Collaborating with Patient Advocacy Organizations in Drug Development

A panel of Patient Advocacy Organization leaders spoke to an audience of 65 senior physician executives from the Boston CMO Network on Dec 2, 2014. The event was moderated by Matt Sherman, MD, CMO of Acceleron and hosted by Biogen Idec in Cambridge, MA.

Walter Capone, President & CEO of the Multiple Myeloma Research Foundation (MMRF)

Walter Capone discussed MMRF’s history and mission and its impact on new drug development. Founded in 1998, the MMRF is the largest private funder of multiple myeloma research. MMRF functions as a virtual drug development entity, providing funding and fostering collaborations from discovery research through target identification, clinical development and registration. MMRF builds extensive networks of partnerships across academic research institutions, pharmaceutical companies and government entities.

Examples of MMRF programs include:

  • • The Multiple Myeloma Research Consortium (MMRC). Integrating 21 leading centers and facilitating tissue banking, the MMRC speeds Phase I/II clinical trials
  • • MMRF Research Cores – a collaboration with leading academic research institutions to accelerate pre-clinical R&D into the clinic
  • • Partnerships with industry across a pipeline of 50 products addressing multiple pathways
  • • Establishment of business processes across institutions and companies to speed clinical trial initiation and enrolment
  • • CoMMpass Study – a landmark, 8 year study to collect clinical and molecular profiling data on 1,000 newly diagnosed patients at over 90 sites globally. These data are provided via a Research Gateway to MMRF industry partners
  • • MMRF recently brought together NCI, FNIH and FDA along with academic and industry partners to initiate the development of a MM Master Protocol to allow patients to participate in clinical trials evaluating several investigational therapies at once.
  • • Patient Support Programs ranging from a myeloma/oncology trained nurse call-center, to on-line Community Gateway of >3,000 ‘connected’ patients, to continuing medical and patient education programming


Link to Slides: Walter Capone – MMRF Overview

Gina Cioffi, Esq., National Executive Director of the Cooley’s Anemia Foundation (CAF)

Gina Cioffi described the CAF’s mission and some of its initiatives to accelerate medical research in thalassemia:
• Fellowships to launch the careers of young scientists
• Funding to support academic researchers
• Grants to support trials of gene therapy

CAF support of medical research has led to advances in the treatment and understanding of thalassemia by exploring the role of hepcidin as a regulator of iron metabolism, evaluating the efficacy of iron chelator combination therapy as well as gene therapy and gene editing research. CAF is working with thalassemia experts to develop evidence-based guidelines for treatment.

Registries are central to research in rare diseases. CAF has also developed a registry of patients in the U.S. of ~1,000 patients and is expanding that registry via a collaborative agreement with CDC. Registries allow researchers to assess feasibility of clinical trials and trial planning.

A major initiative of CAF is to help patients understand clinical trials. CAF is conducting nationwide focus groups to explore patient attitudes toward clinical trials, as well as barriers and facilitators to adherence.

The goals are to:

• Better understand demographics, epidemiology, population size and access to care.
• Provide information on modifiable patient behaviors
• Evaluate co-morbid conditions
• Enhance and refine health messaging to the thalassemia community.

Link to Slides: Gina Cioffi – Clinical Trials in Thalassemia Insights from the Patient Community

Christine McSherry, RN, Executive Director of Jett Foundation, Co-Founder of Duchenne Alliance

Christine McSherry discussed her personal story as the mother of a child with Duchenne’s Muscular Dystrophy (DMD), the founder of a patient advocacy organization and the co-founder of an alliance of patient organizations.

The Duchenne Alliance is a group of independent non-profit organizations dedicated to defeating DMD. Christine co-founded the Duchenne Alliance to bring together many grassroots organizations to achieve a common goal.

Working together gives the members of the Duchenne Alliance a stronger voice. Each member seeks to achieve its own mission while collectively serving the entire Duchenne community.

Some of the initiatives of the Duchenne Alliance include:

  • • Communicating with sponsors and regulatory agencies to gather information and provide access to that information to families and patients
  • • Collaborating to co-identify, co-review, and co-fund the most promising biomedical research
  • • Establishing the Duchenne Alliance Research Fund to receive monetary donations and direct these resources to advancing the top biomedical research and clinical trials


The Jett Foundation and the Duchenne Alliance collaborate with pharmaceutical companies. Christine noted that the Duchenne Alliance and the companies have the same mission and the same purpose; to discover and develop a safe and effective drug for DMD. Christine stressed the importance of learning about sponsor limitations and how patient organizations can have the most impact.

