How OneGlobal Trinity M Consulting Supports Orphan Drug Development

Over 30 million Americans are affected by rare diseases—medical conditions that impact fewer than 200,000 individuals nationwide. Yet, the development of innovative treatments for these conditions has long lagged behind more common diseases. One of the primary barriers has been the high cost and financial risk associated with developing therapies for such small patient populations. In response, the U.S. government enacted the Orphan Drug Act in 1983 to incentivize pharmaceutical companies to invest in these high-need areas.

At OneGlobal Trinity M Consulting, we partner with pharmaceutical, biotech, and medical device companies to navigate the unique regulatory, clinical, and strategic pathways required for successful orphan drug development and approval. From pre-submission planning to regulatory strategy and lifecycle management, we bring the expertise needed to maximize success in this challenging yet critical therapeutic space.

Understanding the Orphan Drug Act

The Orphan Drug Act provides targeted incentives to promote the research and development of drugs, biologics, devices, and medical foods intended for rare conditions. A disease qualifies as "rare" when it affects fewer than 200,000 individuals in the U.S., or when the costs of developing and marketing a treatment are unlikely to be recovered through sales.

Since the Act’s inception, over 4,500 orphan designations have been granted, leading to the approval of more than 730 unique therapies. To put this into context, fewer than 10 orphan drugs were brought to market between 1973 and 1983. This growth represents a vital shift in industry focus toward previously neglected patient populations.

How Trinity M Consulting Helps: We guide sponsors through every stage of the orphan drug designation process, from drafting FDA submissions to building strategic development plans that meet regulatory requirements and accelerate approval timelines.

Incentives for Sponsors of Orphan Products

To encourage development, the FDA offers several valuable incentives for companies pursuing orphan drug designation, including:

• 7-year market exclusivity for approved orphan products

• Federal tax credits covering up to 25% of qualified U.S.-based clinical research expenses

• Waiver of PDUFA fees, valued at approximately $2.9 million (2021 figures)

• Access to federal research grants from the Office of Orphan Products Development (OOPD)

• Tailored regulatory guidance throughout the development lifecycle

Our Approach:Our consultants specialize in optimizing these benefits. We ensure that our clients leverage every available incentive—especially tax credits, grants, and exclusivity—to create a commercially viable pathway for their orphan drug development programs.

Qualifying for Orphan Drug Designation

A sponsor may apply for designation for an unapproved drug or for a new orphan indication of an existing product. While multiple sponsors can seek designation for the same rare disease, each must submit a distinct and fully compliant application. Notably, even a drug similar to an already approved orphan product may be granted designation if it can be shown to offer clinical superiority.

Our Approach: Trinity M Consulting provides full regulatory support, including eligibility assessments, strategic differentiation for "clinically superior" claims, and preparation of robust designation applications that withstand FDA scrutiny.

Navigating the Orphan Drug Designation Application Process

The application process is initiated using FDA Form 4035. The FDA typically completes its review within 90 days. Required components include:

• Sponsor and drug product details

• A well-documented description of the rare condition and unmet needs

• Scientific rationale for the drug's use

• A comprehensive summary of the regulatory and marketing history

• Documentation supporting the rarity of the condition

Our Approach: We develop scientifically sound, well-researched applications tailored to meet FDA expectations. Our regulatory professionals assist with data interpretation, prevalence justification, and thorough documentation to ensure submissions are complete and compelling.

Grants and Support Programs for Orphan Products

The OOPD offers funding through two key grant mechanisms:

1. Clinical Trial Grants Program – Supports studies that assess the safety and effectiveness of potential orphan products.

2. Natural History Studies Program – Funds research into the course and progression of rare diseases to inform development strategies.

Why Partner with Trinity M Consulting? We help sponsors identify the right funding opportunities and prepare competitive grant proposals. Our clinical and regulatory teams work together to design studies that meet FDA expectations while maximizing the chance of funding success.

Strategic Insights for a Successful Designation Request

Two areas of focus consistently stand out in orphan drug applications:

• Scientific rationale – Must be clearly presented, well-supported by data, and backed by literature

• Prevalence analysis – Requires systematic review of epidemiological data and transparent methodologies

The FDA also evaluates whether a subset of a broader non-rare disease qualifies as an “orphan subset,” which involves strict criteria around scientific plausibility and treatment applicability.

Strategic Consulting Guidances Includes:

• Prevalence analysis and justification frameworks

• Orphan subset assessments

• Structured scientific rationales aligned with FDA expectations

• Development of adaptive, model-based, and umbrella trial designs for clinical studies

Recommendations for Early Engagement and Expedited Development

The FDA encourages early interaction to align on innovative study designs and designation strategy. Early submission of orphan drug designation requests unlocks key benefits, including early access to tax credits and expedited regulatory pathways.

Trinity M Consulting Delivers: We support early-stage planning and pre-submission meetings with the FDA to de-risk development and create an efficient roadmap toward orphan drug approval.

Conclusion

The Orphan Drug Designation Program presents a significant opportunity for life sciences companies to bring life-changing therapies to underserved populations—while accessing substantial incentives that make rare disease drug development financially feasible.

At OneGlobal Trinity M Consulting, we specialize in helping companies navigate the complexities of orphan drug development with confidence. From strategic planning and regulatory submissions to FDA engagement and grant application support, we offer full-spectrum consulting services that drive innovation in rare disease treatment.

Ready to advance your orphan drug program? Partner with OneGlobal Trinity M Consulting and let our expertise lead the way. Contact us today to learn more about our regulatory services.