european regulatory development

Regulatory Strategy

Strategic thinking transcends conventional "regulatory guidance" encompassing a holistic approach that aligns various functions within an organization to establish mutually formulated and embraced goals. At ProPharma, we understand that a successful regulatory strategy extends beyond the surface, covering the entire lifecycle of a development program. We take a forward-looking approach, aiming to identify changes, assess risks, anticipate roadblocks, and surmount hurdles while advising on pertinent mitigations. This approach is not only global but also adaptable to specific regional or subregional needs, allowing for nuanced focus on sub-strategies, such as pediatric and orphan development, and the ability to address single questions, review clinical development planning, and provide gap analyses.

ProPharma brings substantial expertise to the table when partnering with clients to develop and realize their regulatory strategies. We have supported over 50 clients in crafting tailored regulatory strategies for complex development programs spanning various therapeutic areas and product types. Our profound knowledge across nonclinical, clinical, and CMC domains ensures meticulous consideration of potential hurdles and their effective mitigations. With ProPharma as your partner, you can confidently navigate the optimal path forward for the development of your product in Europe, secure in the knowledge that your regulatory strategy is in expert hands.

Nonclinical Development

In the realm of pharmaceutical development, a robust nonclinical package is the cornerstone of success, especially in the early phases and beyond. It's the compass that guides you through regulatory expectations and provides the precise data needed to bolster your clinical development efforts. At ProPharma, we recognize the paramount importance of this phase, and our dedicated team of nonclinical experts collaborate closely with each client to deliver a nonclinical package that not only meets regulatory requirements but also aligns seamlessly with the client's overarching drug development strategy.

Our nonclinical experts are adept at defining the specific requirements for nonclinical studies, meticulously identifying and mitigating potential risks, and proactively addressing any gaps or development issues. With this holistic approach, we ensure that your nonclinical package is not only compliant, but of the highest quality, providing the foundation upon which successful clinical development is built.

Moreover, ProPharma operates with a flexible mindset, recognizing that each client's needs and circumstances are unique. We collaborate within our nonclinical team and with our counterparts in other critical areas, including CMC and clinical development, to ensure a cohesive strategy that harmoniously integrates nonclinical data with other facets of drug development. We are unwavering in our commitment to closely monitor the ever-evolving regulatory landscape, ensuring that the pathway to clinical development is well-mapped and seamless.

When you partner with ProPharma, you can rest assured that your nonclinical package will not only meet regulatory expectations but will be a powerful asset in driving your drug development journey forward. Our dedication to quality, adaptability, and regulatory expertise ensures that your path to clinical development is marked by success and confidence at every step.

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CMC Plan Development

CMC is a critical component and a ‘first step’ in the creation of a development strategy for an asset. Without a solid development plan for the investigational product, regulatory agencies will not be able to adequately assess the safety and efficacy for approval of clinical trials and later approvals.

We understand that companies need to carefully balance the costs of early investment in CMC-related activities, when cash burn needs to be more tightly controlled, versus early under-investment in CMC leading to significant extra costs and critical delays later in development. It is especially important for companies developing treatments for indications with high unmet need where despite expedited pathways offering accelerated approvals based on early clinical data, the CMC package must be complete at the time of filing.

ProPharma consultants have decades of experience in the development of small molecules, biologicals and ATMPs, and partner with companies to develop a phase-appropriate CMC plan leveraging our expertise in bringing innovative medicines to patients and our experience of regulator hot topics and needs.

Submissions & Applications

Expedited Pathways

ProPharma offers comprehensive expertise in navigating the evolving regulatory landscape in Europe to expedite the approval process for innovative products. In a rapidly changing environment, understanding the nuances of regulatory programs is crucial for sponsors seeking efficient development and market access. While the FDA's Accelerated Approval, Priority Review, Fast Track Designation, and Breakthrough Designation programs are well-known in the U.S., ProPharma emphasizes the need for clients to explore their European counterparts. The European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) offer incentives like PRIME Designation, Conditional Marketing Authorization (CMA), Accelerated Assessment, and Exceptional Circumstances, each with its distinct benefits and requirements.

