Critical Regulatory & Development Support for Rare Disease Innovations

We Don’t Just Consult.
We Champion Breakthroughs.

Behind every development program we support through the U.S. Food and Drug Administration (FDA) submission process is a person — a child, a parent, a sibling, a friend — whose life has been profoundly affected. Our mission isn’t just accelerating approvals for novel therapies that improve patient outcomes. We are driven to:

• Start with the Patient — Insights from patients, caregivers, and advocacy groups can guide a competitive program and make a real commercial difference.
• Move Urgently — We operate with a sense of urgency that matches the needs of patients who cannot wait.
• Ensure Access — We design access programs from day one so that when treatments are approved, every patient who needs them can obtain them.
• Stay the Course — We remain committed to rare disease communities for the long term, through setbacks, pivots, and ultimately, breakthroughs.

The FDA Is Opening New Doors for Rare Disease Patients

The FDA has made rare diseases one of its highest priorities, launching a suite of grant programs, new offices, and initiatives specifically designed to accelerate the development and approval of therapies for patients with unmet medical needs. Initiatives include:

• Office of Orphan Products Development (OOPD) — Seven years of market exclusivity, tax credits on clinical trial costs, and waived FDA application fees for qualifying rare disease therapies.
• Rare Pediatric Disease Priority Review Vouchers — Awarded to sponsors who obtain approval for a rare pediatric disease therapy; these vouchers have been valued at approximately $100 million and provide a powerful financial incentive to develop treatments for the youngest and most vulnerable patients.
• Breakthrough Therapy & Accelerated Approval — For programs showing early evidence of substantial improvement over existing therapies, Breakthrough Therapy designation provides intensive agency guidance and rolling review. Accelerated Approval allows the use of surrogate endpoints for earlier patient access.
• Rare Disease Innovation Hub & Gene Therapy Frameworks — FDA’s Rare Disease Innovation Hub serves as a central coordinating body across FDA centers, facilitating faster, more consistent regulatory pathways for next-generation rare disease treatments.
• Patient-Focused Drug Development (PFDD) — Formally integrates the patient and caregiver voice into drug development and review, giving rare disease patients a structured mechanism to ensure FDA reviewers understand the full burden of their disease.
• Natural History Studies & Biomarker Qualification — Foundational tools that help sponsors design approvable clinical programs even when patient populations are small and traditional trial designs are impractical.

Facet works hard with rare disease developers to leverage these initiatives to accelerate efficacious and safe products to patients in need.

How Facet Supports Rare Disease Development From Day One

Facet engages at the earliest stages, helping programs build the scientific and regulatory foundation needed to successfully advance toward patients. The benefits of partnering with Facet include:

Shortening Patients’ Wait for Treatments — Nearly half of rare disease patients wait five or more years for a correct diagnosis — and many wait far longer for a treatment. Facet works with development teams to apply every available regulatory tool to compress timelines and get therapies to patients sooner.

Unlocking Accelerated Pathways for Eligible Programs — Programs such as the FDA’s Orphan Drug, Rare Pediatric Disease, and Breakthrough Therapy designations are specifically designed to advance rare disease development. We help eligible programs secure and leverage critical designations.

Navigating the Road to FDA Approval The regulatory journey from early development to an approved therapy is long and complex. Our expertise in IND, NDA, and BLA submissions means fewer delays, fewer missteps, and a clearer path to the finish line where patients are waiting.

Amplifying the Patient Voice: The FDA’s Patient-Focused Drug Development initiative gives rare disease communities a formal voice in how treatments are developed and evaluated. We help development teams incorporate patient and caregiver perspectives into regulatory strategy and study design.

Confronting the Cost of Rare Disease Development

Developing an FDA-regulated therapy for a rare disease is extraordinarily expensive — and Facet acknowledges this reality head-on. Our team works together to:

• Identify every available FDA incentive & grant program and keep promising programs strategically funded and financially viable.
• Leverage a network of dedicated parters and connect development teams to philanthropically minded organizations and service providers who help families navigate the financial hardships of this space.
  • These partners frequently offer discounted services to programs that might otherwise never reach patients.
• Keep programs moving forward by proactively identifying development efficiencies that keep products on track for the patients who need them.

Committed to Accelerating Individualized Therapy Development for N=1 Diseases

Some diseases affect not thousands, not hundreds, but a single known patient — also known as n=1 diseases. At Facet, we believe that no patient number is too small to matter.

The FDA’s February 2026 draft guidance on the Plausible Mechanism Framework represents a landmark step forward for patients with ultra-rare genetic conditions who have no other options.

Facet assists n=1 programs with identifying the appropriate regulatory pathway, preparing for early FDA interactions, designing nonclinical and safety programs, structuring the clinical investigation, and building the CMC package required to support an IND and ultimately an NDA or BLA — because in this space, there is no time to lose.