Christine discussed a case history on the impact of the Duchenne Alliance. She became aware of a medication in a small clinical trial that was showing efficacy. She and her colleagues realized that there was an unmet need to include patient involvement in the regulatory process and became very active in interactions with FDA. They were able to educate the agency on the daily life of a child and family with DMD and provide input on permissible risk in clinical trials. As a result of these efforts, the FDA has allowed expanded access to this medication in a clinical trial for older children with more advanced disease.

Link to Slides: Christine McSherry – Case Study, From Advocacy to Action

Glenn Pierce, MD, PhD – Former SVP, Hematology, Cell and Gene Therapies, Biogen-Idec
Past President of the National Hemophilia Foundation

Glenn Pierce shared his unique experience from both sides of the Advocacy-Biotech axis. Glenn has been a volunteer for hemophilia organizations since age 14, having lived with hemophilia until his liver transplant in 2008. He is also an accomplished biotech executive and worked in hemophilia R&D since 2002. He led the teams at Biogen Idec that developed Alprolix (rFIXFc) and Eloctate (rFVIIIFc).

Glenn reviewed the history of hemophilia and the evolution of its treatment. He noted that hemophilia has strong advocacy groups, in part as a result of the past history of viral transmission, (HIV, HCV, HBV), prior to the development of recombinant clotting factor replacements.

Glenn discussed a number of best practices for Biotech companies with advocacy groups:

  • • Partner with an advocacy relations team and/or encourage corporate support to develop an advocacy relations function
  • o Understand the advocacy organization’s values and partnership parameters
  • o Seek to develop innovative partnerships that are win/win
  • • Identify corporate internal needs that can benefit from partnering with advocacy (e.g., direct sales support, clinical trial recruitment)
  • • Engage with, listen to and learn from the advocacy organizations in your space:
  • o Crucial for all functions to be involved; demonstrate caring and commitment
  • o Understand the advocacy organization’s priorities and unmet needs
  • o Real commitment and transparent communications foster trust and long-term relationships
  • • Identify a natural intersection that also enables the advocacy organizations to maintain independence


Some common areas of collaboration can include:
• Clinical trial education and awareness
• Unbranded or disease-state educational programming
• HCP/Patient educational programs
• Advisory Board/focus group recruitment
• Government advocacy & payer relations

Glenn described how Biogen Idec reached out to the National Hemophilia Foundation, including early, frequent communication with CBER and physician organizations. Biogen Idec was able to enrol Phase 3 studies in ~2 years as a result.

Patient Advocacy and Drug Development – Accessing The Patient Voice

Glenn noted that health reforms worldwide are altering relationships between providers of goods and health systems, bringing increased importance to the patient voice in determining value. Increasing patient engagement early presents opportunities to improve understanding of real world clinical effectiveness and the ultimate value of drugs to patients.

Patients often won’t know to comment or engage unless formally invited or asked. Biotech companies should recognize this and reach out to patient advocacy organizations. Biotech companies should ensure that patients are not just observers of the process.

Link to Slides: Glenn Pierce – Advocacy and Drug Development

About the Boston CMO Network

The Boston CMO Network includes senior physician executives in the greater Boston area who are active in the biotech and pharmaceutical industry. The Network sponsors events for Boston-area physician executives to meet, interact and learn from each other.

A Steering Committee of physicians plans the group’s events:

Akshay Vaishnaw, M.D., Ph.D. (Chair) EVP & CMO, Alnylam Pharmaceuticals
Julie Krop, M.D. VP, Clinical Development, Vertex
Alfred Sandrock, Jr., M.D., Ph.D. SVP, Development Sciences & CMO, Biogen Idec
Matt Sherman, M.D. CMO, Acceleron Pharma
Nancy Simonian, M.D. CEO, Syros Pharmaceuticals
Gloria Vigliani, M.D. Principal at Vigliani Consulting
Chris Wright, M.D., Ph.D. Former SVP, Global Medicines Development, Vertex
Steven Rauscher (Organization) BioPharm Physicians


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