ProPharma's in-depth knowledge extends to the PRIME initiative, which provides accelerated assessment criteria and early Rapporteur appointments. Despite its potential, PRIME designation can be challenging to secure, and ProPharma guides clients through this process. CMA, analogous to U.S. Accelerated Approval, offers a route for products addressing unmet medical needs, but ProPharma emphasizes the importance of demonstrating a positive benefit-risk balance. The exceptional circumstances pathway recognizes the challenges posed by ultra-rare diseases, allowing flexibility while maintaining rigorous evaluation.

ProPharma also delves into accelerated assessment in Europe and its major public health interest criteria. Our team utilizes the emerging concept of rolling review and its potential benefits, along with the UK's Innovative Licensing and Access Pathway (ILAP), offering regulatory collaboration and rolling reviews. The global collaboration initiatives, including Project Orbis and international recognition frameworks, demonstrate the UK's commitment to expedited access.

Scientific Advice

At ProPharma, we specialize in supporting clients with their scientific advice projects, recognizing the critical role this procedure plays in drug development. Scientific advice is a powerful tool that facilitates direct dialogue between sponsors and regulatory authorities, enabling the refinement of data requirements tailored to a specific development program. While guidelines and regulations provide a foundation, there's no substitute for the insights gained from engaging with regulatory agencies. Scientific advice serves as a cornerstone in the creation of robust marketing authorization dossiers, ultimately reducing attrition rates, mitigating negative regulatory outcomes, and expediting time-to-market while controlling costs.

What sets ProPharma apart is our deep expertise in European scientific advice procedures and initiatives. Our team includes experts who have spearheaded and actively participated in numerous procedures within regulatory agencies, accumulating a wealth of experience. We have supported over 150 scientific advice procedures, equipping us with an intricate understanding of 'when,' 'how,' and 'what' to ask for during these engagements—knowledge that holds strategic significance. Moreover, our exceptional European Regulatory team comprises former members of European regulatory agencies who have led scientific advice procedures from the regulator's perspective. This unique perspective enables us to provide invaluable insights and adeptly manage document drafting and discussions with regulatory agencies, optimizing our clients' scientific advice interactions.

With ProPharma as your partner, you can confidently navigate the complex landscape of scientific advice, harnessing the full potential of this crucial process to advance your drug development endeavors efficiently and effectively.

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Clinical Trial Applications including GMO and IVDR

Navigating the intricate landscape of clinical trial submissions in Europe demands expert touch; we know this because we’ve done it over and over again.

Clinical Trial Application (CTA) procedures in the UK and EU have undergone significant transformations. The harmonization of CTA procedures across these regions has streamlined the process, allowing for a single submission that covers both competent authorities and ethics committees. At ProPharma, we've been at the forefront of navigating these changes and ensuring our clients' success.

Our experienced team actively participated in pilot programs leading up to the introduction of the UK Combined Review Service and the EU Clinical Trial Information Systems. Now, with the EU Clinical Trial Regulation in full effect, ProPharma continues to stand as a reliable partner, guiding clients through this transition phase. In 2023, we proudly achieved numerous initial clinical trial application approvals for our clients and continue to support additional initial applications through to success.

But our commitment doesn't stop there. We also excel in supporting Additional Member States procedures, managing Substantial and Non-Substantial Modifications, and facilitating Transition applications through CTIS.

With a proven track record that spans clinical trial applications, GMO submissions, and performance study submissions for companion diagnostics and IVDs, we're your trusted partner in achieving regulatory success. European regulatory requirements can be daunting, but our seasoned team possesses the in-depth knowledge and experience to guide you seamlessly through the process. Whether it's the rigorous documentation needed for clinical trial applications, the precision required for GMO submissions, or the specialized expertise necessary for companion diagnostics and IVDs, we've got you covered. At ProPharma, we understand the unique nuances of each submission type and have successfully supported a diverse range of clients across various therapeutic areas. With our comprehensive approach and unwavering commitment to compliance, we ensure that your submissions are not only complete but also optimized for swift approval. 

Orphan Drug Designation

ProPharma is dedicated to assisting clients in their mission to bring critical treatments to patients suffering from rare diseases. Acquiring orphan designation in Europe offers a plethora of incentives to support the development of therapies for these underserved populations, including coveted market exclusivity, reduced fees, and access to invaluable scientific advice.

