Innovation in pharmaceutical sciences comes not only from R&D activities run internally by pharma and biotech companies. University labs and innovative, small-size start-up companies are nowadays the main contributors of innovative and early stage technologies, that are developed mainly with the financial support of public funds, i.e. EU funded projects. Only the most promising approaches are also sustained by private capitals, that may assume the form of venture capitals supporting the creation of a start-up or see the direct contribution of a pharmaceutical company by mean of a strategic collaboration agreement. Furthermore, current models of pharmaceutical development approach the planning of the regulatory strategy at a very early phase in order to optimise study design, data collection and, consequently, time-to-market. This is putting a major risk on activities run into the academic contests, where regulatory knowledge might be insufficient to properly address all issues and requirements needed to obtain results from clinical studies that are truly suited to be used for regulatory purposes.
A specialised Office of the AEMPS that follows the European model
Spain is characterised by a very pro-active R&D sector in life sciences, including for example one of the main biotech research district in Europe, the BioRegion Cataluña around Barcelona. To sustain the integration and coordination of different activities targeted to clinical research and run by academic centres, independent investigators, cooperative groups, hospitals, research foundations or companies, in 2016 the Spanish Agency of Medicines and Medical Devices (AEMPS) created the Spanish Office for Support of Innovation and Knowledge of Medicinal Products (OSIKMP).
The approach to promote innovation used by the Spanish authority follows examples at the European level, such as the PRIME scheme (PRIority Medicines) and the EMA’s Innovation Task force (ITF). The first one is managed by the European Medicines Agency and supports the development of medicines for unmet medical need. Companies may adhere to the PRIME scheme under a voluntary base to access enhanced interaction and early dialogue with the regulatory agency. Scientific advice and accelerated assessment are at the core of PRIME’s activities, as well as they are central to the Spanish OSIKMP. Up to February 22nd, 2018 (date of the last report published by EMA), the CHMP granted 35 positive recommendations on PRIME eligibility, denied other 128 requests (plus 7 out-of-scope ones). Oncology and haematology are the main therapeutic areas interested by the granted requests of access to the program (12 and 7 projects, respectively).
EMA’s Innovation Task Force (ITF) is a multidisciplinary group that includes scientific, regulatory and legal competences created to provide early dialogue with applicants – particularly SMEs and academic institutions – on innovative aspects in medicines development. It evaluates the impact of emerging therapies and technologies, identifies the need for specialised expertise at an early stage and supports other Committees of the Agency in decisions regarding eligibility to procedures, borderline products and emerging therapies and technologies. The ITF also collaborates with the Innovation offices of the various national regulatory agencies, under the mandate of the EU-Innovation Network of October 2016.
The activities run by the OSIKMP
The Office for Support of Innovation and Knowledge of Medicinal Products offers to interested parties a 360° degrees collaboration – under a global perspective – to enhance the possibility of success of the technologies and products under development. The first of its four main goals is to favour the integration of its services into the wider structure of the European Union, something essential to guarantee a smooth transition to EU’s centrally managed regulatory procedures. R&D activities are also supported by scientific advice tailored on the specific needs of the applicants and products, from discovery along the entire life cycle of a new medicinal product. Pre-access activities are intended to favour the production of high quality data from clinical trials and to ensure the availability of compassionate use schemes and registries. After authorisation, parallel advices is integrated with HTA at the national level, and therapeutic positioning reports – or the recommendations of access under different conditions from those authorised – are developed together with Spain’s Autonomous Communities.
Information and access to the services offered by the OSIKMP can be requested very simply by sending an e-mail to the address INNOV@aemps.es. The Office covers any kind of medicinal product at the initial stage of development. The Office uses various modalities to interact with partners, from face-to-face meetings to individual teleconferences, from simple consultations to collective sessions or courses. During this phase of the consultancy the adhering partner should be able to reach a better understanding of the degree of innovation of its projects, acquiring deeper knowledge of the most appropriate regulatory tools available and how to integrate the regulatory development into the European system for authorisation, or using other support tools at a national level.
The Independent Clinical Research Support Office of the AEMPS provides information and support to plan and perform non-commercial clinical trials. The Office facilitates the interaction of the investigators with the regulatory authority and provides regulatory or scientific support for the application, authorisation or classification of a clinical trial. The OSIKMP allows also to access the AEMPS’s national scientific advice unit, providing opinions on the general development of medicinal products or studies designed to improve knowledge of a medicinal product once authorised. The unit is also the point of contact to access the EMA’s procedure of scientific advice.