GovWire

Applying for a Promising Innovative Medicine (PIM) designation

Guidance

A Promising Innovative Medicine (PIM) Designation is an early indication that a medicinal product is a candidate for the Early Access to Medicines Scheme (EAMS), intended for the treatment, diagnosis or prevention of a life-threatening or seriously debilitating condition, with the potential to address an unmet medical need. The designation will be issued after an MHRA scientific designation meeting on the basis of non-clinical and clinical data available on the product, in a defined disease area. Following the PIM designation, the applicant completes the clinical development programme and continues with an application under the EAMS Scientific Opinion (SO) assessment step see later guidance. A PIM designation is a prerequisite to enter the EAMS SO step.

When to apply for a PIM Designation

Applicants are encouraged to apply when data from early stages in a clinical development programme indicate that the medicinal product fulfils the designation criteria, namely the product is likely to demonstrate significant benefit for patients in life-threatening or seriously debilitating conditions. This is because early regulatory and other stakeholder engagement is generally recommended to maximise the benefits within the scheme.

Applicants may also apply towards the end of their clinical development programme and could consider applying for a PIM designation and the EAMS SO pre-submission meeting (PSM) simultaneously see later guidance.

The PIM designation criteria

All three criteria, which must be fulfilled in order to gain a PIM designation, are:

Criterion 1: There is a life threatening or seriously debilitating condition and a high unmet need

The severity of the disease should be justified based on objective and quantifiable medical or epidemiologic information, in terms of mortality and morbidity, with special emphasis on patient quality of life. For high unmet need, the Applicant should consider if there is no method of treatment, diagnosis or prevention available in the UK for the condition, or if methods already in use in the UK have serious limitations. From a regulatory perspective, methods already in use include medicinal products that have a GB or UK marketing authorisation as well as standard of care and non-pharmacological therapies. The health technology assessment, commissioning or supply status of a product is not taken into account by the MHRA. A critical review of the methods already in use for treatment, diagnosis or prevention in the UK should be provided, including an evaluation of the performance of these methods based on quantifiable data (e.g. data on survival, disease progression/relapses, or patient-reported outcomes). The Applicant should provide a justification for why methods already in use are not adequate.

Criterion 2: The medicinal product is likely to offer a major advantage over methods of preventing, diagnosing or treating the condition already in use in the United Kingdom.

The Applicant should submit preliminary evidence, based on clinical data, indicating that the advantage and magnitude of effect claimed for the product is predicted to be of significant relevance to the patients and will address their unmet need. The major advantage could be improved efficacy, or similar efficacy but better overall tolerability, compared to methods already in use. A well-argued evaluation of the likelihood of achievement of the products claims should be provided, based on the totality of information available at the time of designation. This should include direct or indirect comparison to methods already in use. From a regulatory perspective, methods already in use include medicinal products that have a GB or UK marketing authorisation as well as standard of care and non-pharmacological therapies. The health technology assessment, commissioning or supply status of a product is not taken into account by the MHRA.

Criterion 3: The potential adverse effects of the medicinal product are likely to be outweighed by the potential benefits, allowing for a reasonable expectation of a positive risk-benefit balance

The potential adverse effects of the medicinal product are likely to be outweighed by the benefits, allowing for the reasonable expectation of a positive benefit/risk balance. A positive benefit/risk balance should be based on preliminary scientific evidence, as justified by the applicant, that the safety profile of the medicinal product is likely to be manageable and acceptable in relation to the estimated benefits.

The wording of the PIM condition in the designation application

The applicant is encouraged to seek the broadest possible indication in a defined condition for the PIM designation. This would allow for future sequential EAMS SO indication submission(s), where appropriate (e.g., different line setting, treatment combination), without the need to seek a new PIM designation. The final wording of the PIM indication will be agreed at the PIM designation meeting.

Joint PIM designation/ pre-submission meeting

For the joint PIM designation/PSM applications, both a PIM designation application template and a PSM template should be submitted at the time of the request. You will be invited to a single meeting where both the PIM criteria and the aspects of an EAMS SO application will be addressed. You do not need to replicate information provided in the PIM in the PSM request form (you may cross refer from your PSM form to the PIM application for relevant information). See also section 2.

How to apply

Companies seeking a PIM designation should complete the PIM designation template in full, indicating how the product fulfils the criteria of designation.

Completed forms should be sent by email to the MHRAs EAMS coordinator (eams@mhra.gov.uk).

PIM designation application meeting and decision process

Your submitted designation application will be reviewed by the assessment team and an agreed designation meeting date will be set (normally within 6 weeks of the receipt of your request). The focus of the designation meeting is restricted to the potential of the medicinal product to fulfil the three criteria and is expected to last for up to one hour. You should prepare a PowerPoint presentation (maximum of 20 minutes length), covering your position. Please provide the presentation to the EAMS coordinator at least 5 working days in advance of the meeting. Following the designation meeting, the assessment team will make a recommendation to the internal scientific consistency review group, who will determine whether to grant a designation or not (usually within 4weeks).

If you are awarded a designation, you will receive a positive designation letter, which will include your unique EAMS number. This number should be included with any future application for an EAMS SO step. You will be encouraged to utilise the 