If you are developing a potential orphan drug, ProPharma is your trusted partner to navigate the EU orphan drug designation process. With our expertise and unwavering dedication, we increase your chances of success in delivering life-changing treatments to those in need. We can accelerate your orphan drug development journey.

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Paediatric Development

In Europe and the United Kingdom, securing Agency agreement on your paediatric development plans is not just a choice—it's mandatory. This critical step is achieved through the submission of a Paediatric Investigation Plan (PIP), and it's essential that each Agency involved agrees to its terms. A PIP may encompass deferrals, permitting the initiation or completion of studies later once additional data becomes available, or it may include mandated measures, potentially granting a waiver from conducting studies in specific paediatric subsets.

Once a PIP is agreed upon, you are firmly committed to executing what has been outlined in the plan. A rigorous compliance check is a prerequisite before the validation of your MAA, ensuring that every detail aligns precisely with the agreed PIP. This meticulous adherence to timelines and obligations is paramount, as any deviations could hinder the validation of your MAA, even for adult filings. Furthermore, if your product is already on the market, a PIP compliance check becomes necessary to demonstrate continued adherence to the plan before any variations for new indications, pharmaceutical forms, or routes of administration can be considered.

If you find yourself in need of submitting a PIP application and are unsure where to begin, whether to submit to the FDA, EMA, or MHRA first, or if you should submit to all simultaneously, ProPharma is your trusted partner. We specialize in planning paediatric strategies and optimizing submission timelines to ensure a globally agreed-upon approach. Over the past five years, we have supported the preparation of more than 40 PIPs, including those submitted to the European Medicines Agency's Paediatric Committee (PDCO) and the MHRA. Our expertise and commitment to regulatory excellence will guide you through the complex landscape of paediatric development, ensuring your strategy aligns with global standards. Contact us today to take the first step towards a successful paediatric development plan.

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Marketing Authorisation Applications (MAA) in Europe

A well-crafted regulatory strategy is the compass that guides you through the intricate journey towards Market Authorisation (MA) approval. ProPharma understands the significance of this strategy and provides a comprehensive roadmap that delineates the steps, timelines, and key milestones required to navigate the complex regulatory landscape successfully. Whether you're pursuing a standard, conditional, exceptional MA, or aspire for an accelerated assessment, these strategic elements are carefully considered.

The pre-submission phase plays a pivotal role in preparing for a European centralized, decentralized, or national MAA. This preparation can commence as early as 18 months before the scheduled MAA submission date. The intricacies involved are multifaceted and vary depending on the procedure, encompassing eligibility assessments, legal entity considerations, enterprise status evaluations, European Medicines Agency (EMA) systems setup and access, orphan maintenance, paediatric compliance, brand name review, prior scientific advice/protocol assistance, risk management plans, environmental risk assessments, supply chain setup, Qualified Person (QP) Pharmacovigilance, and QP + license to import/batch release, among numerous others. In the case of a centralized MAA submission, multiple interactions may occur, including pre-submission meetings with EMA delegates and discussions with the appointed (Co)Rapporteur and teams. All of these activities require meticulous planning, thorough preparation, and precise execution. Parallel discussions may further complicate matters, particularly in the context of conditional approval or accelerated assessment. ProPharma's Regulatory Sciences Team stands ready to support, coordinate, organize, and lead all pre-submission phase activities, ensuring a seamless process.

ProPharma has a proven track record of successfully supporting clients during pre-submission and assessment procedures across a wide range of therapeutic areas, including generics, biosimilars, biotech, and cell & gene therapy (ATMPs) products, having supported over 25 marketing authorisation applications, including the successful approvals for the first gene-therapy medicine in the EU and the first histology-independent treatment for solid tumours in the EU. Our Regulatory Sciences Team comprises a mix of ex-regulators and seasoned industry professionals, and for every project, an experienced Project Lead (Single Point of Contact) is assigned to ensure seamless communication and coordination.

With ProPharma as your regulatory partner, you can navigate the intricate journey to Market Authorization with confidence, knowing that you have a team of experts dedicated to your success. Contact us today to embark on a path to regulatory excellence.

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