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Industry Perspective: Expanded Access Programs

Industry Perspective: Expanded Access Programs. Barbara Klencke, M. D. Senior Vice President, Development Onyx Pharmaceuticals, an Amgen Subsidiary. Disclaimer.

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Industry Perspective: Expanded Access Programs

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  1. Industry Perspective: Expanded Access Programs Barbara Klencke, M. D. Senior Vice President, Development Onyx Pharmaceuticals, an Amgen Subsidiary

  2. Disclaimer The views expressed herein represent those of the presenter and do not necessarily represent the views or practices of the presenter’s employer or any other party.

  3. Types of Access • Approved medicines • Regulatory approval provides broadest access • Expanded Access • Single patient and • treatment protocols • Primary goal: access • Clinical Trials • Evaluation of safety and efficacy of medicines to support approvals

  4. Goals of Expanded Access/Compassionate Use Provide seriously ill patients, who have no other treatment options, access to investigational new drugs that are not yet approved, based on a request from the treating physician

  5. Expanded Access • 1 • 2 • Unapproved • Therapies • ApprovedTherapies Access in a region beyond where a product is currently approved Access for an unapproved indication Access to a product not yet approved anywhere in the world, in any indication

  6. Expanded Access Considerations Sufficient Benefit/Risk Data Patient Need Defined Approval Pathway Program Viability • Medicine is under active development in the indication • Regulatory strategy determined and underway • Sufficient clinical evidence of safety and effectiveness established • Sufficient medicine to ensure adequate supply for clinical trials • Sufficient staff to support program • Regulatory mechanism in the region to support expanded access The patient has a serious/ life threatening disease or condition No comparable or satisfactory alternative therapy

  7. Case Study: Partnering for Patient Access Collaborationwith Multiple Myeloma Research Foundation Patient access prior to FDA accelerated approval Access for more than 300 patients

  8. Next Steps • Shared goal: Broad patient access to important medicines Consider all types of access: • Rapid regulatory approval of safe, effective new medicines • Clinical trials • Expanded Access Partnership and support of FDA, clinical trials

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