In opening the conference, Dr Andrzej Rys, Director for Health Systems and Products, at the European Commission’s directorate general (DG) for health and food safety, argued that EU rules respond to real problems in the industry. He noted that this half century anniversary coincided with the European Medicines Agency’s (EMA) 20th birthday and said that this was ‘a perfect moment to reflect on what has been achieved’.
Delegates were keen to recall how the thalidomide tragedy of the early 1960s had inspired EU legislation, institutions and mechanisms, which established centralised authorisation procedures for the assessment of new pharmaceutical medicines. The EU health and food safety Commissioner Dr Vytenis Andriukaitis told attendees: ‘We cannot think of the future, without thinking of the past.’ And addressing the conference, thalidomide survivor Kevin Donnellon stated that while he did not see his life as a tragedy – he is living a full life in spite of his condition – he recognised the trauma inflicted on his parents, who had to manage and fight for his right to be treated equally.
Reminding authorities of their negligence in letting the thalidomide drug onto the market, Donnellon noted that while UK thalidomide survivors had received some compensation, although not enough in his mind, in some continental European countries survivors had received no compensation and so he would continue to campaign against this injustice.
Regulators, legislators, pharmaceutical and healthcare professionals agreed that a culture of collaboration has to spread throughout the pharmaceutical pipeline, from therapy discovery right through to patient usage
One of the main themes of the conference was the growing importance of collaborative partnerships – both in terms of developing new innovative medicines and assessing new therapeutic drug treatments. Regulators, legislators, pharmaceutical and healthcare professionals agreed that a culture of collaboration has to spread throughout the pharmaceutical pipeline, from therapy discovery right through to patient usage. Where collaborative partnerships exist, these need to be deepened and adapted to respond to the medical, technical and societal changes impacting on the medicine development and approval chain. In certain areas of the medicine product pipeline, however, work still needs to be done to create and nurture a culture of partnership, for instance regarding innovative medicines.
Michel Goldman, Professor at the Université Libre de Bruxelles and former Executive Director of the EU’s Innovative Medicines Initiative (IMI), told attendees that future developments in pharmaceutical regulation ‘must be based on robust science and multi-stakeholder collaboration, such as public/private partnerships (PPPs)’ – approaches which foster inter-disciplinary cooperation. Goldman said he was convinced that the IMI, ‘under the leadership of its recently appointed Executive Director, Dr Pierre Meulien, will continue to embody these principles as we move forward.’
Speakers were in agreement that greater efforts will have to be made to create new partnerships and encourage collaborative programmes if the new challenges around drug therapy developments and innovative medicines are to be met. What was less clear is how these new collaborative partnerships are going to be formed in practice. In which part of the process, from discovery to patient usage, will different stakeholders be involved in collaboration? And how will issues of data protection, intellectual property rights and trade secrets be tackled when so many pharmaceutical sector interests, often with very different agendas, are trying to work together? Creating new models of engagement and involvement in all stages of medicine discovery and assessment will be crucial in the coming years, delegates heard.
Can organisations and institutions fundamentally change their culture and show a greater commitment to co-operation
Giulia Del Brenna, Deputy Head of the cabinet of the EU research, science and innovation Commissioner Carlos Moedas, wondered whether institutions and regulators are willing and ready to work more collaboratively together in this rapidly changing world. Indeed, can organisations and institutions fundamentally change their culture and show a greater commitment to co-operation? Professor Guido Rasi, a former EMA Executive Director who has recently resumed that post, argued that a softly, softly approach would be best: Europe does not need to be pushed into collaboration, as this is inherent to the values of the EU and its organisations, he suggested.
Commenting on the trend towards partnerships, Goldman believed that there is an encouraging trend among healthcare providers to look beyond traditional ways of providing therapeutic options. These new ways would have to take on board the input of all pharmaceutical sector partners, including patients.
Professor Rasi reminded attendees that the European Commission has been at the forefront of fostering co-operation among EU member states. He believes governments will in future have a greater role in helping integrate patients’ perspectives into the medicine approval process, by helping patient groups get seats on assessment panels and by making sure that patients’ voices are heard by healthcare payers. EU regulatory authorities can bring their substantial experience and expertise to bear in terms of creating and nurturing networks, which could incorporate agencies, business, industry and civil society, bringing stakeholders together in a centralised space, both in their individual groupings or across disciplines and fields, he said.
According to IMI boss Dr Meulien, regulators will need to be trained to provide appropriate services responsive to the growing complexity of medicine development. The medical curricula of higher education will also need to be adapted to make it more forward-thinking, claimed the new IMI head.
