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Abstract
Sotorasib was the first health technology from the UK’s ILAP scheme to be approved by NICE.
April 2022
BACKGROUND
Launched by the UK MHRA in January 2021 with the aim to accelerate the marketing of innovative medicines, ILAP works in conjunction with the ‘innovation passport’ medicine designation of the MHRA and integrates the role of several partner health agencies in the UK, including the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC), the All Wales Medicines Strategy Group (AWMSG), and NHS England and NHS Improvement (NHSE&I).
ILAP allows early dialogue on evidence requirements across regulatory and HTA, enhanced coordination and close alignment of evaluation and access pathways in the UK, and expedited regulatory routes. New chemical entities, biological medicines, new indications and repurposed medicines all fall under the scope of ILAP. The MHRA Innovation Passport fee is £3,624, the Initial Target Development Profile (TDP) fee is £4,451, and additional fees for the NICE Office of Market Access and Scientific Advice Services may apply.
As of December 2021, the MHRA had awarded a total of 41 innovation passport drug designations.
THE 4 KEY FEATURES OF ILAP
INNOVATION PASSPORT
New medicine designation from MHRA linking directly access pathways
TARGET DEVELOPMENT PROFILE (TDP)
Identify and plan for key regulatory and evidence generation steps
ILAP TOOLKIT
Identify and plan for key regulatory and evidence generation steps
INTEGRATED PATHWAY
mHRA, NICE, SMC, AWMSG, and NHSE&I, all working in coordination
CRITERIA FOR INNOVATION PASSPORT DESIGNATION FROM MHRA
| CRITERIA 1
Condition is life-threatening or seriously debilitating There is a significant patient or public health need |
Expected submissions
Summary of the life threatening or seriously debilitating condition Symptoms, life span and quality of life aspects Current treatment landscape Clearly defined evidence of a specific need Magnitude of the issue(s) along with the identified gaps in the current treatment landscape |
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| CRITERIA 2
Medicine fulfills one or more of the following: innovative medicine such or new chemical o biological entity or novel drug device combination Medicines being developed in a clinically significant new indication for an approved medicine Medicines for rare disease and/or other spe populations such as neonates and children, elderly and pregnant women development aligning with the objectives for public health priorities |
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Expected submissions
Depending on type of medicine, the following are needed: Full regulatory description of product Description of new indication Description special target population Description of how/where public health priorities are being fulfilled |
| CRITERIA 3
The medicinal product has the potential to offer benefits to patient |
Expected submissions
Available clinical data in a relevant population of patients can be provided Views from patients or patient organizations if possible |
A single positive MHRA Innovation Passport can cover multiple indications for the same medicine.
THE TARGET DEVELOPMENT PROFILE TOOLKIT (TDP)
Adaptive Inspections: Flexible, live phase preclinical/clinical inspection, defined pre/post authorization inspections.
Certifications: Once defined in the original TDP application, the certification tool provides enhanced official regulatory review of packages of Common Technical Document (CTD) and also allows early engagements to set up batch testing facilities for biological medicines, vaccines and blood products.
Continuous benefit risk assessments integrating real world evidence (RWE): Proactive data collection for post authorization safety/efficacy study execution and scientific advice on study protocols. The tool seeks to increase the utility of RWE to support the benefit-risk profile.
Clinical Practice Research Datalink (CPRD) Assisted Patient Recruitment and
enhanced patient engagement: The UK CPRD’s patient recruitment tool will enable easy identification and recruitment of patients for clinical trials. The patient engagement tool will offer patient engagement facilitation across a range of diseases.
Flexible and innovative licensing routes: Several potential routes are available including accelerated assessments (within 150 days of application), rolling reviews, conditional marketing authorizations and approvals with conditions/under exceptional circumstances when comprehensive data cannot be provided with normal conditions of use. This also includes activities of project Orbis, a collaboration with HTA agencies from US, Australia, Singapore, Switzerland and Brazil allows concurrent submissions and review of oncology products.
Novel clinical trial design and HTA access tools: MHRA will support innovative trial designs and facilitate acceptability of methodology with stakeholders. The HTA access tool will aid early planning of care pathways, service delivery systems in the NHS and confirm suitability of support drug value propositions.
ASSESSMENT OF SOTORASIB
LUMYKRAS (sotorasib) is the first health technology to come through the ILAP and receive a positive recommendation from NICE.
It is the first available treatment for patients with KRASG12C mutated NSCLC. It was the first investigational KRASG12C inhibitor with a novel mechanism of action that successfully progressed to clinical trial stage. Prior to sotorasib, the KRAS oncoprotein was considered impossible to target with drugs. Prior to authorization of sotorasib, there were no recommended treatment options for KRASG12C mutated mNSCLC.
In the UK, the product is indicated as monotherapy for treatment of adults with KRASG12C-mutated NSCLC who have progressed on, or are intolerant of, platinum-based chemotherapy and / or anti-PD-1/PD-L1 immunotherapy.
The NICE appraisal committee reviewed all submitted data and concluded that sotorasib may fulfill its end-of-life criteria, however, additional data collection will be needed to confirm this. NICE therefore recommended sorotasib within the Cancer Drugs Fund (CDF) as part of a managed access agreement.
IMPACT OF THE ILAP SCHEME
The ILAP scheme has proven to be much more popular than originally expected at the time of its launch in 2020. Oncology product applications have been the most frequent for the ILAP scheme and a majority of applications are from products with early stage data on efficacy and safety.
The MHRA innovation passport along with the TDP toolkit provide unique opportunities for early stage engagement with regulatory authorities and healthcare service providers, a feature that is currently not available for routine MHRA applications.
CONSIDERATIONS FOR FUTURE ILAP APPLICATIONS
Consider carefully, at an early stage, if your product may fulfill eligibility criteria for an MHRA innovation passport.
Early stage planning and engagement with MHRA partner organizations through the ILAP scheme could facilitate time-efficient regulatory approvals, successful pricing and reimbursement discussions making way for smoother access pathways for your product.
Remember that the ILAP does not replace the already established, early access to medicines scheme (EAMS) and promising innovative medicine (PIM) designations. The EAMS pathway may still be a viable option for products at late stages of development for high unmet need indications.
