Biotech and pharmaceutical companies face numerous challenges when commercialising their products. Navigating complex, ever-evolving regulatory requirements, meeting stringent safety and efficacy standards and adhering to tight approval timelines can be daunting.
Gaining marketing authorisation in the UK is a particularly rigorous undertaking, demanding comprehensive dossiers, strict adherence to timelines and in-depth knowledge of evolving regulations. This complexity is magnified for innovative treatments targeting rare diseases, where specialised data and small patient populations present unique hurdles.
For many small and medium-sized enterprises (SMEs), the lack of in-house regulatory expertise can turn the regulatory submission process into an arduous journey.
In this blog, we explore three common challenges faced during the Medicines and Healthcare Products Regulatory Agency’s (MHRA) 150-day approval timeframe — examining how these hurdles intersect with drug development in the rare disease space and how an EU/EEA-based marketing authorisation holder (MAH) service can streamline your path to new drug approval.
Challenge one: tight regulatory submission timeframes
The MHRA mandates a strict 150-day timeframe from dossier acceptance to final decision. Within this window, you must prepare and submit a flawless dossier and promptly address any clarification requests. Missing a deadline can lead to automatic refusal, restarting the entire regulatory submission cycle and incurring significant delays.
Many biotechnology and pharmaceutical SMEs lack dedicated regulatory affairs teams. However, rapidly assembling the multidisciplinary expertise required — including clinical, non-clinical, chemistry manufacturing controls (CMC) and quality systems — can be overwhelming. Early and meticulous planning is, therefore, vital: defining a detailed project timeline, allocating responsibilities and building in contingency for MHRA queries are essential to meet the clock.
For therapies targeting rare diseases, the 150-day constraint is particularly sharp. These programmes often rely on small, geographically dispersed patient cohorts, making data collection slower. Any request from the MHRA for additional patient-level data or clarifications can threaten the submission’s viability within the regulatory timeframe, amplifying the risk to project timelines and budgets.
Challenge two: the complexity of rare disease regulations
The MHRA recognises the ethical and logistical challenges in drug development for rare diseases, but it still requires robust evidence of safety, efficacy and manufacturing quality. Sponsors must present tailored non-clinical models, often leveraging orphan drug designation incentives, while ensuring the scientific rigour aligns with regulatory expectations.
Compiling the necessary clinical data for a rare disease regulatory submission typically involves heterogeneous sources: natural history studies, compassionate use programmes and small-cohort clinical trials. Integrating these disparate data streams into a coherent Module 5 (clinical study reports) of the common technical document (CTD) requires both scientific acumen and regulatory insight.
While incentives such as fee reductions and protocol assistance are invaluable, they introduce additional steps in the regulatory submission process. As such, you must carefully manage orphan drug designation applications alongside the marketing authorisation application, ensuring alignment in endpoints, patient populations and clinical trial designs to avoid divergent regulatory expectations that could stall your new drug approval.
Challenge three: post-Brexit adjustments
Since the UK’s withdrawal from the EU, companies must secure separate UK marketing authorisation in addition to any EU-wide approvals. As such, if you only have an EU headquarters, you now face the added complexity of establishing UK legal infrastructure, registering UK Qualified Persons and updating pharmacovigilance systems to satisfy MHRA pharmacovigilance obligations.
Coordinating parallel EU and UK regulatory submission timelines can lead to misaligned review cycles. Equally, divergent queries from the European Medicines Agency (EMA) and MHRA may necessitate separate responses, doubling the workload. For new drug approvals, sponsors must balance these dual pathways to optimise market access across both territories without compromising quality or compliance.
There are two options here: you can either create a UK subsidiary — which often adds time, cost and administrative burden to already strained resources — or engage a third-party marketing authorisation holder service.
How a marketing authorisation holder can help
Engaging a professional marketing authorisation holder shifts the regulatory burden away from you, allowing you to focus on core drug development activities instead.
A marketing authorisation holder’s main role is to act on your behalf — managing all regulatory and legal accountability, complying with the MHRA 150-day timeframe requirement for product authorisation and guaranteeing your product meets the highest safety and efficacy standards.
The three key objectives of an MAH service include:
- Advising you in the preparation of a regulatory-compliant dossier for your marketing authorisation application (MAA).
- Acting as your marketing authorisation applicant if you don’t have a suitable UK/EEA entity.
- Acting as your UK marketing authorisation holder following MHRA approval, which allows you to commercialise your new product.
By partnering with an experienced MAH, you gain access to in-house regulatory specialists familiar with UK-specific and rare disease regulations, significantly reducing timetables and mitigating the risk of non-compliance.
Fast-track your marketing authorisation application
Navigating the MHRA’s stringent 150-day timeframe, the intricacies of rare disease regulations and the post-Brexit landscape presents substantial challenges for biotech and pharma SMEs.
At TMC, our unique EU/EEA-based marketing authorisation holder service offers an end-to-end solution.
TMC is highly experienced in the UK regulatory environment and can act as your marketing authorisation applicant and, if required, your UK marketing authorisation holder. Our extensive experience with regulatory submissions for rare disease drugs ensures we’re equipped to handle the unique challenges these products encounter. Thanks to a deep understanding of the science behind these treatments, coupled with our knowledge of complex regulatory requirements specific to each therapeutic area, we provide tailored solutions that accelerate the new drug approval process.
TMC’s ability to serve as a UK marketing authorisation holder for its clients has proven to be a game-changer in the commercialisation process. By taking on the full legal and regulatory accountability of your product — managing all aspects of the regulatory submission process — we ensure your product can compliantly enter the UK market sooner.
To support your marketing authorisation application and help you gain faster market approval for your novel rare disease treatment, contact TMC’s regulatory team today at regulatory.services@tmcpharma.com.