The Target Development Profile Toolkit
This toolkit is for Innovation Passport holders following the Innovative Licensing and Access Pathway (ILAP) and provides activities to support the design and development of medicines.
These tools are a collection of activities and assessments to support you as you follow the ILAP. The ILAP partners are working to refine and refresh the ILAP which will be launched later in 2024. Further updates will be published on the ILAP website.
Clinical Practice Research Datalink (CPRD) Assisted Patient Recruitment
CPRD has developed an innovative service to efficiently locate patients across the UK who are potentially eligible for a clinical trial via a centralised search of the CPRD electronic health records (EHR) database, followed by GP clinical review.
Find out more about CPRD
How it works
If you want to make use of this tool you can contact the CPRD directly enquiries@cprd.com. Representatives from CPRD will advise you on the feasibility of using this approach and provide you with an outline of the process.
Subsequent discussions would be held to undertake a detailed assessment of potential patient numbers, geographical distribution of potential trial subjects, to assess the operational feasibility of using the tool and to outline estimated costs.
Delivery of the service would be undertaken with the customer or an agent with responsibility for clinical trial recruitment, and in collaboration with the sponsor.
Delivery partners
The whole process is centrally managed by CPRD. The service will be delivered directly by CPRD in collaboration with the sponsor.
Innovative and Flexible Licensing Routes
Innovative and Flexible Licensing Routes can provide expedited timelines for review, pragmatic approaches to evidence requirements and international options where appropriate and available.
Regulatory licensing flexibilities can be used to expedite the approval process once a product or new indication has sufficient data for regulatory review. Independent laboratory testing can also support authorisations based on reduced data packages through the generation, post-licensure, of data supporting batch-to-batch consistency.
The routes are:
- Accelerated assessment
- Rolling review
- Approval with conditions
- Conditional Marketing Authorisations
- Approval under exceptional circumstances
- Project Orbis
- Access Consortium
Accelerated Assessment
An accelerated assessment procedure will reach opinion on approvability of a marketing authorisation application within 150 days of submission of a valid application. This option is available for good quality marketing authorisation applications for both new and existing active substances.
Eligibility will also include those applications seeking an orphan MA approval in Great Britain and those submitted for conditional and full marketing authorisations as well as those submitted for approval under exceptional circumstances.
Rolling Review
The Rolling Review is a new route for marketing authorisation application intended to enhance development of novel medicines. It does this by offering ongoing regulatory input and feedback. The process is envisaged as a phased, modular, iterative approach to evaluation of marketing authorisation applications.
The quality, non-clinical and clinical parts may be submitted singly or in combinations depending on the individual circumstances as data becomes available. It is expected that each module will be near completion to avoid multiple iterations of assessment of the same module. Each assessment phase will progress independently permitting early identification of issues.
Each assessment cycle with points of clarification raised will offer the applicant the opportunity and time for a comprehensive update of the modules prior to final submission. The final phase will involve submission of a complete application including the remaining module together with updated versions of the modules evaluated previously.
Find out more about rolling review
Approval with Conditions
Conditions are obligations that are imposed at the time that the marketing authorisation is granted. Conditions include PASS/ PAES that can be used to provide reassurance that gaps in the evidence generation will be filled appropriately.
Conditional Marketing Authorisation
In order to meet unmet medical needs of patients and in the interest of public health, it may be possible to grant marketing authorisations on the basis of less complete data than is normally required. In such cases, the granting of a marketing authorisation is subject to certain specific obligations to be reviewed annually.
These medicines are for the treatment, the prevention or the medical diagnosis of seriously debilitating diseases or life-threatening diseases, medicinal products to be used in emergency situations, in response to public threats and medicinal products for rare diseases. Conditional marketing authorisation will allow medicines to reach patients with unmet medical needs earlier than might otherwise be the case and will ensure that additional data on a product are generated, submitted, assessed and acted upon.
