MHRA – Target Development Profile Toolkit for the ILAP licensing route

Last updated: 28 Sebruary 2022

See updates at the end of the post.

The UK MHRA has recently published the Target Development Profile (TDP) Toolkit to accompany the Innovative Licensing and Access Pathway (ILAP) licensing route available since the beginning of 2021.

Who is the toolkit for?

It is for Innovation Passport holders following the ILAP and provides activities to support the design and development of medicine.

About the tools provided

The tools are a collection of activities and assessments to support you as you follow the ILAP. You may request any tool that you want to use and is relevant to your application through the Target Development Profile (TDP). The partners will consider the request in the TDP Roadmap.

What tools are available?

The following tools are available:

  1. Adaptive Inspections
  2. Certifications
  3. Continuous Benefit Risk Assessment integrating Real World Evidence
  4. Clinical Practice Research Datalink (CPRD) Assisted Patient Recruitment
  5. Enhanced patient engagement
  6. Innovative and Flexible Licensing Routes
  7. Novel methodology and innovative clinical trial design
  8. Rapid Clinical Trial Dossier Pre-Assessment

Information about the tools is provided in brief in the table below.

You can find further detailed information on the MHRA website.

NumberTool nameHow it worksDelivery partnersRelated tools
1.Adaptive inspectionsAdaptive inspections provide tailored, flexible and pragmatic supervisory and licensing inspections, specific to the Good Practice guidelines and the needs of the product as identified through the TDP. A risk-based approach to the inspection strategy will be appliedExperts from the MHRA divisions and centresThis tool can be used alongside tools 2,3,6 and 7 in this table.

The Adaptive Inspections tool can be used alongside the the following tools in this table:
2. Certifications,
3. Continuous Benefit Risk Assessment integrating Real World Evidence (RWE), 6. Innovative and Flexible Licensing Routes,
7. Novel Methodology and Innovative Clinical Trial Design tools.
2.CertificationsYou can apply to have packages of CTD data (modules 1,3, 4 and 5)assessed by an MHRA multi-disciplinary team including licensing, pharmacovigilance and inspectorate colleagues who will review the submission against marketing authorisation requirements and expectations for regulatory compliance.As aboveThe Certification tool could be used alongside rolling review in the the following tools:
6. Innovative and Flexible Licensing tool and
1. Adaptive Inspections tool.

3.Continuous Benefit Risk Assessment integrating Real World EvidenceProducts most suitable for the tool would typically be those where:
i) Post-Authorisation Safety (or Efficacy) Study (PASS/ PAES) is likely to be desirable or mandated 
ii) medicines granted through a conditional marketing authorisation or authorisation under exceptional circumstances, iii) where an appropriate control group may be lacking or where there may be other uncertainties about the generalisability of the clinical data to real world use in the UK.

Key outputs from the tool could include:
-advice to further refine the TDP roadmap and support development of the RMP, focussing on plans for post-approval evidence requirements, including RWE.
-provisional agreement of study protocols, in full or as synopses
MHRA, Health Technology Assessment and NHS bodies will be important partners in influencing plans for further studies.The tool could operate independently of other tools but could work with the following tools:
Innovative Licensing Routes
4. Clinical Practice Research Datalink (CPRD) Assisted Patient Recruitment
1. Adaptive Inspections
5. Enhanced Patient Engagement
4Clinical Practice Research Datalink (CPRD) Assisted Patient RecruitmentTo make use of this tool ,you can indicate that in the TDP. Representatives from CPRD will advise you on the feasibility of using this approach and provide you with an outline of the process.The whole process is centrally managed by CPRD. The service will be delivered directly by CPRD in collaboration with the sponsor.The CPRD Assisted Patient Recruitment tool could be used with the tool 3. Continuous Benefit Risk Assessment integrating RWE tool to manage any longer term follow up of enrolled patients.
5.Enhanced patient engagementMHRA will ask if you have already engaged with patients in developing the product and explore what additional approaches you have identified for patient engagement. It will also seek your views on what you would like to gain from new or further patient involvement.

