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Voice of the Patient Report for Spinal Muscular Atrophy

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Voice of the Patient Report for Spinal Muscular Atrophy V OICE OF THE PATIENT REPORT A summary report resulting from an Externally-Led Patient Focused Drug Development Meeting reflecting the U.S. Food and Drug Administration (FDA) Patient-Focused Drug Development Initiative Spinal Muscular Atrophy (SMA) Externally Led Public Meeting: April 18, 2017 Report Date: January 10, 2018 Title of Resource: The Voice of the Patient Report for Spinal Muscular Atrophy Authors: Contributors to the collection of the information and development of the document are: Cure SMA: Rosangel Cruz, Megan Lenz, Lisa Belter, Kenneth Hobby, Jill Jarecki Medical Writer: Theo Smart Cruz, Lenz, Belter, Hobby, and Jarecki are all employees of Cure SMA and have no disclosures. Cure SMA has received funding from certain companies for work on projects unrelated to the Patient-Focused Drug Development meeting. Funding Received: The report was funded by grants received from the SMA Industry Collaboration to support Cure SMA’s production and execution of the Externally-Led Patient-Focused Drug Development initiative for SMA and the engagement of an outside medical writing professional to assist in the development, editing, and production of The Voice of the Patient report for SMA. The members of the SMA Industry Collaboration are Astellas Pharmaceuticals, AveXis, Inc., Biogen, Genentech/Roche Pharmaceuticals, Cytokinetics Inc., Novartis Pharmaceuticals, and Ionis Pharmaceuticals, Inc. Version Date: January 10, 2018 Revision Statement: This resource document has not been revised and/or modified in any way after January 10, 2018. Statement of Use: Cure SMA has the necessary permissions to submit the “The Voice of the Patient for SMA” report to the U.S. FDA. Linking to the report from the FDA website will not violate the proprietary rights of others. Point of Contact/Corresponding Author: Rosangel Cruz Associate Research Director Cure SMA Email: [email protected] The Voice of the Patient A report resulting from an Externally-Led Patient---Focused Drug Development Meeting corresponding to FDA’s Patient-Focused Drug Development Initiative Spinal Muscular Atrophy Externally Led Public Meeting: April 18, 2017 Report Date: January 10, 2018 Hosted by: Cure SMA Submitted to: Center for Drug Evaluation and Research (CDER) & Center for Biologic Evaluation and Research (CBER) U.S. Food and Drug Administration (FDA) This report represents a comprehensive summary report composed by a patient advocacy organization as a result of an Externally-Led Patient-Focused Drug Development meeting; a parallel effort to FDA’s Patient-Focused Drug Development Initiative. This report reflects the Cure SMA’s account of the perspectives of patients and caregivers that participated in the public meeting. ii Table of Contents Introduction ................................................................................................................................................................. 1 Overview of SMA ..................................................................................................................................................................... 1 SMA type I ........................................................................................................................................................................... 2 SMA types II through IV ...................................................................................................................................................... 2 SMA treatment overview .................................................................................................................................................... 2 Meeting overview ................................................................................................................................................................... 3 Report overview and key themes ............................................................................................................................................ 4 Topic 1: Burden of disease in SMA ..................................................................................................................................... 4 Topic 2: SMA patient perspectives on Treatment Options ................................................................................................. 5 Benefit-Risk overview ......................................................................................................................................................... 6 Topic 1, SMA type I: Most significant symptoms and their impact on daily life ................................................................ 7 SMA type I: Perspectives on symptoms that matter most to patients and their caregivers .................................................. 7 SMA type I: Impact on daily life of patients and their caregivers ......................................................................................... 10 Topic 1, SMA type II/III: Most significant symptoms and their impact on daily life ........................................................ 12 SMA type II/III: Perspectives on symptoms that matter most to patients and caregivers ................................................... 13 SMA type II/III: The daily impact on patients and their caregivers ....................................................................................... 15 Topic 2, SMA type I: Patient and caregiver perspectives on treatment.......................................................................... 19 SMA type I: Experiences with prescription treatments and supplements ............................................................................ 19 TM Nusinersen/Spinraza ...................................................................................................................................................... 20 SMA type I: Experiences with multidisciplinary care to treat the symptoms of SMA ........................................................... 20 SMA type I: Perspectives on future treatments and considerations in treatment decisions ................................................ 22 Gains in function and strength, however small ................................................................................................................ 23 SMA type I: Perspectives on benefit-risk analyses ................................................................................................................ 23 SMA type I: Perspectives on clinical trials ............................................................................................................................. 23 Placebos ............................................................................................................................................................................ 24 Combination therapy and other SMA enhancing drugs ................................................................................................... 24 Topic 2, SMA types II and III: Patient and caregiver perspectives on treatment ............................................................. 25 SMA type II and III: Experiences with prescription treatments and supplements................................................................. 26 TM Nusinersen/Spinraza ...................................................................................................................................................... 26 SMA type II/III: Experiences with multidisciplinary care to treat symptoms ........................................................................ 27 SMA type II/III: Benefit-risk ................................................................................................................................................... 31 Perspectives on clinical trials ................................................................................................................................................ 31 Placebos and other clinical trial design issues .................................................................................................................. 32 Benefit-Risk survey for SMA ........................................................................................................................................ 33 Incorporating patient input into a benefit-risk assessment framework for SMA............................................................. 34 Conclusion .................................................................................................................................................................. 36 Appendix 1: Meeting Program, Includes Agenda and Discussion Questions .................................................................. 37 Appendix 2: FDA, expert, and meeting panel participants ............................................................................................ 38 Appendix 3: Polling Questions and Results (SMA type I-III) .......................................................................................... 40 Appendix 4: Post-meeting survey - SMA Patient-Focused Drug Development Meeting ................................................. 50 Appendix 5: Key Meeting Demographics (Polling results*) ........................................................................................... 56 Appendix 6: The Benefit-Risk survey, abridged results ................................................................................................. 58 iii Introduction On April 18, 2017, Cure SMA hosted an externally led Patient---Focused Drug Development
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