Approval under exceptional circumstances
For licensing under exceptional circumstances, the applicant must demonstrate that they are unable to provide comprehensive data on the efficacy and safety under normal conditions of use. This may be because:
- the indications for which the product in question is intended are encountered so rarely that the applicant cannot reasonably be expected to provide comprehensive evidence or
- in the present state of scientific knowledge, comprehensive information cannot be provided or
- it would be contrary to generally accepted principles of medical ethics to collect such information
Under these circumstances an authorisation may be granted with a reduced clinical data package than would ordinarily be required.
Project Orbis
Project Orbis is an initiative of the Food and Drug Administration (FDA) Oncology Center of Excellence (OCE) that provides a framework for concurrent submission and review of oncology products among international partners including the UK.
Find out more about Project Orbis
Access Consortium
The Access Consortium is a collaborative initiative of medium-sized regulatory authorities between Australia’s Therapeutic Goods Administration (TGA), Health Canada (HC), Singapore’s Health Sciences Authority (HSA), the Swiss Agency for Therapeutic Products (Swissmedic) of Switzerland and MHRA. The purpose of the consortium is to build synergies and share knowledge amongst the regulatory authorities thereby enhancing the efficiency of regulatory systems.
Find out more about the Access Consortium
How it works
Regulatory licensing flexibilities can be used to expedite the approval process once a product or new indication has sufficient data for regulatory review. Independent laboratory testing can also support authorisations based on reduced data packages through the generation, post-licensure, of data supporting batch-to-batch consistency.
Early engagement with NIBSC will enable the rapid establishment of the relevant tests to make sure that, where possible, legally mandated batch testing can be performed in parallel to manufacturer’s in-house testing, ensuring no delays in access to these medicines.
You can find out more from the NIBSC website
Optimising the Market Access Approach - ILAP Access Forum
This tool aims to promote understanding of the market access approaches that will optimise the route to patient access.
Unpacking market access challenges and addressing these upfront through early engagement with system stakeholders from England, Scotland and Wales is a key enabler for achieving timely patient access.
This tool, which is delivered by the NICE Office for Market Access (OMA) in collaboration with the Scottish Medicines Consortium (SMC) and All Wales Therapeutics and Toxicology Centre (AWTTC), aims to provide a joined-up view from system stakeholders in England, Scotland and Wales on themes such as:
- Where the new technology may potentially fit in the care pathway in order to contextualise the value proposition. This is particularly important in situations where the current care pathway is complex or undefined, or in situations where the clinical trial was not conducted in the UK.
- Service delivery implications of introducing the technology into the NHS, including any commissioning considerations, testing requirements, infrastructural changes, and other implementation activities necessary to facilitate the use of the technology once it receives a positive reimbursement decision.
- High level HTA challenges. This could include helping you to navigate the different HTA routes and understanding the implications of the processes and methods for the technology. It is also an opportunity to understand how to best engage with the HTA agencies and to optimise your HTA submission strategy. (In-depth technical questions should be addressed via NICE Scientific Advice).
- Clarification on how processes will apply in practice. As the healthcare landscape continues to evolve rapidly, it can be difficult to navigate current and new processes / initiatives and what these could mean for your technology in practice.
- Commercial options and suitability of approaches to support your value proposition and minimise the financial impact on the NHS.
Engaging early in the planning process means that feedback from system stakeholders from England, Scotland and Wales can be built into your ongoing market access strategy. Acting on this feedback will help you to develop the right approaches to optimise the route to patient access.
This tool is particularly useful for products that need careful management into the system. It is most suitable for products that may involve complex HTA and commercial challenges, radical changes to an established care pathway, and significant changes to service delivery, infrastructure, training and workforce. The tool is not a mechanism for making HTA routing decisions or challenging existing decisions made by the guidance programmes.
This is a not-for-profit service - a fee is charged to cover our costs.
How it works
Market access challenges are systematically ‘unpacked’ through preparatory discussions to understand your needs and the market access insights you are looking for.
We develop 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.
We 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 NICE Advice.
Delivery partners
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.
Related tools
- Innovative and Flexible Licensing Routes
- 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.
Last updated 5 April 2024 + show all updates
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Updated information on tool kit.
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Info added about ILAP HTA tool
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First published.