What it is, why it matters, how you can apply and how we can help.
What is the PRIME Scheme?
You might be forgiven if you don’t know what the PRIority MEdicines (PRIME) scheme is; but if you are in the business of medicine development it wouldn’t hurt to learn about it.
PRIME is a relatively new scheme launched by the European Medicines Agency (EMA) in 2016. It aims to support the development of novel medicines that target an unmet clinical need in conditions that are seriously debilitating or life-threatening.
It is an entirely voluntary scheme that offers enhanced support to medicine developers to optimise the development of a medicine and expedite the achievement of a marketing authorisation in the EU. Essentially, PRIME aims to get new treatments for serious conditions to patients faster – an aim that no doubt you share.
Why does PRIME designation matter?
Any regulatory specialist worth their salt is going to tell you that the earlier you open a dialogue with the regulatory authorities, the better. Regulator feedback is invaluable at all stages of the drug development process and reduces uncertainty when the time comes to apply for your marketing authorisation.
PRIME gives medicine developers a chance to open communication with the EMA’s Committee for Medicinal Products for Human Use (CHMP), or the Committee on Advanced Therapies (CAT) early in development.
This can optimise clinical development plans and speed up the process of getting to the point of a conditional or full marketing authorisation approval, saving time and money along the way.
5 Benefits of PRIME
Achieving PRIME designation for your novel drug has numerous benefits, and ProPharma can help you from the initial PRIME application through to the submission and approval of your MAA, supporting you with each of the following activities.
- Fee waiver incentives for SMEs and applicants in academia
If you hail from academia or a small-medium enterprise (SMEs), you’ll likely be aware that SMEs already benefit from fee waivers and/or deferrals on EMA services (scientific advice, MAAs, etc), however achieving PRIME status can further reduce these fees.
You could receive a 100% fee reduction for any scientific advice related to your development program; considering EMA scientific advice fees currently range from €46,900 to €94,0001, this is also a substantial benefit. PPG can support you in obtaining SME status to take advantage of these fee reductions and waivers.
- Dedicated CHMP/CAT rapporteur and point of contact at EMA
Early assignment of a rapporteur enables enhanced support on the clinical development and preparation of a marketing authorisation application for your product. Familiarising the rapporteur with your product at an early stage of development enhances their understanding of your product and target indication, which will be beneficial to you during the review process.
- Dedicated kick-off meeting with CHMP/CAT
The PRIME kick-off meeting aligns the initial regulatory strategy and overall development plan of your product with a multidisciplinary team from the EMA. Ultimately, you formulate your development strategy, but cohesion with regulators and early focused guidance will ensure that your regulatory strategy is endorsed, and the development process runs smoothly.
- Scientific advice guaranteed at Day 40
One of the main issues companies face with drug development in the EU is the often-lengthy timelines to receive scientific advice from the Agency. PRIME status enables you to receive expedited scientific advice at critical points in development. In addition, this can also include parallel meetings with Health Technology Assessment (HTA) bodies. This opens discussions to identify the data required to support reimbursement at an early stage in development for various countries throughout Europe. Our regulatory team at ProPharma are experts in conducting successful scientific advice interactions with national agencies and CHMP, and can support you from early-stage development, through to any post-approval requirements you may have.
- Accelerated assessment
Pharmaceutical and biotech companies who are awarded PRIME designation can expect to be eligible for accelerated assessment of their MAA when they come to file. Although accelerated assessment is open to any eligible products, achieving PRIME status provides assurance from an earlier stage of the process, facilitating planning for an expedited review.
Accelerated assessment essentially reduces the assessment time of medicines applying for marketing authorisation through the centralised procedure from 210 days to 150 days. If you are a US-based company, you could be used to a shorter assessment time than the EMA’s usual 210 days, particularly if you aim to use the Priority Review pathway. This benefit of PRIME means quicker access to medications globally for patients in need, and quicker recouping of development costs for you.
Do you meet the eligibility criteria for PRIME?
The bar for meeting the eligibility criteria is set high. As of May 2022, the overall approval rate for PRIME sits at 25% of applications, 40% of which were granted to SMEs and 29% granted to oncology products.2
PRIME focuses on novel medicines that have the potential to offer patients a treatment option where currently none are available, or which offer a significant therapeutic benefit over existing medicines that are already approved. As previously mentioned, it has to offer a major therapeutic advantage to patients by addressing a significant unmet medical need in a seriously debilitating or life-threatening condition. Proof of this should be provided from preliminary clinical evidence demonstrating that the medicine has the potential to provide a clinically meaningful improvement in efficacy or improves mortality/morbidity rates.
