How successful are the EMA’s Pharmaceutical SME initiatives?

by | Feb 22, 2024 | European Medicines Agency, Regulatory Affairs

Highlights

  • A re-cap on why SMEs are integral to pharmaceutical innovation
  • How successful are the EMA’s SME initiatives?
  • Highlights of the EMA’s SME initiatives to date
  • What SMEs think of the EMA’s SME initiatives

Re-cap on the importance of SMEs to pharmaceutical innovation

In Part 1 of this SME blog series–How the EMA helps SMEs–we covered the importance of SMEs to medicine development and pharmaceutical innovation, supported by statistics from the European Medicines Agency (EMA).

To re-cap, the EMA published two reviews investigating the profiles of each marketing authorisation holder (MAH) between 2010 and 2019, to identify the type of enterprise each new and innovative medicine originated from. They reported that out of 369 new and innovative medicines approved between 2010 and 2019, 23% originated from SMEs.1

The EMA summarised the effect of SMEs, academia and public bodies on pharmaceutical innovation brilliantly:

“SMEs, academic institutions, public bodies and PPPs represent an important source of innovation and enrich the product pipelines of larger companies. Notably, the data presented here highlight the important role of SMEs in the upstream phase of pharmaceutical innovation, especially for orphan drugs.”2

Since its inception in 2005, the European Commission’s SME Regulation and the EMA’s SME initiatives have offered support, guidance and regulatory fee incentives to SMEs developing medicines in Europe. Now, nearly two decades on, it’s interesting to see how successful the EMA’s SME initiatives have been in practice.

Latest figures from the EMA’s 2022 SME Annual Report

As of the end of 2022, there were 1922 SMEs registered with the EMA. Of these registered SMEs, 36% were micro-sized organisations; 35% were small organisations and 29% were medium-sized organisations.3

Additionally, 13% of registered SMEs were made up of SME service providers and regulatory consultancies. US-based medicine developers receiving SME incentives accounted for 8.4% of SMEs, and they did so through European SME service providers.3

Somerville Development Partners BV holds SME status. For our US clients, who do not have a legal entity based in Europe, partnering with us is invaluable to benefit from the additional support and fee reductions the EMA offers.

As of 2022, 78% of registered SMEs reportedly developed pharmaceuticals and 19% developed pharmaceuticals and medical devices.3

Types of medicines being developed by SMEs as of 2022:3

  • 52% were chemical-based products,
  • 21% were biologics,
  • 23% were orphan medicines,
  • 11% were paediatric medicines,
  • 9% were advanced therapies, and
  • 5% were vaccines.

 SME success highlights reported by the EMA in 2022:3


Marketing Authorisation Applications (MAA):
13 MAA submissions in total; 5 were for new active substances.
5 positive opinions: 1 new active substance and 2 orphan medicines.
4 negative opinions and 3 withdrawals.

PRIME designation:
7 out of 13 companies that received positive eligibility for PRIME were SMEs.

ATMP classification:
21 out of 46 recommendations for ATMP classification were granted to SMEs. 

Services most frequently used by SMEs in 2022:3

  • 43% of all qualifications of novel methodologies
  • 29% of all protocol assistance requests (for orphan medicines)
  • 22% of all scientific advice requests were from SMEs
  • 19% of all scientific advice requests for PRIME were from SMEs

The EMA publishes annual figures concerning its SME initiatives, which helps us track its progress and trends over time. However, it’s important for SMEs who aren’t currently registered as SMEs to hear from organisations that are registered and have utilised the EMA’s initiatives.

What do SMEs think of the EMA’s SME initiatives in practice?

In 2020, the EMA conducted an online surveyto gain feedback from SMEs (85% of respondents)4 and stakeholder organisations developing and marketing medicines. The survey aimed to investigate the SMEs’ overall experience of the EMA’s initiatives, as well as the challenges they continue to face.

Overall satisfaction with the EMA’s SME initiative

Overall, the satisfaction of the EMA’s SME qualification process was very high. This process was split into 3 sections: guidance, contact with the SME office and qualification timelines, as illustrated in Figure 1.

Bar chart showing the opinions of pharmaceutical and biotech SMEs regarding the EMA SME qualification process
Figure 1: Source: EMA’s Outcome of SME Office Survey 2020.4

Additionally, 75% of respondents were also satisfied with the overall scope of the EMA’s support to target SMEs (24% – “very satisfied” and 51% – “satisfied”). In terms of how relevant SMEs found the EMA’s SME support overall, of 516 respondents, 37% deemed the EMA’s SME support ‘very relevant’ and 52% found it ‘relevant’.4

Awareness of the EMA’s SME support

In total, 80% of respondents were aware of the EMA’s support for SMEs, with only 23% of respondents knowing ‘to a great extent’.4 The survey expanded the question of awareness to the individual SME initiatives the EMA provide, which gives a granular view of the degree each tool was utilised (see Figure 2).

Bar chart showing the opinions of pharmaceutical and biotech SMEs regarding how aware they were of EMA SME support activities.
Figure 2: Source: EMA’s Outcome of SME Office Survey 2020.4

The highest level of awareness for SME activities:4

  • SME newsletter: 39%
  • Fee incentives (pre-authorisation) 38%
  • SME user guide/public SME register/reg assistance services 27%

Lowest awareness:4

  • Support to PRIME 15%
  • ATMPs certification 11%
  • EMA’s policy 0070 (publication of clinical data) 7%

Challenges SMEs faced

The EMA’s survey delved into the main challenges SMEs faced (see Figure 3).

