Seminar für Wissenschaftsjournalisten «Krebszellen mit dem Immunsystem bekämpfen»

Transcription

1 Zürich, 15. März 2017 Stiftung zum Glockenhaus Seminar für Wissenschaftsjournalisten «Krebszellen mit dem Immunsystem bekämpfen» Herausforderungen in der Zulassung mit der Präzisionsmedizin Claus Bolte, M.D., M.B.A. - Zulassung (Marketing Authorization) Swissmedic Schweizerisches Heilmittelinstitut Hallerstrasse 7 CH-3000 Bern 9 1

2 Onkology - Intro 2

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4 Challenges Das Gesuchsvolumen (in der Onkologie / Hämatologie) hat in den letzten Jahren rapide zugenommen Die Pipelines fast aller forschenden Unternehmen sind gut gefüllt mit Onkologiepräpataten Die Arzneimittelentwicklung in der Onkologie unterliegt einem drastischen Wandel damit auch schwer übertragbare ausländische Gesuchsverfahren Orphanisierung (Sub-/Populationen, Indikationen) 4

5 Volume 2500 # (all applications - ATC) Dez 2000 Nov Okt 1500 Sep Aug Jul 1000 Jun Mai 500 Apr Mrz 0 Feb Jan 5

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9 Application - Timelines (days) Formal control Answer company Review 1 LoQ Answer LoQ Review 2 Prelim decision Answer of company Review of answer Total company time Total Swissmedic time NAS normal NAS Fast Track (BZV) NAS Prior Notification

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16 Limitations 16

17 Limitations 17

18 Example FULL PRESCRIBING INFORMATION (US) 1 INDICATIONS AND USAGE DARZALEX is indicated for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent. This indication is approved under accelerated approval based on response rate [see Clinical Studies (14)]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. Why is Darzalex approved? (EPAR summary/public) Darzalex has been shown to be effective at treating multiple myeloma in patients whose disease had progressed despite at least two previous treatments. These patients have limited treatment options and Darzalex, which works in a different way to existing treatments, represents an alternative. Darzalex s safety profile is considered acceptable and manageable. Although there were limitations in the studies, including lack of control group and low number of patients, the Agency s Committee for Medicinal Products for Human Use (CHMP) decided that Darzalex s benefits are greater than its risks and recommended that it be approved for use in the EU. Darzalex has been given conditional approval. This means that there is more evidence to come about the medicine, which the company is required to provide. Every year, the European Medicines Agency will review any new information that becomes available and this summary will be updated as necessary. E. SPECIFIC OBLIGATION TO COMPLETE POST-AUTHORISATION MEASURES FOR THE CONDITIONAL MARKETING AUTHORISATION This being a conditional marketing authorisation and pursuant to Article 14(7) of Regulation (EC) No. 726/2004, the MAH shall complete, within the stated timeframe, the following measures: In order to address the uncertainties related to the single arm design of the pivotal study supporting the approval of DARZALEX, the MAH should submit the results of study MMY3003, a phase III randomised study investigating lenalidomide and dexamethasone with or without daratumumab in patients with previously treated multiple myeloma. Due date: 30 September 2017 In order to address the uncertainties related to the single arm design of the pivotal study supporting the approval of DARZALEX, the MAH should submit the results of study MMY3004, a phase III randomised study investigating bortezomib and dexamethasone with or without daratumumab in patients with previously treated multiple myeloma. Due date: 31 December

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21 ASCO 16 Vice President Joe Biden Discusses Cancer Moonshot Initiative During ASCO 2016 June 6, 2016 As part of the ambitious Moonshot effort to eliminate cancer as we know it, Vice President Joe Biden encouraged collaboration and team science within the field of oncology during a speech at the 2016 ASCO Annual Meeting. The whole world is looking to you, Vice President Biden told the crowd of oncologists and medical professionals. Your success can literally change the world. We need you now more than we ever have. The speech highlighted the White House s Cancer Moonshot Initiative to accelerate cancer research efforts and break down barriers to progress by promoting data sharing and facilitating collaborations to advance cancer prevention, treatment, and care. 21

22 CDER Office of New Drugs Richard Pazdur, M.D. is the acting director of the FDA s Oncology Center of Excellence (OCE), which leverages the combined skills of the FDA s regulatory scientists and reviewers with expertise in drugs, biologics and devices to expedite the development of novel cancer products as part of the Vice President s National Cancer Moonshot Initiative. In his role as acting director of the OCE, Pazdur is responsible for leading the effort to develop and execute an integrated regulatory approach to enhance the cross-center coordination of oncology product clinical review. Pazdur previously served as the director of the Office of Hematology and Oncology Products (OHOP) in the FDA s Center for Drug Evaluation and Research. 22

23 Challenges Das Gesuchsvolumen (in der Onkologie / Hämatologie) hat in den letzten Jahren rapide zugenommen Die Pipelines fast aller forschenden Unternehmen sind gut gefüllt mit Onkologiepräpataten Die Arzneimittelentwicklung in der Onkologie unterliegt einem drastischen Wandel damit auch schwer übertragbare ausländische Gesuchsverfahren Orphanisierung (Sub-/Populationen, Indikationen) 23

24 Challenges Interconnected world post-trust Society Social Media & Transparency Medical need grows faster than healthcare budgets Empowered patients Personalized, Stratified, Precision Medicine HTA (NICE, IQWiG, etc.), cost-benefit analyses Pediatric (& Geriatric) Data Data Exclusivity New facilitated (expedited) licensing pathways New trial designs; RCT : RWD Master protocols, basket trials 24

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