Current figures (as of May 2017) show that only 23% of PRIME applications are successful. Furthermore, nearly 5% of all applications are considered to be out of scope and are therefore not even assessed. Below, we outline some top tips for preparing for a PRIME submission that hopefully will assist applicants to realise a successful application.
Firstly, ensure your application will be in scope
Although the reasons for the out of scope rejections are not published, applications that do not meet the criteria for initial assessment for eligibility to the PRIME scheme would likely have the following deficiencies:
- Incorrect stage of development for PRIME entirely (e.g. product already in the pre-submission phase of an MAA)
- Incorrect stage of development for the type of company i.e. only proof of principle data submitted by a big pharma company
- Medical device – currently PRIME is only for medicines
- Veterinary product – currently PRIME is only for human medicines
- Generic products
- New indications for existing products
These would result in the application being rejected as out of scope.
You should be submitting for PRIME as soon as you finish the relevant stage of development and ideally before you apply for scientific advice. Of course the risk of not being accepted for PRIME means that you should not unduly delay your scientific advice request, but since the PRIME application is a maximum of 30 pages and therefore is significantly smaller and less labour intensive than a scientific advice briefing package, it makes sense to submit this first.
If you are an SME and you intend to submit at the earlier entry point to PRIME, you will first need to ensure that you have registered with the EMA as an SME. It can take a while to collate all the information needed for such registration, so this should be done as early as possible and certainly before the PRIME application begins to be authored.
If you are an academic, you should contact the EMA in advance of planning your submission to ensure that you meet their criteria.
Plan your submission according to the deadlines published on the EMA website – if you miss the deadline your application will not be reviewed for at least another month, sometimes two months if you miss a summer or end of year deadline.
Lastly, ensure you have a Eudralink account set up so you can actually physically submit your application!
Use the templates and guidance provided
This may seem like an obvious one, but according to the EMA’s own comment on out of scope applications, submissions are rejected if they are submitted “with a format and content inadequate to support their review”. There is a specific template that is very useful available on the EMA’s website, along with a guidance document and a mailbox email@example.com that can assist if you have questions about your application (although do not expect them to provide any form of validation in terms of the data you intend to submit!).
The guidance states that your justification template should be no more than 30 pages long – be sure that you stick to this (additional documents submitted in support of the application do not count in this page limit).
Get your content right
In terms of content, per the Guidance for Applicants, your application needs to contain the following data to support your application:
- availability of preliminary clinical evidence in patients to demonstrate the promising activity of the medicinal product and its potential to significantly address an unmet medical need (proof of concept).
In exceptional cases, applicants from the academic sector and micro-, small-and medium-sized enterprises (SME):
- compelling nonclinical data in a relevant model provide early evidence of promising activity (proof of principle) and
- first in man studies indicate adequate exposure for the desired pharmacotherapeutic effects and tolerability.
You should complete all sections of the justification document template. Your application should cover the current unmet medical need using supportive literature, assess the issues associated with current available therapies (if they exist) and identify where your product can fill that gap. You should summarise your currently available non-clinical and clinical data, in particular ensuring that its efficacy and any critical aspects that enable it to fill the unmet medical need are at the forefront of the discussion.
You can submit supportive documentation (such as your IB, CSR synopses for previous studies, literature references etc.) which you can refer to throughout your justification document, however your justification document should stand alone in presenting the relevant information for the assessment.
Your product is eligible for PRIME if it has the potential to fulfil a high unmet medical need that is of major public health interest – this is an exciting product! This should come across in your application. Of course your application will not be rejected because you don’t sound excited enough, but you should be presenting your data in a way that highlights its potential to meet the criteria for PRIME.