Euro Roundup: EMA seeks feedback on plans to cut the clinical data needed for biosimilar approval
The European Medicines Agency (EMA) has proposed reducing the amount of clinical data needed to develop and approve biosimilars, according to a draft reflection paper designed to improve access to biosimilars in the EU.Currently, biosimilar developers need to file comparative clinical pharmacokinetic, pharmacodynamic, safety, and efficacy data as part of their applications for EMA approval. Requirements can be waived if an accepted pharmacodynamic or surrogate endpoint exists, but EMA now believes “even this prerequisite might not be needed.”
EMA’s proposals reflect the knowledge that a molecule’s biological activity stems from how it interacts with its receptor. As EMA has gained experience with biosimilars and analytical methods have improved, the agency has begun to think comparative efficacy studies “may no longer be required for approval of biosimilars that can be thoroughly characterized and have shown high similarity on an analytical and in vitro pharmacology level.” The need for pharmacodynamic data may also be waived in some cases.
EMA said comparative clinical pharmacokinetic studies are still an essential part of biosimilar development, but some adjustments to the data requirements could be considered. EMA proposed the inclusion of immunogenicity parameters and changes to the study design as potential adjustments.
The agency proposed waiving comparative studies early last year (RELATED: EMA proposes waiving comparative efficacy studies for certain biosimilars, Regulatory Focus, 2 February 2024). While last year’s concept paper sketched a plan over a few pages, the draft reflection paper discusses EMA’s views on the necessity of running comparative efficacy studies.
The discussion is divided into two main sections. In the quality section, the agency outlines how the similarity assessment process could give it the confidence to reconsider the need for comparative efficacy studies.
EMA would need to see a data package that shows the mechanism of action and structure-function relationship are well understood, resulting in critical quality attributes that are well known and can be reliably evaluated in a quality risk assessment. The agency cited many cell-based medicinal products as examples of products that would not meet that requirement.
Other prerequisites include using sensitive analytical methods and characterizing enough batches to estimate batch-to-batch variability correctly. EMA expects similarity assessments to be preplanned and performed using defined conditions and criteria.
Today, EMA typically requires comparative efficacy studies even if a molecule and program meet those requirements. This reflects its reliance on clinical data to address uncertainties about biosimilarity and confirm equivalent performance to the reference product in humans.
EMA is accepting feedback on the proposals until 30 September.
Press Release, Draft Paper
EMA starts consultation into draft guideline on the quality aspects of mRNA vaccines
EMA seeks feedback on manufacturing, characterization, specifications, and analytical control of mRNA vaccines against infectious diseases. The agency shared its position in a draft guideline.
The COVID-19 pandemic accelerated the progress of mRNA vaccines against infectious diseases. Existing guidelines for human vaccines apply to products such as Moderna’s Spikevax and Pfizer’s Comirnaty, but those documents do not specifically address mRNA. The gap led EMA to draft guidance on development, manufacture, and control requirements specific to mRNA vaccines.
Only the mRNA is considered an active substance, and EMA proposes that companies submit the complete mRNA sequence. The section on the active substance addresses topics such as the control of starting materials, which for mRNA vaccines include components such as nucleotides and the 5’ cap, and characterization studies. EMA outlines controlling the active substance and determining a product’s shelf life.
Subsequent sections address the finished product, including details such as controlling excipients and the finished product, and discuss regulatory considerations specific to mRNA vaccines. The regulatory section covers considerations for changes in existing mRNA vaccine strains, bivalent and multivalent vaccines, self-amplifying mRNA vaccines, and delivery systems other than lipid nanoparticles.
The consultation will close on 30 September.
Draft Guideline
EMA publishes draft reflection paper on providing electronic product information
EMA has begun a consultation into how companies can use existing two-dimensional codes on medicine packaging to link patients to the latest electronic product information (ePI).
The European Medicines Regulatory Network is currently developing ePI for human medicines, with a view to creating and updating electronic information as part of routine regulatory procedures. The initiative is built on the belief that ePI can benefit public health by expanding patient access to up-to-date information on the medicines they take.
A draft reflection paper outlines how the system could work. EMA proposes linking to the ePI using the two-dimensional DataMatrix, which products carry to meet falsified medicine requirements.
“This same identifier can allow the linking of the package to the ePI with no change needed to the DataMatrix printed on the package today, ensuring the display of the correct, authorized ePI for the medicine in the consumer’s hands,” EMA said. “Directing the user as easily as possible to the electronic package leaflet for the medicine should be the highest priority” for solutions with patients as a target group. Prioritization reflects that up-to-date, regulator-authorized information is specifically targeted to patients.
EMA is accepting feedback on the draft until 30 June.
Draft Paper
Swissmedic updates meeting application options to enable early talks with sponsors
The Swiss Agency for Therapeutic Products (Swissmedic) has offered sponsors the option of engaging in earlier talks about their programs.
Swissmedic already allowed companies to arrange meetings to ask questions related to the development and authorization of drug candidates. This week, Swissmedic changed its process to allow companies to request a meeting before application submission at any phase of development of a medicinal product.
“The newly designed meeting enables companies to have an early dialogue with Swissmedic and should help companies to clarify content- and process-related questions with Swissmedic,” the agency said. “The meeting before application submission is not linked to a specific future authorization application and is valid for all procedures. It is possible to address a combination of discussion topics.”
Swissmedic has combined the former scientific advice, pipeline, and pre-submission meetings to create the new meeting before the application submission option. The agency updated its guidance on meetings to reflect the changes, which took effect on 1 April.
Press Release
Other news
Swissmedic has expanded its position paper on real-world evidence. The agency said the expanded third version of the document provides an “overview of relevant international guidelines and publications to reflect the current situation in science and technology.” Swissmedic disclosed the update alongside changes to guidance on fast-track and temporary authorizations. Position Paper, Fast-track Guidance
The Medicines and Healthcare products Regulatory Agency (MHRA) published guidance on how to apply for exceptional use authorization for medical devices. As the guidance explains, the regulations covering the authorizations are different in Northern Ireland and the rest of the UK. The criteria are similar, though, with devices being authorized for public or patient health. MHRA Guidance
EMA published a draft concept paper on revising a plasma master file guideline. The agency is accepting feedback until 30 June. Draft Paper
×
Welcome to the new RAPS Digital Experience
We have completed our migration to a new platform and are pleased to introduce the updated site.
What to expect: If you have an existing login, please RESET YOUR PASSWORD before signing in. After you log in for the first time, you will be prompted to confirm your profile preferences, which will be used to personalize content.
We encourage you to explore the new website and visit your updated My RAPS page. If you need assistance, please review our FAQ page.
We welcome your feedback. Please let us know how we can continue to improve your experience.