Strategy & Insight

Regulatory Strategy & Insight

Clear regulatory direction shapes timelines and protects programme value. For investors, it is also one of the clearest signals that a programme is being managed well. Strategy & Insight at PrimeRA covers regulatory pathway evaluation, investment due diligence, designation strategy, platform innovation and the advisory structures that allow organisations to build a defensible position before substantive agency engagement begins. It operates independently from our delivery pillar - and informs everything that follows.

What We Do

Strategic clarity built on hands-on programme experience

At PrimeRA, we draw on decades of direct experience developing, submitting and defending advanced therapy products to translate complex regulatory questions into clear, actionable positions. Our recommendations draw on what the agencies have said, the precedents that exist, and what the programme's data and timeline can credibly support.

The scope ranges from evaluating the most efficient development pathway for a novel product to providing investors with an independent read of a programme's regulatory standing. Designation strategy is a core component: identifying which designations to pursue, when to apply, and how to extract maximum benefit from each. Where broader expert input is needed, PrimeRA can form and coordinate a Regulatory Advisory Board, selecting consultants with the specific modality or discipline expertise the programme requires. And for programmes that need a structured planning tool, PrimeRA prepares a bespoke regulatory roadmap: a working document that places every regulatory milestone in the context of data generation and approval timelines.

Pathway Strategy
Development · Designations · Global Strategy

Identifying the most efficient path from data to approval

PrimeRA evaluates all available regulatory pathways - established and emerging - to determine which development strategy best serves the programme's science, timeline and commercial objectives. Where a global development strategy is the goal, PrimeRA advises on combined EU/US approaches that protect the programme across both jurisdictions from the outset, avoiding the costly course-corrections that arise when jurisdictions are addressed sequentially. For ATMPs and other products at the frontier of regulatory science, this also requires familiarity with how agencies are evolving their frameworks in real time: platform approaches, pilot schemes, novel designation criteria and procedural innovations that may not yet be widely understood or fully utilised.

Designation strategy is a core component of this work. Orphan, PRIME, ILAP (MHRA), Fast Track, Breakthrough, RMAT and RPDD (FDA) each carry specific procedural and commercial benefits - but their value depends on timing and sequencing. PrimeRA advises on which designations are appropriate, when to apply, and how to make best use of the associated benefits: early scientific advice, rolling review, fee waivers and priority review. The output that makes this actionable is a bespoke regulatory roadmap: a working planning document placing every regulatory milestone in the context of data generation timelines and the decisions that depend on them.

Regulatory pathway evaluation - all jurisdictions, all stages
Designation strategy - Orphan, PRIME, ILAP, Fast Track, Breakthrough, RMAT, RPDD
Platform and innovative regulatory approach assessment
FDA meeting strategy - positioning, timing and question framing for Type A, B, C and D meetings
Bespoke regulatory roadmap - milestones mapped to data timelines
Regulatory Advisory Board - formation, expert selection and coordination
Investment Due Diligence
Investors · Spin-Out · ATMP

An independent regulatory read for investors and acquirers

Regulatory risk is one of the most frequently misread variables in ATMP investment decisions. PrimeRA supports investors, acquirers and technology transfer offices with independent regulatory due diligence, drawing on direct experience translating cutting-edge cell and gene therapy science into approved medicinal products. The assessment goes beyond a review of existing documents: it evaluates the credibility of the regulatory strategy, the quality and completeness of the dossier to date, the appropriateness of the chosen pathway, and the likely trajectory through to approval.

For academic or institutional programmes transitioning into commercial development - where the regulatory infrastructure is often nascent or absent - PrimeRA provides practical input on what a commercially viable regulatory strategy requires from the outset, and what must be in place before meaningful agency engagement can begin. This includes assessment of the data package, the product characterisation position and the foundational decisions that will shape the development trajectory.

Regulatory due diligence for ATMP investment and acquisition
Regulatory strategy credibility and pathway appropriateness assessment
Dossier completeness and quality review
Technology spin-out regulatory readiness input
Independent expert opinion on development trajectory and risks
Why PrimeRA

Strategy built on the programmes that set the precedents

01
Insight from the programmes that defined the field

PrimeRA's partners have worked on leading ATMP products such as: the first gene therapy approved in Europe (AAV), the first CAR-T for CD19-directed malignancies submitted for approval in Europe, the first CAR-T cell therapy submitted for approval in the US, and the first CRISPR gene editing product approved globally. The strategic positions PrimeRA takes are built on direct experience of the regulatory decisions that established these precedents.

