusion criteria across the clinical program and the implications for treatment of the target population. However, the sponsor has not submitted the updated RMP, and it is unclear when the sponsor is going to submit the updated RMP. This recommendation remains. The consumer medicine information (CMI) and directions for use (DFU) are considered part of the risk Biogen Idec believes that the risk management described in the CMI and DFU is based upon information provided This response is considered unacceptable. In accordance with the
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