Bar chart showing the opinions of pharmaceutical and biotech SMEs regarding the main challenges they face.
Figure 3: Source: EMA’s Outcome of SME Office Survey 2020.4

Administrative and regulatory burdens were specific to how complex regulations are, the need to prepare multiple regulatory documents, and an apparent lack of harmonisation between regulatory authorities regarding regulatory requirements.

Somerville Development Partners has over 20 years of experience preparing and submitting regulatory documents for all pre-authorisation regulatory deliverables; from initiating scientific advice with European regulatory authorities to the marketing authorisation application.

How the EMA could improve its SME initiatives

The survey also asked SMEs if they felt that the EMA’s support activities could be revised or expanded. The activities that respondents agreed could benefit from revision/expansion the most included:4

  • Regulatory assistance services (62%),*
  • SME training events (57%),* and
  • financial incentives (pre-authorisation 53%) post-authorisation (52%)*

*Percentages included responses agreeing with the need to revise or expand ‘to a great extent, ‘to some extent’ or ‘to a minor extent’.

Following the 2020 survey, the EMA offered representatives from the European Commission, SMEs and stakeholders the chance to discuss the results of the survey in an online roundtable event. This was also an opportunity for SMEs to offer opinions on the EMA’s SME initiative and highlight aspects for future improvement.

Fifteen years after the SME regulation was published, the SME stakeholders acknowledged the importance of the SME initiative, as well as its continued relevance; this also included the EMA’s PRIority Medicines scheme (PRIME).

Feedback from SMEs and other stakeholders

Aspects of the SME initiative that were highlighted for improvement included:5

  • Bringing more awareness to the SME scheme,
  • offering earlier and quicker scientific advice,
  • improving access to funding and incentives,
  • improving support for the PRIME scheme, and
  • including health technology assessment (HTA), with its real-world evidence focus; especially for medicines targeting rare diseases.

Include SMEs in legislation

Another point made by the SMEs during the round table was to include SMEs when bringing forward relevant legislation so that the needs of SMEs could be suitably addressed.

Offer support to rapidly growing SMEs

Additionally, the SMEs highlighted the difficulty faced by small but rapidly growing pharmaceutical—and especially biopharmaceutical companies—who may not be compliant with the definition of an SME due to injections of investment made by major venture capital investors. Companies such as this will not fall under the category of an SME and cannot benefit from fee incentives or the additional the EMA offers.

Include other types of organisations

The SME round table participants also suggested that universities and not-for-profit organisations should also be considered SMEs and have access to the associated benefits of receiving SME status.

Summary

The SME Regulation and its subsequent implementation by the EMA has—and continues to—offer invaluable guidance, support and fee incentives to SMEs. After nearly two decades, the value of the EMA’s support is still acknowledged. However, SMEs and other relevant stakeholders voiced their opinions on the challenges they still face and how the EMA could improve its support for SMEs, starting with bringing more awareness to the SME scheme.

Shedding light on the support the EMA offers—both financially and educationally—doesn’t just benefit SMEs individually; it benefits medicine development as a whole because SMEs are an important driving force of medical innovation.

How Somerville Development Partners can help

We are a registered SME in Europe.

This means we can access SME benefits and fee incentives on your behalf if your organisation is not established in the European Union (EU)/ European Economic Area (EEA).

We have decades of experience navigating regulatory agency interactions and regulatory submissions in Europe and can confidently guide you through your regulatory milestones, including;

Get in touch

We welcome the opportunity to discuss scientific advice and regulatory strategy with you!

Nicole

Author

Nicole Brooks, Regulatory Consultant / Copywriter
Nicole@somerville-partners.com

References

1. Provansal C, Dooley D, Ziogas C. Pharmaceutical innovation sourcing. (2022). Nature Reviews Drug Discovery; 21(9):627–627. Available at: https://www.nature.com/articles/d41573-022-00125-y

2. Lincker H, Ziogas C, Carr M, Porta N, Eichler HG. (2014). Where do new medicines originate from in the EU? Nature Reviews Drug Discovery [Internet]. (2):92–3. Available at: https://www.nature.com/articles/nrd4232

3. European Medicines Agency (2022). SME Office Annual Report. Available at: https://www.ema.europa.eu/en/documents/report/small-and-medium-sized-enterprise-sme-office-annual-report-2022_en.pdf

4. European Medicines Agency (2020). Outcome of SME Office survey on the implementation of the SME Regulation (EC) No 2049/2005. Regulatory Science and Innovation Task Force. EMA/277477/2020. Available at: https://www.ema.europa.eu/en/documents/report/outcome-sme-office-survey-implementation-sme-regulation-commission-regulation-ec-no-20492005_en.pdf

5. European Medicines Agency (2020).Report on the European Medicines Agency Roundtable with Stakeholders – 15 years supporting SMEs. Regulatory Science and Innovation Task Force. EMA/671119/2020. Available at: https://www.ema.europa.eu/en/documents/report/report-european-medicines-agency-roundtable-stakeholders-15-years-supporting-smes_en.pdf