Expertises
WE LOVE, LIVE AND BREATHE
REGULATORY AFFAIRS
Eventually, successful drug development and life cycle management pass through the gatekeeping function that regulatory authorities have to protect individual and public health. PhaRA’s experts will develop with you a sound regulatory strategy to support drug development and life cycle management and will advise you on interactions with the several relevant regulatory authorities in the EU, prepare, and accompany you for such interactions.
Developing a strategic regulatory roadmap for the development and successful registration of medicinal products requires in-depth understanding of the complex EU regulatory environment. At PhaRA we can support the development of the target label and draft a sound regulatory strategy plan, optimising the use of the various incentives to drug development in the EU and the UK and possible early access tools such as accelerated review and conditional and exceptional circumstances Marketing Authorisations (MAs). We will consider the need to seek scientific advice or protocol assistance, the opportunity to obtain orphan drug designation, potential eligibility for the PRIME scheme, and compliance with the paediatric regulation. We advise you on the appropriate legal basis for your Marketing Authorisation Application (MAA) and the recommended filing route in the EU (CP, DCP, MRP, NP) and the UK.
Based on detailed assessment of available data on the compound, including HA correspondence from all geographies, and the understanding of the target product profile, PhaRA can perform a GAP analysis and present scenarios and options with pro’s and con’s leading to a recommended strategic approach to present to Health Authorities.
Early-phase development is one of the spearheads of the Federal Agency for Medicines and Health Products (FAMHP) in Belgium. PhaRA can provide regulatory advice for early-phase development projects, guide the risk assessment for First-in-Human (FIH), support the overall editing of Investigator’s Brochures (IB), compile and submit clinical trial applications for FIH and early phase clinical studies in compliance with regulatory requirements (see ‘Clinical trial applications’). We have experience with pre-submission meetings, National Scientific-Technical Advice (STA) requests, and Simultaneous National Scientific Advice (SNSA) to seek advice on specific trial related questions.
The paediatric regulation came into force in the EU on January 26, 2007. All European marketing authorisation applications for new medicines, which are not authorised in the EU prior to the implementation date, have to include the results of studies carried out in children of different ages, unless a deferral or a waiver has been granted. The Paediatric Committee (PDCO) determines what these studies must be and describes these in Paediatric Investigation Plans (PIPs). The timely submission of a PIP and a successful PIP procedure are therefore of key importance for the filing of both adult and paediatric applications in the EU. The paediatric obligation also applies when a company wants to add a new indication, pharmaceutical form or route of administration for a medicine that has already been authorised and patented.PhaRA has first-hand experience with the development and submission of Waivers, initial PIPs, Modifications to agreed PIPs, Compliance checks, and PDCO interactions. Post-Brexit, PhaRA can also help companies submit their UK-PIP through the dedicated MHRA Submissions portal.
PhaRA supports companies to understand the orphan drug legal framework in the EU and to obtain orphan drug designation for medicines for rare diseases. PhaRA has extensive experience with the preparation and submission of the Orphan Drug applications and post-designation activities, including annual reports. PhaRA can advise on orphan related opportunities, such as protocol assistance, fee reductions, market exclusivity, and potential for conditional MA.
On your behalf, we liaise with the European Medicines Agency (EMA), Rapporteur, Reference Member State (RMS), Concerned Member States (CMS), and national agencies. Over the past years, we have established good contacts with competent authorities in several EU countries, and at the EMA.
PhaRA will present options for early dialogue/consultation with regulatory agencies (eg, ITF, national and/or EMA scientific advice, PRIME, PreIND etc), in context of ad hoc early scientific (quality, nonclinical, clinical) development questions or in view of Phase I-II trial start, and/or regulatory or legal interactions
Scientific advice or protocol assistance can be of great value for drug development, however, its preparation demands an intense effort from the project team. Whether advice is obtained from the EMA, at a national level or as part of the simultaneous national scientific advice, it requires profound knowledge of the procedure, careful planning, strategic thinking, and convincing writing skills to complete the process successfully. We offer our skills and knowledge to coach the client team, to help prepare high-quality briefing books, to prepare the team for meetings with the EMA/SAWP or national agencies, or to attend them on your behalf.
The Priority Medicines (PRIME) scheme can be of value for medicines targeting an unmet medical need. This voluntary scheme run by the EMA offers advantages such as enhanced interaction and early dialogue between the Agency and developer in order to optimise the development plans and ultimately enable medicines to reach patients earlier. Our experts have the relevant skills and knowledge to support companies in analysing the potential suitability of their development programs for PRIME as well as support subsequent submission related to the PRIME scheme including the initial eligibility request, kick-off meeting, subsequent scientific advices, submission readiness meeting, etc. PhaRA can also help ensure client teams are prepared for these procedures and relevant interaction meetings with EMA.
