FaegreBD Consulting has provided expert policy, program development and regulatory consultative support to rare disease stakeholders for more than 25 years. Our team is distinctive in that it consists of both seasoned public policy advocates, who have worked on rare disease issues for years, and clinical and regulatory experts, who have spent their careers navigating Food and Drug Administration (FDA) and other agencies around the globe to advance the development of medications to address unmet needs.

Our expertise includes understanding what is necessary to enact legislation to benefit rare disease interests, influence the complex guideline and regulation development process, and guide therapy sponsors and advocates in navigating the challenging FDA review process. Additionally, our perspective recognizes that rare disease policy does not begin with FDA. Rather, it often involves decades of work with the National Institutes of Health (NIH), Centers for Disease Control (CDC) and other agencies — as well as the medical practice community — to shape a robust basic research agenda and to use public health tools to gain invaluable information to inform rare disease research and improve care. Some examples of our areas of focus are highlighted below:

Orphan Drug Research & Development

• Benefit/risk assessment in rare vs. prevalent diseases
• Leveraging expedited process opportunities with FDA: breakthrough therapy designation, accelerated approval and fast track pathways
• Variable and adaptive trial design considerations

FDA Patient Engagement

• Relationship establishment utilizing collaborative platforms
• Part 15 hearings
• Citizens' petition

Advocacy

• Patient-focused drug development  in rare diseases — We focus on the importance of keeping programs in perspective similar to that of FDA. Regulatory sciences is the overarching theme for the FDA programs, one aspect of which is benefit risk assessment. Patient focused drug development is a critical subset of benefit risk. Our expertise is key in preparing companies and advocates to interact with regulators in their standard frame of decision making.
• Patient/caregiver view of benefit expectations and risk tolerance — We offer extensive experience in characterizing benefit and risk factors unique to specific disorders.

Drug Repurposing

• Combination therapies
• NIH, NCATS (National Center for Accelerating Transitional Science) — Our ongoing engagement with NCATS programs such as TRND (Therapeutics for Rare and Neglected Diseases) provides understanding of expectations in these critical endeavors.
• Academic research centers

FDASIA (Food and Drug Administration Safety & Innovation Act)

FDASIA makes clear the strong federal interest in accelerating efforts to develop therapies for rare diseases. Over the coming months and years, FDA will be drafting and seeking stakeholder inputs on a number of relevant FDASIA-related regulations. FaegreBD Consulting supports stakeholders through the following services:

• Engaging with FDA decision-makers before release of proposed regulations to help inform content and facilitate improvement efforts that benefit all stakeholders, especially patients
• Crafting high-impact comments to proposed regulations to help support or amend regulations and rules
• Convening stakeholders and forging robust alliances to bring influential voices to the table in support of one or more critical issues
• Engaging congressional and other leaders to assist on oversight and implementation of rare disease provisions to draft and advance supplementary provisions
• Identifying opportunities to expedite rare disease development programs

Representative Experience

• Successfully advocating for the enactment of legislation to advance the interest of rare disease patient communities, including the Children's Health Act, Muscular Dystrophy CARE Act, annual authorization and appropriations legislation, and rare disease provisions in FDASIA
• Enabled clients to develop public health programs at the Centers for Disease Control and Prevention (CDC), including epidemiological surveillance, patient registries, care standards and other initiatives to improve patient health
• Provided counsel to pharmaceutical and biotechnology companies seeking FDA review and approval of potential therapies for rare disease indications
• Worked with multiple NIH Institutes and Centers to initiate and foster programs focused on rare disease research
• Served as a catalyst and convener among patient, industry, academic and government participants to accelerate treatments for rare diseases and disorders, including an agreement between the NIH, a leading patient advocacy organization, and a top-tier academic research institution to repurpose a therapy for a rare disease application

In addition to advocating before Congress and the FDA, FaegreBD Consulting supports pharmaceutical and biotechnology companies, patient organizations and others in effectively navigating these waters as new or revised guidance and regulations are implemented. With a thorough understanding, our team has the capacity to address the ethical and marketing complexities associated with rare disease drug development, including issues such as compassionate use and combination therapy, as well as how to maximize an applicant's likelihood of success.