Making Lifecycle Management More Efficient in Europe

In March 2024, the European Commission formalized its plan to update the European Union’s variation framework, making lifecycle management more efficient across the region and reducing the administrative burden on industry and regulatory authorities (1). This regulatory update forms part of the EU’s Pharmaceutical Strategy for Europe—an initiative aimed at future-proofing the regulatory framework and supporting industry innovation to help meet unmet needs (2).

“[This update] is really [part of a] wide revision of the pharmaceutical legislation,” said Alexander Natz, secretary general of European Confederation of Pharmaceutical Entrepreneurs AISBL (EUCOPE) in an interview with Pharmaceutical Technology EuropeTM (3). “[There hasn’t been] a revision of the pharma legislation over the past 20 years, so, it’s time to adapt legislation to make sure that new technologies are reflected.”

Fit for purpose

When revising the pharmaceutical framework, the commission assessed whether or not the regulatory system is fit for purpose, Natz explained. As an example, he highlighted the recent COVID-19 pandemic and associated lessons that have been learned. “[There is not] an emergency use marketing authorization in Europe, as many other countries have, and when the pandemic came along, [it was necessary] to look at what the right measure is to get the [COVID-19] therapies to European patients rather quickly,” Natz said.

More specifically, article 21 of the original regulation, pertaining to public health emergency situations, has been amended to allow for exceptional and temporary acceptance of a marketing authorization of a vaccine in the case that pharmaceutical, non-clinical, or clinical data are missing as long as the benefit-risk ratio is favourable. This variation is allowed with the proviso that the company submitting the vaccine, to treat the pathogen causing the public health emergency, provides the necessary data within a specified timeframe that has been laid out by the relevant authorizing body (4).

In addition to novel vaccines, industry has advanced the development of other biological medicinal products, such as recombinant DNA and cell and gene therapies. These advancements have necessitated a need for there to be a better classification of the products, which will in turn simplify the variation procedure and ease administrative requirements (5).

Not only have such lessons about new technologies been factored into the regulatory update, Natz continued, but the commission has also placed emphasis on affordability and access to medicine with its wider Pharmaceutical Strategy for Europe. Within Europe, new innovations are increasingly being successfully approved by the European Medicines Agency and the commission; however, ensuring reimbursement for patients is available when these innovative products get to market has been less successful.

“Of course, that’s the ultimate goal,” Natz stated. “There’s no value of a medicine which is approved, but which is not available in a certain country. So, there’s quite a bit of an emphasis on affordability and access to innovative medicines.”

Discussing exclusivity

Industry bodies, such as EUCOPE, and various industry stakeholders provided feedback and evidence on the regulatory update (1). Much discussion has been centred around industry regulatory data protection, noted Natz. “[This] is actually the period of protection of eight plus two years where generic medicines can use the data of the originator to opt in to apply to obtain their own marketing authorization and to compete,” he said. “So, it’s a type of an exclusivity period.”

From a company perspective, it is important to know what sort of exclusivity will be afforded once a product has been approved, Natz added. For investors, exclusivity is also a priority as they need to know how long they will have to recoup their investment where there will be no generic competition, he stated. “So, regulatory data protection and also often orphan market exclusivity have been very much at the forefront of the political discussion,” Natz specified.

However, despite there being some emphasis placed on improving the competitiveness of Europe as a pharmaceutical market, Natz revealed that there may have been a missed opportunity in terms of orphan legislation. There is a concept of global orphan marketing authorization (GOMA) that only allows for one specific period of exclusivity for therapeutic products with the same active ingredient, which means that if a company wishes to add a new indication, they may only get an extra one or two years of exclusivity, he remarked. “But if [a company] only gets one or two years, then this is not enough [time] to recoup the investment because [the company has] to actually do the same clinical trials as if it were a new compound,” Natz confirmed.

“Of course, some products in the past also in the orphan space, have been very successful to add four or five indications and oncology around one compound, and they all get these 10 years of exclusivity,” Natz said. However, if companies can only gain a further one or two years of exclusivity for each new indication then they will likely not invest, he stated.

Global awareness

The revised general pharmaceutical legislation will not come into effect until 2027 or 2028; however, it is imperative that companies, both local European companies and global companies, are aware of the measures, asserted Natz. “Decisions being made by global companies [include] picking and choosing the right territory to bring the product to first,” he said.

There is a phenomenon called ‘international reference pricing’, Natz explained, whereby governmental pricing models are based on the pricing found in other countries. “That phenomenon is so important and prominent in healthcare,” he stated.

As a result of these pricing habits, it is ever more pressing for the European authorities to not front-load pharma companies too much, in terms of costs and administrative requirements to bring a product to market, Natz confirmed. In the event of facing too high burdens, smaller innovators will be forced to out-licence to larger companies that can afford the development and commercialization costs, he emphasized.

In Europe, there is a need to consider how to broaden the availability of medicines across all 27 member states, Natz specified. “Especially from the smaller company perspective,” he said, “launching a product across 27 EU countries in two or three years is a difficult task, but also one that might not actually be needed.”

If there are no relevant patients in a country at a certain point for a specific therapeutic product, Natz questioned why should a company have to absorb the costs of running through a health technology assessment procedure for that country? “[This process] front-loads costs and [it would be better for Europe] to use more flexible tools like cross-border healthcare,” he said.

“[It is critical] to be diligent enough and careful enough to not overload the system with too many burdens,” Natz summarized.

References

1. EC. Pharmaceuticals—Changes to Marketing Authorizations (Review of EU Rules). EC.europa.eu (accessed 7 April 2024).
2. EC. Pharmaceutical Strategy for Europe. Initiative, 25 Nov. 2020.
3. Thomas, F. Europa Perspectives on Lifecycle Management. PharmTech.com, 9 April 2024.
4. EC. Commission Delegated Regulation (EU) …/... of 11.3.2024 Amending Regulation (EC) No 1234/2008 as Regards the Examination of Variations to the Terms of Marketing Authorisations for Medicinal Products for Human Use. Brussels, Belgium (11 March 2024).
5. EC. Commission Staff Working Document Accompanying Commission Delegated Regulation (EU) …/... of 11.3.2024 Amending Regulation (EC) No 1234/2008 as Regards the Examination of Variations to the Terms of Marketing Authorisations for Medicinal Products for Human Use. Brussels, Belgium (11 March 2024).

About the author

Felicity Thomas is the European/senior editor for Pharmaceutical Technology Group.

Article details

Pharmaceutical Technology Europe
Vol. 36, No. 4
April 2024
Pages: 13–14

Citation

When referring to this article, please cite it as Thomas, F. Making Lifecycle Management More Efficient in Europe. Pharmaceutical Technology Europe 2024 36 (4) 13–14.