risk management approach in in accordance with best Australia.’ practice. 8. The sponsor should Sponsor confirms educational The sponsor’s response ‘The make available the materials will be made available to all has been noted. prescriber education health care professionals involved in However, that a materials to all health care the treatment of a patient taking multidisciplinary team is professionals involved in eliglustat. They will be distributed using likely to provide care for the treatment of a patient a variety of methods which may include patients with Gaucher taking eliglustat. electronic or hard copy formats to disease, the ensure accessibility to all key recommendation stakeholders involved in the remains. management of Gaucher patients. The sponsor should The prescriber education materials make available the for Prescriber) are intended for prescriber education the physicians/specialists who materials not only to initiate/supervise treatment with prescribers, but to all eliglustat only. The physicians have the health care professionals responsibility to discuss/remind the involved in the treatment patient the importance of using the of a patient taking Patient Alert Card. The Patient Alert eliglustat. Card is a liaison tool to provide educational materials to all the other healthcare professionals who are involved in the treatment of Gaucher disease. It focuses particularly on the potential drug-drug interactions prior prescription or delivery of any additional medicinal products (including herbal products). All healthcare professionals are encouraged to to the Product Information or contact Sanofi Medical Information for additional information required. 9. Given that the sponsor rationale for the single dose The sponsor’s response ‘The has not proposed a regimen is explained in detail in the has been noted. titration schedule for clinical documentation included with ACSOM made the eliglustat but proposes that the application and subject of clinical following comments: patients take the 84 evaluation. As demonstrated during mg/100 mg dose from the clinical development the safety profile noting the relatively ‘In start, the sponsor should of eliglustat in GD1 patients does not population group outline what additional warrant any additional monitoring as affected by GD (and that monitoring should occur, part of the initial period of most patients live in or in this initial period of administration.’ around Sydney), the administration. committee made the additional suggestion that it would also be useful to consider implementing ongoing therapeutic monitoring protocols (given the variability of results shown in the clinical trial data) and to also have a set of guidelines to ensure
risk management approach in Australia.’ in accordance with best practice. 8. The sponsor should make available the prescriber education materials to all health care professionals involved in the treatment of a patient taking eliglustat. ‘The Sponsor confirms educational materials will be made available to all health care professionals involved in the treatment of a patient taking eliglustat. They will be distributed using a variety of methods which may include electronic or hard copy formats to ensure accessibility to all key stakeholders involved in the management of Gaucher patients. The prescriber education materials for Prescriber) are intended for the physicians/specialists who initiate/supervise treatment with eliglustat only. The physicians have the responsibility to discuss/remind the patient the importance of using the Patient Alert Card. The Patient Alert Card is a liaison tool to provide educational materials to all the other healthcare professionals who are involved in the treatment of Gaucher disease. It focuses particularly on the potential drug-drug interactions prior prescription or delivery of any additional medicinal products (including herbal products). All healthcare professionals are encouraged to to the Product Information or contact Sanofi Medical Information for additional information required. The sponsor’s response has been noted. However, that a multidisciplinary team is likely to provide care for patients with Gaucher disease, the recommendation remains. The sponsor should make available the prescriber education materials not only to prescribers, but to all health care professionals involved in the treatment of a patient taking eliglustat. 9. Given that the sponsor has not proposed a titration schedule for eliglustat but proposes that patients take the 84 mg/100 mg dose from the start, the sponsor should outline what additional monitoring should occur, in this initial period of administration. ‘The rationale for the single dose regimen is explained in detail in the clinical documentation included with the application and subject of clinical evaluation. As demonstrated during clinical development the safety profile of eliglustat in GD1 patients does not warrant any additional monitoring as part of the initial period of administration.’ The sponsor’s response has been noted. ACSOM made the following comments: ‘In noting the relatively population group affected by GD (and that most patients live in or around Sydney), the committee made the additional suggestion that it would also be useful to consider implementing ongoing therapeutic monitoring protocols (given the variability of results shown in the clinical trial data) and to also have a set of guidelines to ensure
auspar-eliglustat-150818.docx