It is important to apply for PRIME at the correct stage. If you are a non-SME, the scheme is open to you when you have proof-of-concept data. If you are applying from academia or as an SME, in some circumstances you can support your application with convincing non-clinical data and/or tolerability and pharmacokinetics data from early clinical trials. Our regulatory experts at PPG can help you determine whether your data is currently sufficient to support a successful PRIME application.
How do you apply for PRIME designation?
To apply for PRIME you need to provide justification for why your product is eligible for the scheme.
The justification for applying for PRIME designation should include the following information:
- Background information on the disease.
- Is the disease a seriously debilitating or life-threatening condition?
- What is currently available (if anything) and what are the limitations of any existing therapies?
- What is the unmet need?
- Background on the product.
- How does your product work?
- What gaps in treatment (no existing treatment or limitations to existing treatments) does your product have potential to address?
- How does your product address the unmet need you outlined earlier?
- Supporting non-clinical and clinical evidence.
- Can you demonstrate that your medicine is showing promising early signs of having the potential to address this unmet need?
- Overview of the next steps in development.
- What are your plans to develop this medicine to get it to patients in need as quickly as possible?
- Don’t forget that ATMP applications must include information on tumorigenicity, immunogenicity, biodistribution, etc.
If your product is granted PRIME designation, you are invited to attend a kick-off meeting with EMA approximately 3 months later. This meeting is designed to open discussions on the recommended regulatory strategy as well as provide guidance on your overall clinical development plan.
Key things to remember when submitting a PRIME application are to keep it concise and to the point with regards to the data you present, but also be enthusiastic! PRIME medicines represent the most promising therapeutic breakthroughs – it’s exciting – let this shine through in your application. PPG are experts in PRIME applications and can support you in authoring a compelling justification for your product.
Differences between PRIME and The Innovative Licensing and Access Pathway (ILAP)
The ILAP pathway is a collaborative initiative introduced by the Medicines and Healthcare Regulatory Agency (MHRA), and much like the PRIME scheme, aims to support accelerated development, and ultimately marketing authorization, of novel therapies for serious diseases.
Although the PRIME scheme and the ILAP pathway are both initiatives with similar aims, there are several differences between their methodologies.
| PRIME |
ILAP |
| Open to new and novel therapies. |
Open to novel therapies AND new indications of existing therapies. |
| Support from experts within the European Medicines Agency (EMA). |
Support from the MHRA and multiple collaborating stakeholders, including the UK’s Health Technology Authority NICE. |
| Provides guidance on your product development. |
Includes the Target Development Profile (TDP) incorporating several different tools that you can add to help you build your product development programme. |
| Rapporteur and co-rapporteur designation will include experts from different countries within the EU with expertise in country specific regulation. |
The pathway is exclusive to the UK. |
| Historically a difficult scheme to be approved for (approximately a 25% approval rate)2. |
Greater than 50 % acceptance rate onto the pathway3. |
| Fee waivers and reductions for SMEs and academia. |
No additional fee incentives specific to the ILAP pathway. |
| PRIME granted at Day 40. Kick-off meeting approximately 3 months later. |
4-6 weeks to be granted an ILAP passport, followed by 4-6 weeks to receive your TDP. |
How we can help
ProPharma has supported several clients and medicinal product types through the PRIME scheme and can help you with the following activities:
- Formulating the PRIME application and justification.
- Managing the review procedure.
- Preparing a regulatory strategy to support your product development.
- Guidance and support for scientific advice interactions with regulatory bodies throughout your development lifecycle.
- Filing your (accelerated assessment) MAA and achieving approval as well as support post-approval, as required.
Interested in learning more? Contact us today to find out how we can help with your global regulatory needs.
References
- European Medicines Agency (2022, 22 April). Explanatory note on general fees payable to the EMA. Available at: https://www.ema.europa.eu/en/documents/other/explanatory-note-general-fees-payable-european-medicines-agency-01-april-2022_en.pdf. (Accessed 27th June 2022).
- European Medicines Agency (2022, 16-19 May). Recommendations on eligibility to PRIME scheme. Available at: https://www.ema.europa.eu/en/documents/report/recommendations-eligibility-prime-scheme-adopted-chmp-meeting-16-19-may-2022_en.pdf. (Accessed 27th June 2022).
- Medicines and Healthcare Products Regulatory Agency. (2021, 30 March). Innovative Licensing and Access Pathway Guidance. Available at: https://www.gov.uk/guidance/innovative-licensing-and-access-pathway. (Accessed 27th June 2022).