Martin Seychell, Deputy Director General for Health at the Commission’s DG health and food safety, believes medicine regulators should play a greater role in promoting a more productive environment for the development of new therapies. He believes they also have a responsibility to ensure that pharma regulations remain at their current high standards.
He acknowledged that regulators face a number of challenges in a number of areas, such as orphan and paediatric medicines, which are a growing feature of therapy today. Seychell felt the trend towards personalised medicine needs to be encouraged in the context of the growing importance of personalised healthcare. Pharmaceutical regulations are not specific enough, claimed Seychell; they are not personalised enough but they will have to be when it comes to dealing with areas such as genomes, bio-motions and immune system therapies.
The growing complexities associated with personalised medicines points to a greater complexity and increased number of datasets, all of which will have to be understood and managed by regulators monitoring and approving personalised medicines. As the authorisation process becomes more complex, it will also become more technical. Regulators will have to be better informed to diligently carry out their duties, but also so they can carry out their role of keeping the growing number of networks informed – an increasing part of their remit.
Regulatory systems will have to continue to adapt in response to the increasing complexities of science when it comes to developing innovative therapies
Meanwhile, Professor Klaus Cichutek, President of Germany’s Paul-Ehrlich-Institut, the federal institute for vaccines and biomedicines and chairman of the EU heads of medicines agencies’ management group, feels that regulatory activities will become more specialised. Some regulators will focus on gathering and disseminating information to networks consisting of patient groups, consumers, academics, start-ups, biotechs, and pharma companies. Other regulators will focus on developing the new therapeutic mechanisms and procedures which will be needed to assess new medicines, bringing them more quickly to market while ensuring that as many stakeholders as possible are involved in the product approval process, he told the conference.
Regulatory systems will have to continue to adapt in response to the increasing complexities of science when it comes to developing innovative therapies. They will also have to reflect the fact that regulatory operational issues are now so intricate in their own right that they have become a service.
Despite the growing complexities and the regulators’ changing role, Cichutek felt they will have to retain the capacity to step back and engage with patients and other medicine users and traders in a language which they understand, keeping them informed of regulatory, technical and medical developments.
In addition, the regulators will have to develop processes and systems to gather and cope with these larger data sets, while also dealing with the potential stumbling block around data ownership, he noted. How data is sourced, how it is managed, when it comes from various sources, will indeed become more of an issue, as those developing medicines draw on data from several partners to create a new therapy.
Speakers reflected on a trend for payers and other stakeholders to be more focused on outcome and process, stating that greater attention is being paid to the performance of new medicines, as defined and required by healthcare payers and patients. A deepening of the use of sequencing, which will allow for more individual sequencing, with regard to tackling tumour cells, for example, will lead to more specific and targeted treatments in the future, Cichutek told conference attendees. He said there will be more system-biology with different fields of science, such as microtechnology and gene therapy, contributing to the numerous treatments that will play a greater role in healthcare.
A number of presenters addressed the realisation that if new medicines are not affordable to healthcare payers and patients, then they are not really accessible. If new therapies are not accessible it begs the question: why spend time and money developing them in the first place? Hence the need for new business models in the product development field. These new business models are likely to include contributions from the biotech, nanotech, medical device, hi-tech and IT companies and patient groups, the conference was told.
According to Chas Bountra, Professor of Translational Medicine, and Head of the Structural Genomics Consortium at Oxford University, ‘Big Pharma’ will increasingly focus on product development and big clinical trials and studies, while academics, biotech companies and start-ups will take the lead in product discovery. It is also likely that there will be more examples of biotech clusters transferring data into the pipeline of the larger pharmaceutical companies.
There is a lot of good science being undertaken but it does not always translate into innovative products
French centre-right MEP Françoise Grossetête told attendees there is a need to create a cultural change in the pharmaceutical product development system. She noted that there is a lot of good science being undertaken but it does not always translate into innovative products. She believes greater efforts must be made to incentivise all those within the product development pipeline, including better mechanisms for transferring the innovation of start-ups and hi-tech companies into actual products; creating these innovation pathways needs to be a priority, she said.
And in a world where preventative medicine will come to the fore, patients will expect new tools that will enable them to monitor and manage their own health and seek out appropriate cures when they fall ill. Genomes and the stratification of the different types of diabetes or cancers, for example, will aid such developments.
The general public are going to demand to be better informed of their individual susceptibility to specific diseases and they will also need new tools to provide information on what range of protections are on offer to minimise their susceptibilities, was a recurring message at the conference.
