Building Smarter Evidence: How One ATMP Company Future, Proofed Its Market Access Strategy

Advanced Therapy Medicinal Products (ATMPs) are reshaping medicine, but bringing them from approval to long, term commercial success is no small feat. Securing sustainable market access, navigating post, marketing commitments, and scaling globally require more than breakthrough science, they demand a disciplined approach to evidence generation.

This case study explores how one ATMP company transformed a fragmented evidence landscape into a unified, strategic roadmap that not only supported post, marketing commitments but also positioned the product for long, term global expansion.

The Challenge

Despite regulatory approvals in major markets, the client faced mounting hurdles:

• Fragmented evidence planning: Investigator, initiated trials (IITs), registries, and follow, up studies were scattered, duplicative, and lacked alignment with regulators, HTA bodies, and payers.

• High, cost, low, clarity programs: Significant resources were tied up in initiatives with uncertain value for access or reimbursement.

• Need for real, world and long, term data: Pivotal studies had secured approval, but not the kind of RWE, registry insights, or economic modeling needed to prove durability, safety, and cost, effectiveness.

• Global expansion risks: Without a proactive plan, diverse evidence requirements across the EEA and rest of the world could delay launches and limit uptake.

The company needed a unifying evidence generation plan, one that would secure post, marketing success, guide renegotiations, and scale across geographies.

The Approach

The project unfolded in three key steps:

1. Internal Review

All existing documentation was synthesized and benchmarked against competitor strategies. This allowed the team to pinpoint gaps, overlaps, and inefficiencies across the evidence base.

2. Core Team Workshop

A focused session with the client’s internal leadership team identified priorities, mapped existing efforts, and set the structure for an Integrated Evidence Generation Plan (IEGP).

3. Cross, Functional Workshop
A four, hour interactive workshop brought together HEOR, Market Access, Medical, Regulatory, Clinical, and Commercial teams. Activities included:

• Ranking evidence needs by urgency.

• Exploring long, term study designs, IIT optimization, newborn screening initiatives, and diagnostic strategies.

• Aligning on global expansion requirements.

The outcome was a strategic roadmap, a consolidated plan with clear study mapping, prioritized recommendations, timelines, and governance.

The Impact

The results went beyond filling evidence gaps, they reshaped the company’s entire approach to post, marketing strategy:

• Cross, functional alignment: The IEGP became a single source of truth, reducing silos and duplication.

• Smarter investments: Evidence initiatives were streamlined, with resources redirected toward high, value activities.

• Market access readiness: HTA submissions and payer negotiations were strengthened through robust RWE, long, term follow, up data, and economic modeling.

• Better governance: A new decision, making structure, backed by KOL input, improved agility and transparency.

• Scalable success: The IEGP was designed as a repeatable model, one that can be applied to future ATMP launches across global markets.

Why It Matters

For ATMPs, regulatory approval is only the first step. Long, term value demonstration is the real determinant of commercial success. This case illustrates how a structured, integrated evidence strategy can help companies:

• Translate early approvals into sustainable market access.

• Meet diverse regulatory and payer demands across geographies.

• Scale launches without being slowed down by fragmented or duplicative efforts.

By treating evidence generation as a living, strategic asset, ATMP companies can move beyond survival mode, and build a roadmap for global, lasting impact.

If you’re working with ATMPs or other complex therapies, the key takeaway is clear: don’t let evidence evolve in silos. Build an integrated, future, ready plan that balances science, economics, and access.