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LupusPFDDInitiative Title of Resource: Lupus: Patient Voices Authors:

- Lupus and Allied Diseases Association: Kathleen A. Arntsen - Lupus Foundation of America: Pat Wildman - Lupus Research Alliance: Diane Gross

- Consultants:

o Faegre Baker Daniels Consulting: Dave Zook, Lauren Bloch o Hyman, Phelps & McNamara, P.C.: James E. Valentine o YourEncore: Tim Franson

Disclosures: The Lupus and Allied Diseases Association, the Lupus Foundation of America, and the Lupus Research Alliance receive funding from pharmaceutical companies in the form of sponsorship of programs and events. David Zook and Lauren Bloch are employed by Faegre Baker Daniels LLC, a law and consulting firm that represents sponsors developing drugs and patient advocacy organizations. James Valentine is employed by Hyman, Phelps & McNamara, P.C., a law firm that represents sponsors developing drugs and patient advocacy organizations. Tim Franson, M.D. is Chief Medical Officer of YourEncore, a consulting and staffing company and also affiliated with Faegre Baker Daniels Consulting, both of which work with sponsors developing therapies and with patient organizations. Franson is also a member of the Board of Directors for the Critical Path Institute, the US Pharmacopeia, Paratek Pharmaceuticals and Cidara Therapeutics. Other individual authors have nothing to disclose. Funding: The Lupus Patient-Focused Drug Development Meeting was designed and implemented entirely by the Lupus and Allied Diseases Association, the Lupus Foundation of America, and the Lupus Research Alliance. The resulting Lupus: Patient Voices Report also was prepared by these organizations. Sponsorship of the meeting was provided by Aurinia Pharmaceuticals, the Biotechnology Innovation Organization, Bristol-Myers Squibb, Eli Lilly and Company, EMD Serono, Genentech, GlaxoSmithKline, Mallinckrodt Pharmaceuticals, and UCB. These funds were used to secure meeting space and to support other logistical and consulting services for the initiative. The Lupus and Allied Diseases Association, the Lupus Foundation of America, and the Lupus Research Alliance explicitly state that the sponsoring companies had no control over the meeting content or related activities. Version Date: March 6, 2018 Revisions and Modifications: This document has not been revised and/or modified after the version date listed above. The submitters have received all necessary permissions to submit this external resource to the FDA. Linking to this resource from the FDA website does not violate the proprietary rights of others. Points of Contact:

- Kathleen A. Arntsen, Lupus and Allied Diseases Association, kathleen@ladainc.org - Pat Wildman, Lupus Foundation of America, wildman@lupus.org - Diane Gross, Lupus Research Alliance, dgross@lupusresearch.org

Lupus: Patient Voices

Report on Externally-led Patient-Focused Drug Development Meeting: September 25, 2017

Released March 6, 2018

Submitted as patient experience data for consideration pursuant to section 569C of the Federal Food, Drug, and Cosmetic Act to:

Center for Drug Evaluation and Research (CDER) U.S. Food and Drug Administration (FDA)

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A Message of Gratitude

The Lupus and Allied Diseases Association, the Lupus Foundation of America, and the Lupus Research Alliance are pleased to present Lupus: Patient Voices, a summary report composed as a result of an Externally-led Lupus Patient-Focused Drug Development (PFDD) Meeting, a parallel effort to the U.S. Food and Drug Administrations (FDA) Patient-Focused Drug Development Initiative. The report reflects the three organizations account of the perspectives of people with lupus and their representatives who participated in the public meeting, responded to the pre-meeting survey, and/or submitted post-meeting comments.

On behalf of the three collaborating organizations, we would like to recognize and thank the various stakeholders who helped to make this groundbreaking initiative possible. We extend sincere appreciation to the many lupus groups, healthcare providers, and researchers who provided input; the biopharmaceutical sponsors for their generous support; the Food and Drug Administration for working with us; and, above all, the people with lupus and their loved ones who promoted and participated in this project while inspiring us along the way. We gratefully acknowledge the efforts of the entire lupus community for understanding the value of the project in driving new therapies forward and for being engaged from day one in helping to make it a success.

Lupus remains a devastating and poorly understood disease: it is difficult to diagnose, with few proven treatments, and significantly impacts the lives of those affected. Even the current number of people in the U.S. with lupus is unknown, although estimates place it anywhere from 322,000 to 1.5 million. Despite recent improvements in mortality rates, lupus and its associated complications can still be fatal. While many dedicated researchers are diligently working to better understand this elusive disease and identify possible treatments, there is still much to be done in delivering optimal therapies.

For over two years, our organizations have worked tirelessly to make the PFDD meeting and resulting report a reality. Although it was our goal to show the diversity of the population impacted and the variability in disease manifestations through pre-meeting survey data, and over 2,100 people responded, we realize that the information collected is not truly reflective of the overall patient population. We are however, deeply indebted to the many individuals with lupus and their representatives who took the time to complete the survey, which helped us to craft a meaningful meeting agenda and provided valuable insight in understanding their experiences and priorities.

Our community stands united in its desire to improve the lives of people with lupus and this report represents a significant step in advancing therapies for this complex and debilitating disease. We sincerely hope that the FDA and drug developers will use this information to continue to move us forward in achieving that important goal.

Kathleen A. Arntsen Sandra C. Raymond Kenneth M. Farber President and CEO CEO President and CEO Lupus and Allied Diseases Association Lupus Foundation of America Lupus Research Alliance

Contents Report Summary ................................................................................................................................1

Introduction ......................................................................................................................................4

Overview of lupus and available treatments ............................................................................................ 4

Meeting design and data collection .......................................................................................................... 5

Demographic data for survey respondents .............................................................................................. 7

Disease characteristics of survey respondents ......................................................................................... 7

Topic 1: Impact of Disease Symptoms ............................................................................................... 10

Most distressing symptoms .................................................................................................................... 10

Impact of lupus symptoms on daily life .................................................................................................. 18

Topic 2: Perspectives on Treatments for Lupus ................................................................................. 22

Positive impact of treatments................................................................................................................. 24

Negative impact of treatments ............................................................................................................... 25

Factors that impact choice of treatment ................................................................................................ 27

Treatment preferences and perspectives on an ideal treatment ........................................................... 27

Participation in clinical trials ................................................................................................................... 28

Incorporating Patient Input into a Benefit-Risk Framework for Lupus ................................................ 31

Conclusion ....................................................................................................................................... 34

Appendix 1: Meeting Agenda ............................................................................................................. i

Appendix 2: Meeting Speakers .......................................................................................................... iii

Appendix 3: Meeting Discussion Questions ........................................................................................ iv

Appendix 4: Meeting Polling Questions .............................................................................................. v

Appendix 5: Pre-Meeting Survey Questions ....................................................................................... ix

Lupus: Patient Voices 1 | P a g e

Report Summary Lupus is a chronic, systemic, and often disabling autoimmune disease with an unpredictable course and ina