The innovative licensing and adaptive pathway (ILAP) is a novel collaborative scheme aimed at supporting innovative approaches and development programs to improve patients access in the UK. Similar to the PRIME scheme in the EU, PhaRA can advise and support companies considering applying for ILAP for their development programs. With the knowledge of our experts, we help companies successfully navigate UK requirements and ILAP processes including initial eligibility confirmation and innovation passport application, as well as further support development of a target development profile and roadmap together with relevant stakeholders.
Pre-Investigational New Drug (IND) and INTERACT meetings can be of great value to sponsors to gain advice from FDA on their drug development program ahead of submitting an IND application. Pre-IND meetings provide a platform for sponsors to discuss key questions related to regulatory requirements for their development program and provide an opportunity to sponsors to better understand the FDA’s data requirement expectations. INTERACT meetings are intended to facilitate IND-enabling efforts for sponsors of novel products that are facing unique challenges early on in the development (prior to pre-IND) where absence of early FDA advice could delay the product’s entry into the clinic. Preparing for a pre-IND or INTERACT meeting requires understanding of the procedure, careful planning in terms of timing, strategic thinking in terms of proposed questions, and convincing writing skills to maximize the benefit from completing this process successfully. PhaRA can support to sponsors throughout this process, by offering services to help with the preparation of high-quality briefing packages, preparation of the team for meetings with FDA, or by attending them on your behalf.
In addition to a sound regulatory strategy, knowledge of relevant regulatory procedures and applications is critical when determining the success of a drug development program. Here at PhaRA, our experts can help support you in navigating these different procedures and applications throughout the development of your product, ranging from early phase activities to the preparation of a marketing authorisation application and lifecycle management of your product.
The implementation of the EU Clinical Trials Regulation (CTR) has significantly streamlined the process for obtaining clinical trial approval across EU Member States. Instead of submitting separate applications to national competent authorities and ethics committees in Member State, sponsors now benefit from a centralized process via the Clinical Trials Information System (CTIS) and ‘one single decision’ per member state will be provided within the timelines provided in the regulation. Belgium continues to offer a favourable environment for early-phase clinical trials. The FAMHP remains committed to fast-track evaluations, aiming to deliver decisions within 20 days for mono-national Phase I clinical trials.
PhaRA offers support throughout the clinical trial life-cycle and the submission process. Our team assists companies with the preparation of the Investigational Medicinal Product Dossier (IMPD), including high-quality Quality IMPD writing, writing of the clinical study protocol and Investigator’s Brochure, as well as the compilation of Clinical Trial Applications (CTAs) in full compliance with scientific and regulatory requirements, including the redaction of clinical trial documents. PhaRA can also help to develop documents in lay language, such as ICFs, protocol and CSR synopsis. We provide support on CTA submissions via CTIS and end-to-end follow-up throughout the process. In addition, PhaRA brings extensive experience in strategic planning for pivotal trial start-up and coordination of global clinical trials, supporting sponsors in the efficient execution of complex development programs.
Companies can consult the European Medicines Agency (EMA) to determine whether a medicine they are developing is agene therapy, somatic cell therapy, or tissue-engineered product. The procedure allows them to receive confirmation that a medicine, which is based on genes, cells or tissues, meets the scientific criteria for defining an ATMP. This service is particularly valuable for companies seeking regulatory clarity, risk mitigation, and strategic planning in the development of innovative therapies.
The European Medicines Agency's Committee for Advanced Therapies (CAT) provides a certification procedure for advanced therapy medicinal products (ATMPs) under development by micro-, small- and medium-sized enterprises (SMEs). The certification procedure involves the scientific evaluation of quality data and, when available, non-clinical data that SMEs have generated at any stage of the ATMP development process. It aims to identify any potential issues early on, so that these can be addressed prior to the submission of a marketing-authorisation application.
Preparing and successfully filing a Marketing Authorisation Application (MAA) is a major achievement for which external staff are often required to cope with peak workloads. We offer you our expertise at an EU and national BENELUX level and extra hands to assist the team in this major exercise and guide them through the submission and approval process. We review submission components, help compiling the application and respond to questions. We are able to run your regulatory procedure, or support specific steps in the process (e.g. handle BENELUX translations during the CP linguistic process or manage the administrative closing during the national phase of MRP/DCP). We have experience with all types of MAAs and EU procedures (Centralised, Decentralised, Mutual Recognition, or National Procedures), including preparation and participation in pre-submission meetings, oral explanations or discussion meetings. We have also been involved in re-examination procedures and Referrals.
