Integrated non-clinical, regulatory and market access strategy
By addressing the unique scientific, regulatory and economic challenges of ATMPs, we help therapy developers to shape commercially viable therapies from preclinical development through to market access.
Our multidisciplinary team provides expert guidance to de-risk and accelerate translational pathways, meet regulatory expectations, and build compelling and robust development strategies that support clinical progression, reimbursement, and adoption.
Non-clinical strategy
Developing an ATMP from the pre-clinical stage to first-in-human (FIH) trials is complex and requires a robust, well-planned non-clinical strategy. We work with academics, start-ups, and therapy developers to design bespoke approaches that enable effective translation through the non-clinical phases.
With experience in all aspects of non-clinical studies, from designing proof-of-concept (POC) and efficacy studies, through to FIH supporting pivotal safety good laboratory practice (GLP) programmes, our team employ expert knowledge of the contract research organisation (CRO) landscape specialising in ATMPs and a comprehensive understanding of the ATMP development process to rapidly progress your therapy to the clinic and beyond.
We can help you to address the in silico, in vitro, and in vivo scientific and regulatory evidence needed to streamline ATMP development in an ethical, compliant, and efficient manner.
Benefit from our expertise in:
Non-clinical strategy design
- Creating customised “route to clinic” and “route to market” plans for your ATMP
- Structuring of in vitro approaches including advice on vector design
- Design of POC, mode-of-action (MOA), and efficacy studies
- Safety study design, management and support with implementation and monitoring of the studies
- Due diligence of non-clinical programmes for out/in-licencing
Laboratory study design
- Pivotal GLP study design along with CRO engagement to ensure that your strategy is expertly translated to support future Clinical Trial Authorisation (CTA) filing
Risk assessment and gap analysis
- Risk assessment of your candidate ATMP in the selected clinical indication
- Gap analysis of your POC and safety data
Regulatory support and compliance
- Expert support in the preparation of the non-clinical sections of regulatory documents, such as Briefing Document, Investigator’s Brochure, and Investigational Medicinal Product Dossier
- Reporting of non-clinical studies to worldwide regulatory standards
- Combined ATMP strategies (all safety aspects including International Organisation for Standardisation (ISO) compliant medical device strategies)
Regulatory affairs
Advanced therapy medicinal products (ATMPs) face unique regulatory challenges. We support ATMP developers to address specific risks in their product development and satisfy the relevant national and international legislation and guidance.
We can help you to navigate the UK, EU and international regulatory landscape. Our experienced team can advise on the latest applicable regulations, write or review your applications and help you get the most out of key interactions and meetings with national and international agencies.
Benefit from our expertise in:
Development support
- Advice on your national or global regulatory strategy
- Supporting early interactions with regulatory agencies
- CMC gap analysis
Regulatory procedure
- Assisting with product ATMP classification
- Priority medicines (PRIME)
- Regenerative medicine advanced therapy designation (RMAT)
- Raw material evaluation
- Designation application (e.g., orphan drug designation, rare paediatric designation)
- Paediatric plan application
- Starting material procurement support
- Marketing authorisation/licence application including individual documents (e.g., environmental risk assessment)
Clinical trials
- Provide recommendations for a path to CTA and Investigational New Drug (IND) application, as well as Marketing Authorisation and Biologic License Application (MAA/BLA)
- Investigational medicinal product dossier (IMPD)/Module 3 authoring, including conversions clinical trial application (CTA/IND), maintenance and interaction with regulatory agencies
- CTA authoring (IMPD, IB, Protocol etc)
- Genetically modified organism (GMO) application, maintenance and interactions with environmental agencies
- National GMO manual authoring
- Access to our clinical network of ATMP experts
Innovative Licensing and Access Pathway (ILAP)
- Integrated input from our experienced team to support you with the ILAP process and maximise derived benefits (read our guide here)
Health economics and market access (HEMA)
ATMPs face high adoption barriers and commercial risk due to limited long-term clinical data at launch, high manufacturing costs requiring premium reimbursement, and the resource-intensive demands of healthcare system readiness.
We help mitigate these challenges by guiding early development with commercially viable Target Product Profiles, building tailored health-economic strategies and models, engaging key market access stakeholders, and addressing data, affordability, and NHS readiness to maximise the chance of success at launch.
Benefit from our experience in:
Shaping early development (pre-clinical stage)
- Define parameters of commercial viability in terms of therapeutic positioning and thresholds of product performance and manufacturing costs
- Inform clinical manufacturing strategy accordingly
Opportunity optimisation (pre-pivotal clinical trial stage)
- Health economic analyses and key market access stakeholder engagement
- Defining the interrelationship between value story, reimbursed price potential and corresponding evidence requirements
- Inform the evidence generation plan accordingly and support to develop a target product profile (TPP) (read our guide here)
Pre-launch preparations
- Address data uncertainty, healthcare system affordability, infrastructure and treatment pathway constraints
- Leverage Innovative reimbursement mechanisms and post-launch real world evidence collection
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