1. Supporting SME
Eléonore Cornu
M2 Areips
Supporting SMEs

2. Plan SME definition The SME office SME incentives
Results and conclusion

3. Introduction
The SME Regulation was adopted in December to promote innovation and the development of new medicines by SMEs. Its main objective is to provide financial, regulatory and administrative assistance to micro, small and medium-sized enterprises.

4. SME definition
The SME criteria are set out in the Commission Recommendation 2003/361/EC and are based on two factors:
- the enterprise’s size
- the type of enterprises
SME definition introduced in 1996 in order to have a common definition of SME for all the member states. Revised in 2003

5. The type of structure
Companies are classified according to their category and sizes

6. The enterprise’s size
Companies are classified according to their category and sizes

7. Applying for SME status
Electronic declaration form
The most recent annual accounts
Proof of establishment of the company in the EU/EEA
A chart of the ownership structure
Last for 2 years
After review, the Agency issues an SME number and notifies the applicant that SME status has been assigned. This status is applicable solely for Agency-related activities and does not serve for other national or EU funding programmes.

8. The SME Office A single interface Facilitate communication
User guide : explain the main aspects of medicinal product legislation
The office has dedicated personnel who can help SMEs by :
Responding to practical or procedural enquiries
Setting up briefing meetings to discuss regulatory strategy
Organising workshops and training sessions
1738 SME in October

9. SME newsletter, workshop and meetings
Newsletter : key updates on the European regulatory environment
Workshop : to address the particular needs of SMEs
The newsletter for micro-, small- and medium-sized enterprises (SMEs), published four times a year, provides key updates to SMEs on the European regulatory environment.
The SME office hosts educational workshops each year which are available for attendance in person or remotely. Video recordings, presentations and related information for each workshop is available.
Non clinical aspects, statitstiques, chemistry manufacturing and control CMC …
The SME office offers SME briefing meetings, which provide a platform for a company to discuss its planned regulatory strategy. SMEs are encouraged to approach the SME office to request a briefing meeting at any stage of their product development
Free of charge
Meetings : to discuss the planned regulatory strategy

10. Public SME register Online platform
Facilitate interaction, partnering and networking

11. Translation assistance
Translation into all official EU languages by the centre for translation in Luxembourg
Translation of:
Summary of product characteristics
Label
Package leaflet
Assistance with translations of product information into all official European Union languages
Translating product information into all EU languages represents a considerable financial and administrative burden to SMEs entering the EU market. Thus, EMA provides translations of product information (summary of product characteristics, label, package leaflet and relevant opinion annexes) required to grant an EU marketing authorisation. Translation into EU official languages are provided free of charge by the Agency. The Agency initiates translations through the centre for translation (CdT) in Luxembourg at the time of CHMP/CVMP opinion

12. Financial fee incentives
Procedure
Fee incentives
Scientific Advice
90% reduction for non-orphan products
100% reduction for orphan products
100% reduction for products granted eligibility to PRIME
Inspection (pre-authorisation)
90% reduction and deferral
Application for marketing authorisation
Fee deferral
Conditional fee exemption
Post-authorisation procedures
100% reduction for micro-sized enterprises
40% reduction for SMEs
100% reduction for orphan products during the first year after MA
Scientific services
90% reduction for non-orphan products
Administrative services (excluding parallel distribution)
100% reduction
Inspection (post-authorisation)
90% reduction
EMA fee incentives are applied if an enterprise has been assigned SME status by the Agency. Companies are advised to register as an SME or renew their status well in advance of a planned regulatory submission to the EMA.
Conditional fee exemption, where EMA scientific advice is followed and a marketing authorisation application is not successful

13. ATMP classification
Consult the CAT to determine whether the medicine is an ATMP or no.
The criteria for ATMPs are set out in Article 17 of regulation 1394/2007
Optional procedure
Response in 60 days
Advanced therapy medicinal products (ATMPs) are defined in legislation as gene therapy, somatic cell therapy and human tissue engineering.
Sponsors requiring clarification as to whether their product is classified as an ATMP can receive confirmation from the committee for advanced therapies (CAT) prior to submitting any application to the Agency. This advice is provided free of charge within 60 days of receipt of a valid request from an applicant. The Agency publishes summaries of these recommendations after deletion of all information of a commercially confidential nature.

14. ATMP certification exclusively for SMEs
Certification provides a scientific evaluation of experimental data quality and non clinical data generated with the product
90 day review by the CAT
Certification: Confirms that available data comply with the standards required for Marketing Authorization Application (MAA)
Identify any potential issues
Not legally binding
The certification procedure is defined in Article 18 of Regulation 1394/2007
9 requests -> 7 certifications
Exemple : ATIR 101 Kiadis Pharma
(Allodepleted T-cell ImmunotheRapeutic) is an innovative cell-based therapeutic that addresses the
key risks and limitations of current HSCT treatments in blood cancers and inherited blood disorders, being
opportunistic infections, Graft-versus-Host Disease (GVHD), cancer relapse and limited donor availability.
Using Kiadis Pharma’s Theralux platform, T-cells that attack the patient, causing GVHD, are eliminated. At the
same time, the full immune repertoire of donor immune cells, including immunological memory, is retained in
the final product to fight infections. ATIR101 also contains T-cells from the donor that could eliminate residual
cancer cells reducing the risk of return of the disease, which is called the graft-versus-leukemia (GVL) effec

15. Number of SME
The number of SMEs registered with the Agency at the end of year 2015 was 15 times higher than in 2006 and increased by 24% compared to the previous year. Significant increases in 2012 and 2015 were due to companies registering respectively for MedDRA fee incentives and for Pharmacovigilance fee incentives. the process for applying for SME status has been simplified in order to reduce the administrative burden on applicant companies. An automated electronic SME declaration form which calculates the headcount, annual turnover and balance sheet total in line with the ownership structure of the enterprise was launched in 2011
October 2016, 1738
Celixir Heartcel Allogeneic immunomodulatory progenitor cell. Tissue engineered ATMP
Designed to regenerate heart tissue scarred by myocardial infarction
Advice from SME office on administrative and procedural issues around
ATMP classification
Orphan designation
Paediatric investigation plans

16. MAA outcome

17. Type of medicine

18. Thank you for your attention

19. Bibliography SME user guide User guide to the SME definition
Commission regulation 2049/2005
Commission recommendation 2003/361/EC
Explanatory note on general fees payable to the EMA
Report on the 10th anniversary of the SME initiative
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