BIO and PPMD Launch Initiative to Share Best Practices on Patient Preference Studies

Today, the Biotechnology Industry Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) announce the launch of a new initiative designed to share best practices for the development of disease-specific patient preference studies. Through this effort, BIO and PPMD will produce a document outlining key considerations to help guide stakeholders on the development of patient preference studies, which can be used for multiple purposes, including informing the drug development and regulatory processes.

Because each rare disease is unique, PPMD has proposed that special considerations and requirements should be made in benefit-risk decision-making. PPMD has recommended new approaches for regulatory benefit-risk assessments be considered for Duchenne and other rare conditions.

Terming this new approach as “BRAVE-rare diseases” (Benefit Risk Assessment, Valuation (and) Epidemiology (for) rare diseases), PPMD first launched a pilot study in 2013, and followed that with an industry specific study completed this past fall. PPMD is currently in the process of further expanding studies to better understand meaningful benefit and uncertainty. PPMD believes providing a community-centered approach for the benefit-risk framework is fundamental to the regulatory process and to reflect the community’s tolerance of potential risks or uncertainty of benefit associated with new treatment options.

“This timely project emerged in response to the clear need within our communities to share best practices and equip stakeholders with the tools and capabilities needed to advance patient engagement initiatives, such as the patient preference study pioneered by PPMD and Johns Hopkins. People are eager to learn from this and other experiences to advance these kinds of studies to help all of us meet the needs of patients.”

-- BIO President and CEO Jim Greenwood

“For over 20 years, PPMD has sought to ensure that the patient voice was included in the decision-making process. We’ve often said this is about quantifying tears – how can we capture the patient voice and turn it into usable data for industry and the FDA who need more than just patient stories to advance drug development.”

-- Pat Furlong, Founding President and CEO, Parent Project Muscular Dystrophy

To help guide this initiative, BIO and PPMD have assembled an Expert Review Committee (ERC) of thought leaders representing the patient community, academia, and the biopharmaceutical industry. The ERC will provide insights individually and as a group to shape and inform the output of this project.

This initiative will examine key considerations related to patient preference study development and implementation, interaction with the regulatory process, utilization of patient preference data, and model collaborations among the stakeholder groups, including patient organizations, academia, and industry.

Members of the ERC include: Marc Boutin, JD, Chief Executive Officer, National Health Council; John Bridges, PhD, Associate Professor, Johns Hopkins University; Paul Hastings, President and CEO, OncoMed Pharmaceuticals, Inc.; Stacy Holdsworth, PharmD, Senior Advisor, US Regulatory Policy and Strategy, Eli Lilly and Company; Reed Johnson, PhD, Senior Research Scholar, Duke University; Bennett Levitan, MD, PhD, Senior Director, Benefit-Risk Assessment, Janssen Research & Development, LLC, Johnson & Johnson; Kimberly McCleary, Managing Director, FasterCures; and Bray Patrick-Lake, Director of Stakeholder Engagement, Clinical Trials Transformation Initiative. The project is being led by a team of BIO and PPMD staff, working with Wendy Selig of WSCollaborative.

"Our organizations are honored to have such distinguished individuals as part of the ERC for this initiative. The members of the ERC are visionaries in the space of patient engagement, and their work has created tremendous opportunities to advance the needs of patients”

-- BIO’s Jim Greenwood and PPMD’s Pat Furlong

About BIO

BIO is the world's largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial and environmental biotechnology products. BIO also produces the BIO International Convention, the world’s largest gathering of the biotechnology industry, along with industry-leading investor and partnering meetings held around the world. BIOtechNOW is BIO's blog chronicling “innovations transforming our world” and the BIO Newsletter is the organization’s bi-weekly email newsletter. Subscribe to the BIO Newsletter.

About PPMD

Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy – their mission is to end Duchenne. PPMD invests deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. They advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. They demand optimal care, and strengthen, unite and educate the global Duchenne community. Everything PPMD does – and everything they have done since their founding in 1994 – helps people with Duchenne live longer, stronger lives. For more information, visit

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