02
At the forefront of regulatory innovation

PrimeRA has presented on platform approaches and novel regulatory frameworks at the EMA Quality Innovation Group (November 2024) and the MEB Science Day (2025), and has applied emerging pathways in client programmes. Understanding of how agency frameworks are evolving is embedded in every engagement.

03
Strategy that translates directly into execution

PrimeRA's strategy and delivery functions sit within the same organisation. When the team advising on the pathway is the same team executing it, the strategy stays anchored in what is achievable. Unrealistic positions are identified before they become costly commitments, and the move from plan to action is immediate. For clients managing investor pressure and tight development timelines, this integration is a meaningful protection.

What Is In Scope

Advice and insight - before and alongside delivery

Strategy & Insight covers the analysis, planning and advisory work that determines how a regulatory programme is structured — from initial pathway selection through to the designation and agency engagement strategy that shapes the development trajectory. Hands-on execution of the resulting submissions and procedures is delivered under the Delivery pillar.

✓ In scope
Regulatory pathway evaluation and development strategy
Designation strategy and timing - Orphan, PRIME, ILAP, Fast Track, Breakthrough, RMAT, RPDD
Platform and innovative regulatory approach assessment
FDA meeting strategy - positioning, question framing and approach (Type A, B, C and D)
Bespoke regulatory roadmap preparation
Regulatory Advisory Board formation, expert selection and coordination
Investment due diligence on ATMP programmes
Technology spin-out regulatory readiness input
→ Scoped separately
IND, CTA, IMPD, MAA and BLA authoring and submission - see Delivery
Designation application preparation and filing - see Delivery
Regulatory meeting package preparation and execution - see Delivery
Senior regulatory leadership and fractional Head of Regulatory - see Leadership
Regulatory representation with FDA, EMA and MHRA - see Representation
FAQs

Common questions

Strategy & Insight covers defined advisory engagements - pathway evaluation, due diligence, designation strategy, roadmap preparation - where the output is a structured analysis, a strategic plan or a written recommendation. Leadership covers the ongoing senior oversight function: an embedded Head of Regulatory or equivalent, directing the programme week to week and accountable for its regulatory direction on a sustained basis. The two can run concurrently, but they serve different needs and are structured accordingly.
Yes. Strategy & Insight is available as a standalone engagement. Some clients come to PrimeRA for an independent view on their development approach or to support an investment or acquisition process, without any concurrent delivery requirement. Others use it to build the strategic foundation before a period of active delivery begins. The pillar is designed to function independently in either case.
The scope is agreed at the outset based on the investor's or acquirer's specific questions. At a minimum it involves reviewing the existing regulatory strategy documentation, assessing the appropriateness of the chosen development pathway and designation position, evaluating any agency interactions to date, and providing a written independent assessment of the regulatory risks and the credibility of the path to approval. For programmes transitioning from academic or institutional settings, the engagement also addresses what foundational regulatory infrastructure needs to be established before the programme can progress commercially.
Designation strategy involves more than identifying which designations a programme may qualify for. PrimeRA advises on which to pursue first, when the data package is strongest to apply, and how to sequence interactions with agencies to build and maintain momentum. Equally important is planning to use the associated benefits - early scientific advice, rolling review eligibility, fee waivers, priority review - at the moments where they will have the greatest impact on the programme's development trajectory and commercial timeline.
PrimeRA can contribute to an existing RAB or lead its formation from scratch. Where PrimeRA leads, this includes hand-selecting consultants with the specific expertise the programme requires - whether in CMC, clinical, non-clinical or a particular modality - preparing pre-read materials that frame the questions with precision, facilitating the meeting, and consolidating the outputs into clear, actionable guidance that integrates directly into the programme's planning. The objective is advisory input that is specific, practically implementable and timed to where it will have the most impact.
Yes. An independent strategy review is one of the most common Strategy & Insight engagements. PrimeRA reviews the existing approach, identifies any gaps, inconsistencies or missed opportunities - including emerging pathways, novel designation criteria or evolving agency frameworks that may not have been fully considered - and provides a structured written assessment with clear recommendations. This is particularly valuable ahead of a significant agency interaction, a financing round or an acquisition process, where a credible and defensible regulatory position is a material consideration.

PrimeRA · Strategy & Insight

Protect your programme's development trajectory

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