Accelerate Your Cell and Gene Therapy Development Through Regulatory Expedited Programs - PRIME, RMAT, ILAP & Co.

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MF BIOTECH supports you with your requests for PRIME, RMAT, ILAP and other expedited programs and beyond the designation.

Cell and gene therapies are at the forefront of today’s medicine. Their development is complex and requires significant investment, resources and expert knowledge over a substantial period of time. Additionally, innovative medicinal products carry inherent risks due to their novelty in multiple areas. Some of the challenges during development include setting up a manufacturing process suitable for clinical trials and commercialization, generation of non-clinical data packages taking into account the mode of action and specific nature of the product, demonstration of clinical safety and efficacy in patients, and ensuring market access and acceptable pricing.

TAILORED DEVELOPMENT STRATEGIES & CONTINUOUS REGULATORY INTERACTIONS

Regulatory agencies of all major markets have recognized that innovative therapies such as cell and gene therapies require a tailored approach for successful development. Expedited programs by regulatory agencies offer several tools to address this need. The most important benefit is continuous interaction between the authority and the applicant to align the development strategy and requirements towards clinical development and marketing authorization. Expedited programs include, for example:

• EMA: PRIME

• FDA: RMAT

• UK: ILAP

To enter an expedited program, the development candidate must meet strict criteria in terms of unmet medical need of the target disease as well as scientific evidence of its potential to address this unmet medical need. Therefore, the designation for an expedited program also acknowledges the therapeutic value of the product and increases the product value and attractiveness of the company and platform technology.

STEP-BY-STEP GUIDE TO PREPARE AND ENTER AN EXPEDITED PROGRAM

1. Select the expedited program that is right for your development program

   • Check if you meet the eligibility criteria (take note of potential gaps and challenges)

   • Consider your regulatory strategy in terms of regulatory interactions and target markets for commercialization

2. Align activities related to the expedited program with overall development program and strategy

   • Timing for preparation and submission of request as well as subsequent actions to implement expedited program

   • Resources for preparation of request as well as management of expedited program

3. Obtain preliminary feedback from Agency (if offered)

   • Validate your eligibility for the designation and obtain specific points for consideration for the request

4. Prepare full request including content and evidence to meet specific criteria

5. Be ready to answer additional questions during the assessment of your request

6. Designation granted

7. Make best use of the expedited program to accelerate your development program

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PRIME · EU RMAT · US ILAP · UK

Priority Medicines Scheme (PRIME)

AGENCY

European Medicines Agency (EMA)

PROGRAM TYPE

Designation

Requirements

AND

Major public health interest

AND

AND

Target conditions with unmet medical need

AND

AND

Demonstrate the potential to address the unmet medical need

AND

Available data supporting claim that product has potential to bring a major therapeutic advantage to patients in proposed indication, through a clinically meaningful improvement of outcomes

Benefits

+ Early appointment of a Rapporteur

+ Kick off meeting with Rapporteur and multidisciplinary group of experts from EMA / European medicines regulatory network

+ EMA dedicated contact point

+ Iterative scientific advice and expedited follow up scientific advice

+ Submission readiness meeting

+ Confirmation of potential accelerated assessment

When to Submit

+ Preliminary clinical evidence to demonstrate promising activity of the medicinal product and its potential to address to a significant extent unmet medical need (proof of concept) available

+ SMEs can submit PRIME request at earlier stage

Regenerative Medicine Advanced Therapy (RMAT)

AGENCY

US Food and Drug Administration (FDA)

PROGRAM TYPE

Designation

Requirements

AND

A drug is a regenerative medicine therapy

AND

AND

the drug is intended to treat, modify, reverse, or cure a serious condition

AND

medical needs for such disease or condition

preliminary clinical evidence

indicates that the drug has the potential to address unmet

medical needs for such disease or condition

Benefits

+ All breakthrough therapy designation features, including early interactions to discuss any potential surrogate or intermediate endpoints

+ Statute addresses potential ways to support accelerated approval and satisfy post approval requirements

When to Submit

+ With IND or after

+ Ideally, no later than the end of phase 2 Meeting

Innovative Licensing and Access Pathway (ILAP)

AGENCY

Medicines and Healthcare products Regulatory Agency (MHRA)

PROGRAM TYPE

Designation

Requirements

OR

Condition is life-threatening or seriously debilitating

OR

AND

Significant patient or public health need

AND

OR

Innovative medicine such as an advanced therapy medicinal product (ATMP) or new chemical or biological entity or novel drug device combination

OR

OR

Medicines being developed in a clinically significant new indication for an approved medicine

OR

OR

Medicines for rare disease and/or other special populations such as neonates and children, elderly and pregnant women

OR

AND

development aligning with the objectives for UK public health priorities such as the Chief Medical Officer, Department of Health and Social Care (DHSC) or Life Sciences Sector Deal (including those in Devolved Administrations, where appropriate)

AND

Medicinal product has the potential to offer benefits to patients

Benefits

+ Development of a Target Development Profile (TDP) with the support of product-specific experts

+ Definition of road map for access to patients

+ Dedicated Project Manager

When to Submit

+ Very early entry possible based on non-clinical data

+ Products with mid-development ‘global’ dossiers