Our BENELUX services and expertise cover submission and maintenance at national level of MAA for Rx and OTC products, notifications for borderline products, compassionate use and medical need programs, support to RMA and DHCP letters, review of advertising and promotional materials, including local RIP services in Belgium. PhaRA is the selected provider of the Clarivate BELUX regulatory intelligence services.
In order to manage a product’s lifecycle in a competitive market place and environment of continuous scientific and technical progress, the marketing authorisation holders (MAHs) are faced with the need to submit Renewals, Variations, Line Extensions, Label changes, license transfers, switches of legal status, DHCP-letters, and other regulatory maintenance responsibilities. PhaRA can help MAHs to cope with these regulatory challenges and administrative burdens.
PhaRA also provides expert due diligence and divestiture services to support clients during mergers, acquisitions, portfolio reviews, and product transfers. Our team conducts thorough regulatory assessments to evaluate the compliance status, risks, and opportunities of products or product lines under consideration. We assist in preparing regulatory documentation for licensing deals or asset transfers, and help manage the regulatory aspects of divestitures, including variations, license transfers, and communications with health authorities. With our deep regulatory knowledge and experience in cross-border transactions, PhaRA ensures a smooth and compliant transition throughout the entire process.
Dossiers submitted to regulatory authorities include numerous scientific documents written in English. Well-written documents contribute to the success of a submission. Our experts ensure that your documents are lean, in line with the applicable legislation, and fit for purpose (e.g. target audience: submission to authorities or sharing information with the public).
PhaRA can support companies with the writing of high-quality CMC documents for their products in line with applicable CMC requirements. CMC writing support can be provided for quality related documents, including Module 3, throughout a product’s lifecycle including for clinical trial applications (IND/CTA), initial MAA/BLA submissions as well as lifecycle management submissions such as variations.
Not only are PhaRA’s medical writers trained in good writing and reviewing practices, they are also aware of relevant legislation that should be considered when delivering high-quality documents in a timely manner. PhaRA can help support companies with a wide variety of documents including clinical trial level documents such as investigator’s brochures, protocol and protocol synopsis, etc. as well as other documents including DSURs, RMPs, clinical evaluation reports for medical devices, etc.
Transparency is becoming a necessity not only due to recent EU legislation but also more generally due to the wish to better inform the public (patients and family), a stakeholder who is requesting more information about clinical trials. PhaRA can support the writing of documents in lay languages thanks to reader-friendly templates and lay translation processes. This includes the writing of ICFs and protocol synopsis in lay languages, before the start of the trial, to facilitate patient recruitment and engagement and the development of a lay summary linked to the CSR finalization in line with EU regulatory requirements. Further, as regulatory strategic partner, PhaRA can support the anonymization/redaction of your documents and their posting to the relevant registries (e.g. EudraCT, CTIS, other registries).
Our experts have developed skills allowing for the accurate presentation of scientific data in a regulatory context. Our focus is on the development of Module 2 regulatory documents and strategic documents including Paediatric Investigation Plans and product specific waivers, Orphan Drug Applications, and Scientific Advice packages.
The product information(summary of product characteristics/SmPC, labelling and package information leaflet/PIL) provides the essential information for the physician, pharmacist, and patient to ensure the proper use of medicines. The PI needs prior approval by the competent authority as part of the MAA/NDA/BLA and needs to be kept up to date on an ongoing basis. PhaRA can support companies as of the initial development of the target label, draft/update the PI compliant with European and national regulatory requirements, handle QRD conversions, provide high-quality translations in all BENELUX languages, and organise translations for the other EU languages. We can also coordinate artwork and readability testing, and can be instrumental for compliance checks and proofreading.
PhaRA has experience in supporting global CMC development plans with specific focus on EU regulatory requirements from both a procedural and strategic perspective. Our experts have a sound knowledge of applicable CMC requirements and can help ensure these are adhered to. CMC expertise at PhaRA has been gained across various product classes (small molecules, mAb, vaccines, ATMPs, other biologics, ADCs and combination products), disease areas and development stages.
PhaRA can help companies optimize their internal processes in order to enhance organizational efficiency and effectiveness. This support includes help with the development of new procedures and procedural documents, as well as the evaluation of existing procedures/documents, and other tailored support and advice.