Regulatory Challenges

It is generally accepted that vaccines designed to prevent infectious diseases are one of the most cost-effective interventions in public health and have prevented illness and death from a variety of diseases. Yet, there remain many infectious diseases that have an important medical impact for which safe and effective vaccines remain elusive. At the same time, there are infectious diseases for which vaccines are already available, but for which it is known that the efficacy should be further improved. The development of innovative vaccines will require innovative technologies that are inherently complex and whose development is both expensive and risky. For several of these innovative vaccines it is expected that demonstration of efficacy through classical randomised clinical trials will be very difficult if not unfeasible. Regional aspects that have to be taken into account during vaccines development add further complexity and challenges. The EU environment brings a series of specific challenges (e.g. regulatory decision making, decision making on reimbursement and pricing) that are not conducive for vaccine development. Therefore, there is a risk that new and innovative vaccines for which there is a real medical need will not be developed and available to the EU citizens. In general regulatory requirements are further increasing, which drives up both the cost and time required to authorise new vaccines. In addition, since the full benefit/risk profile of a vaccine can be assessed only once it gets used, large amounts of real time safety and effectiveness data are key, resulting in a substantial increase in costs associated to the development. Generating such data greatly depends on external networks and organisations. At present there is only limited support from EU and country governmental institutions to help the generation of such real time data. Since for some innovative vaccines it will not be feasible to generate the type of evidence that regulators currently are expecting to have available even prior to authorisation, it will be critical to improve the collaborations that are needed to generate robust data-packages in the post-approval phase.

Another challenge is that the data generated to support the marketing authorisation of the vaccines is not necessarily in line with the data that recommending bodies/payers in the different EU member states want to have available prior to decision making. However, in order to commit to the effort to develop a new vaccine, there need to be clear pathways to ensure that once successfully developed and authorised by regulators innovative vaccines will be recommended and used. A mechanism is needed by which exchange and interaction across stakeholders early in development is possible, to ensure that precious resources are not expended on development activities for vaccines for which the likelihood of approval, recommendation and implementation would be low. In the EU, an active debate is on-going on adaptive pathways for medicinal products, aiming to bring important medicinal products earlier to the patients. The time could be ripe to discuss whether adaptive pathways also need to be developed for vaccines in order to ensure that new innovative vaccines will become available for the public. Key stakeholders involved in vaccine development, registration, recommendation and implementation should get together to design and put in place new mechanisms that build on the latest progress of science and technology to support sustainable vaccine innovation in EU while keeping vaccine safety, efficacy and quality at the core of all activities. Importantly, the non-conducive environment in the EU is also hampering the development of vaccines in the rest of the world, as the EU today still takes a leading position and is seen for many countries as a reference worldwide.