Envestia is among the most experienced regulatory consultancies in this area, having participated in more than 100 PIP procedures under Articles 7, 8 and 30 of the Paediatric Regulation. These projects have included full plans, complete waivers, modifications to agreed plans and re-examinations. This has brought us considerable experience not only with written submissions, but also with PDCO teleconferences and oral hearings. Products concerned have included small molecules, therapeutic proteins, advanced therapy medicinal products (both gene therapy and tissue engineered products), radionuclides and imaging agents. Our comprehensive PIP services include:

• Identification of PIP condition according to EMA guidance.

• Strategy development across all relevant age groups.

• Advice on product-specific waivers.

• Advice on design of individual studies.

• Advice on deferral strategy.

• Preparation of all necessary documentation.

• Submission to EMA and PDCO members.

• Ongoing liaison with EMA.

• Preparation of responses to PDCO Requests for Modification.

• Support to oral hearings.

• Preparation of subsequent requests to modify an agreed PIP.

• Support to compliance check applications.

As a natural extension to these services, we are also able to prepare PSP applications for submission to FDA.

Achieving orphan drug designation can be a key milestone, especially for smaller companies. Having prepared and submitted and subsequently supported more than 40 orphan designation applications, Envestia is well-placed to help with both the strategic and practical aspects:

• Assessment of data package suitability, especially regarding medical plausibility.

• Application preparation (including prevalence calculations and strategy for demonstration of significant benefit).

• Publishing and submitting the application.

• Downstream support and regulatory liaison.

• Conversion of EU applications for submission in the US and vice-versa.

We can also act as the sponsor for the application and/or the principal point of contact with EMA, support protocol assistance procedures and ensure that your designation is appropriately maintained.

Clinical study design is a significant challenge when patients are scarce. We will help to design clinical development plans that are realistic whilst meeting the regulatory requirements.

The outcome of scientific advice procedures is critical in determining future drug development strategy, costs and timelines. Proper preparation is crucial. Envestia has experience with EMA and numerous national competent authorities in Europe.

We will help you maximise the value of scientific advice by:

•Identifying key issues.

•Developing and refining appropriate questions.

•Preparing optimal company position statements.

•Writing briefing packs that are specifically focused on the topics of interest.

•Taking the lead in meeting rehearsals and debriefings.

We can also act as your key contact point with the regulatory authorities, ensuring smooth and efficient liaison throughout. .

The preparation of clinical dossiers to support marketing authorisation applications is a significant proportion of our workload. We have prepared approximately 100 clinical overviews (Module 2.5) and more than 50 clinical written summaries (Module 2.7). These projects encompass everything from the preparation of the first clinical dossier for a new compound through supplementary packages for new indications / pharmaceutical forms to the simplest of generic applications.

The preparation of robust responses to regulatory authority questions is of paramount importance, regardless of the context. We have supported this process within our areas of expertise for more than 40 marketing authorisation applications plus the wide range of paediatric investigation plans, orphan designation applications and scientific advice procedures with which we have been involved. Our philosophy is that good responses must (a) directly address the question asked, (b) contain the right data and (c) be presented using clear, concise language supported with appropriate tables and figures.

We place great emphasis on the value of a clear storyline within each individual question and throughout the response package as a whole.