Experience

Our people have experience from rare diseases to blockbuster indications and from first into man to marketing success.
Here is a selection of some key successes of interest

Challenge:

Medical Devices in USA that are classified differently in Europe

Solution:

It is vital to validate correct classification early in healthcare product development, in order to avoid expensive problems later on. A US company is developing an innovative measurement of kidney function using an injectable constituent together with an external device to measure the blood concentration of the injected component. This product is classified as a device in the USA. Members of our team, together with the client, took this to the EMA Innovation Task Force and confirmed that the injectable component was a medicinal product in Europe, whilst the external measuring device was a medical device. SME status allowed cost-effective scientific advice from the EMA and a robust pathway to approval was created. European expert panel meetings helped develop the strategy, whilst creating advocates for research and regulatory negotiations. Worldwide development is proceeding. Our team members have also been involved in similar strategic solutions for a microbubble with external ultrasound for thrombotic stroke and an autologous tissue product in neuropathic bladder. 

Challenge:

Rescue study in ICU medicine

Solution:

A US based company was developing an intravenous product for severely ill patients in an Intensive Care environment. This is a challenging type of study as patients need to be identified rapidly and enrolled into the study within a short space of time. The study was proceeding poorly in terms of the recruitment and retention of patientsThe client asked members of our team to intervene and assist due to our experience with this type of study.  The timewindow to identify, recruit and treat eligible subjects is very short in some ICU studies and subjects can present at any time (day or night).  This type of study requires rigorous identification of appropriate investigational sites, strong relationships with each site, very tight management of the recruitment process, provision of appropriate medical monitoring (24 hours a day) and the meticulous completion of study records. The study was taken over from the original CRO and completed successfully. A member of our team was involved in regulatory strategy and negotiations with the EMA and the product is now authorised in Europe as well as the USA. 

Challenge:

Navigating regulatory submissions for blockbuster approvals

Solution:

A member of our team has been involved in studies and regulatory negotiations for some of the world’s largest selling drugs. This included pravastatin (Pravachol), omeprazole (Losec/Prilosec) and rabeprazole (Pariet/Aciphex), for which our team member was instrumental in gaining the world’s first authorisation for prn (when required) use of a Proton Pump Inhibitor. 

Challenge:

Navigating trial timelines in difficult disease areas

Solution:

A US based company is developing a product for Myelodysplastic Syndrome (MDS) after failure of a hypomethylating agent (HMA). Recruitment was extremely difficult due to the rarity of the condition and competing studies. The trial was running behind time and over budget. A takeover was instituted and the study is now complete and awaiting its pivotal data analysis. 

Challenge:

Design and implementation of worldwide PV & Medical Information systems

Solution:

A member of our team has designed and overseen the implementation of European and worldwide Pharmacovigilance/vigilance systems for a Chinese generic company, a US based surgical device company and a US based oncology company. This has involved set up and training of teams from Japan to the USA, and careful integration with Medical Information systems. 

Challenge:

Salvage of a failed EU Orphan Designation Application

Solution:

The approach of the European COMP is often different from that of the FDA, for example with regard to indication subclassification. A US based company was advised by a regulatory consultancy that they could adapt their successful US Orphan Designation application for late stage bladder cancer to use for in an application for Orphan Designation in Europe. This attempt was unsuccessful. The European COMP is concerned that simply targeting a stage for orphan product indications may lead to more widespread use than the target indication. Members of our team oversaw the recreation of the Orphan Designation application for Europe, this time targeting a specific histological subtype. This application was successful as an argument can be made that the orphan drug would have a greater effect in this subtype than others. 

Challenge:

Set Up, Implementation and Management of Pharmacovigilance and Medical Information Systems for a Generic Marketing Authorisation Holder

Solution:

A new generic Marketing Authorisation Holder was established in the UK; part of a global group of generic companies. A member of our team was appointed as the EU Qualified Person for Pharmacovigilance (QPPV) for the group and was responsible for establishing the PV and Medical Information departments and systems at the UK office. The EU QPPV designed and implemented the systems and prepared the required Standard Operating Procedures. The department was initially two people (including the EU QPPV), conducting all PV and Medical Information activities and this grew to ten people under the EU QPPV’s management. The EU QPPV successfully collated the Detailed Description of Pharmacovigilance Systems (DDPS) – this was a challenge for several reasons, including the complexity and number of agreements with parties such as co-licensees, distributors and own label suppliers, and the number of licenses held by the group worldwide. After the group was acquired by a US company, the EU QPPV was a key member of the Global Pharmacovigilance Integration team, providing input from an EU perspective and ensuring that the requirements of EU legislation were met by all new systems and procedures put in place. 

Challenge:

Obtaining EU centralised marketing approval under exceptional circumstances for a recombinant fusion protein (interleukin inhibitor) for the treatment of a rare disease with orphan designation

Solution:

This project was managed in its entirety from initial interactions with the EMA regarding eligibly for the centralised procedure, including a pre-submission meeting through to granting of the marketing approval (CHMP opinion and EC Decision). The regulatory team compiled the EU dossier (Module 1 to 5) and managed the eCTD publishing and submission process. The regulatory project manager was the main point of contact, liaising with the EMA and the Applicant throughout the entire review process, managing the responses to Day 120 and Day 180 questions and co-ordinating and reporting the user testing on the readability of the patient information leaflet. Post CHMP opinion the project manager was responsible for managing the translation of the product information annexes and providing to EMA for linguistic review, interacting and liaising with EU Member States as required. 

Challenge:

Successful transfer of a MA from an approved MAH to a new MAH following a commercial decision to divest the MA

Solution:

This project started with the compilation of the MAH transfer documentation package with the reguatory afffairs team liaising with the clients regulatory, manufacturing and pharmacovigilance teams. Advice and guidance was provided on choosing an optimum implementation date for the transfer and which activities were to be completed in an agreed transition period (i.e. batch release, adverse event reporting).

Challenge:

Provision of All Post-Authorisation PV Activities, including EU QPPV. Implementation of 2012 PV Legislation.

Solution:

An Australian company requested full PV services to support one product authorised in the EU, including provision of the EU QPPV. The appointed EU QPPV managed the project in its entirety and provided all PV services requested by the MAH. Following review of existing Standard Operating Procedures (SOPs), a new over-arching PV SOP was prepared in line with 2012 PV legislation. A Pharmacovigilance System Master File was prepared and registered in the XEVMPD, and regularly updated to ensure compliance with PV legislation. A worldwide network of local PV contacts/ QPPVs was established from a contracted experts and existing partners of the MAH. Safety Data Exchange Agreements put in place with all parties, including distributors, and a PV training program was established, to ensure that all adverse drug reactions occurring worldwide were captured.


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