Post-product authorisation, you will be given the opportunity to access support and advice on patient information and evaluations including real world data, and advice on how post-market surveillance can best meet the needs of patients.
This tool is delivered by the MHRA, NICE, the Scottish Medicines Consortium, and potentially in collaboration with other organisations such as the Health Research Authority and NHS England and NHS ImprovementThis tool can be used alongside the following tools:
3 Continuous Benefit: Risk Assessment integrating Real-World Evidence.
4. CPRD assisted patient recruitment,
6. Innovative and flexible licensing routes, 
7. Novel methodology and Innovative Clinical Trial Design
6.Innovative and Flexible Licensing RoutesThe TDP will recommend the most suitable routes to market based on the product and patient population to treat in the context of the ongoing development programme.This tool can be used with NICE, SMC and other system partners such that the impacts of licensing routes on Health Technology Assessment and commissioning, including timeliness of patient access and the potential need for managed access arrangements, can be prospectively considered.Tool 2 Certifications is relevant to Rolling review, as is tTool 3 Continuous benefit risk assessment tool for approval with conditions.
7.Novel methodology and innovative clinical trial designAccording to the individual requirements of the applicant, there are several key outputs from the tool and include:
-securing multi-stakeholder agreement for novel and innovative approaches to evidence generation.
-qualification of the new methodologies and support via (joint) scientific advice procedures (e.g. with NICE) offered during the ILAP, including targeted inspections of novel technologies.
This tool can be used with partners such as MHRA, NICE and the SMC so that implications of novel trial designs for Health Technology Assessment, can be prospectively addressed. The tools can also be used with other health research system partners such as the Health Research Authority (HRA) and the National Institute of Health Research (NIHR) to provide reassurance that innovative clinical trial proposals are deliverable.The tool is wide ranging and can be used with a number of different tools in the pathway such as the the following:

1. Adaptive Inspections tool
4. CPRD Assisted Patient Recruitment tool
8. Rapid Clinical Trial Dossier Pre-Assessment tool
8.Rapid Clinical Trial Dossier Pre-AssessmentYou can submit draft clinical, quality and/or non-clinical documentation for pre-assessment. MHRA will evaluate the data and documentation, provide rapid feedback and identify any potential issues to be rectified prior to formal submission. An informal scientific advice meeting will be held with the sponsor so that any questions or required clarifications can be addressed swiftly.

An iterative approach can be taken, and for the tool to maximise its benefits, it is essential that trial sponsors also respond to any feedback in an expedited manner.
The tool will be delivered by the MHRA Clinical Trials Unit. Where appropriate additional input may be obtained from the HRA to reduce duplication of review of common documentation.The tool may have interdependencies with the following tool:
7. Novel methodology and Innovative Clinical Trial Design tool.
9. Optimising the Market Access Approach – ILAP HTA Access ToolMarket access challenges are systematically ‘unpacked’ through preparatory discussions to understand your needs and the market access insights you are looking for.

MHRA develops the scope of the meeting and work with you to finalise the structure, content, attendance, and logistics.

NICE, SMC, & AWTTC identify and bring together the most appropriate people in the healthcare systems in England, Scotland and Wales for you to engage with to gain insight for your ongoing market access planning.

Engagement meetings are facilitated and conducted in a safe harbour environment. This ensures confidential, free-flowing, peer-to-peer discussions, which can act as the basis for ongoing engagement with system stakeholders.

You will benefit from signed confidentiality agreements (for people not already covered by their system contracts), sharing of relevant declarations of interests for those involved, and assurance that discussions are non-attributable, non-binding and non-transmissible.

MHRA will agree the fee with you and the stakeholders. To make invoicing and payment as easy as possible, you pay the fee to NICE OMA and we make sure that all stakeholders are paid.

This tool is not part of formal HTA guidance processes. It provides expert feedback to support development of the optimal market access approach.

Read more information on Safe harbour engagement meetings.
If you use this tool you will work with Health Technology Assessment bodies, NHS representatives, and clinical experts (where appropriate) from England, Scotland and Wales.This tool can be used with the following tools: 3. Continuous Benefit Risk Assessment integrating Real World Evidence
6. Innovative and Flexible Licensing Routes
7. Novel methodology and innovative clinical trial design

The need for detailed scientific advice, can be prospectively considered (not-for-profit fees will be apply). Read more information on HTA/joint scientific advice.
Where can you find more information about each of these tools?

More detailed information is available on each of the tools, on the MHRA website here.

28 Feb 2022The table has been updated to add information about tool 9. Optimising the Market Access Approach – ILAP HTA Access Tool in line with informatio on the MHRA website. Additionally, last column of the table has been updated to make it easier to read.