Interview with Richard Bergström: The merging of biology with digital technologies is personalising healthcare
27th May 2015
From gene sequencing in target discovery, to patient self-monitoring with mobile apps, digital platforms are transforming healthcare. EFPIA’s members are applying these tools to develop better, safer, targeted medicines. And by reducing uncertainty for payers this movement will improve access and affordability, says Richard Bergström
The convergence of biology and information technology is proceeding apace and the ripples are now being felt across the whole chain of healthcare, from sequencing and high throughput screening in drug discovery, patient selection, recruitment and monitoring in clinical development, real world evidence collection for health technology assessment and on to supply chain monitoring to combat counterfeiting and pharmacovigilance reporting of adverse events.
A range of technologies and services are being deployed around digital health. New start-ups are being formed and established search engine, smart phone, software and electronics giants – with no previous interest in health – are entering the market. In their wake is a profusion of new terms: digital health, precision medicine, the consumerisation of healthcare.
There may be more noise than signal currently, but at its heart this movement is about using the power of digital tools to look into individual biology as the basis of personalised health care. “There are different flavours here, but for EFPIA’s member companies the essence lies in building a deep knowledge of the biology underlying a disease and finding gene or protein biomarkers that can form the basis of prognostic tests, to ensure the right patient gets the right therapy,” says Richard Bergström, Director General of EFPIA.
Personalising health care in this way is not only of benefit to patients, “It means drugs arrive with the evidence needed for health technology assessment and gives payers a sense of how a product will perform in their health system,” Mr Bergström says.
Broadening personalised healthcare beyond cancer
Oncology is the field in which development of drugs targeted at specific tumour mutations or receptors is most advanced, with more and more treatments having an associated diagnostic.
“This is an important start, but much more research is needed to increase the number of targeted treatments in cancer, and also broaden personalised health care out into other disease areas, in particular common, chronic diseases,” says Mr Bergström.
In 2000 it was promised that the sequencing of the human genome would lead to a new generation of targeted drugs. But in common diseases where there is an array of genetic risk factors, and diet, lifestyle and other environmental factors play such a large role, it has proved hard to single out the significant underlying genetic factors.
“Now however, next generation sequencing technology is becoming low-cost and sensitive enough to be applied at the requisite scale,” Mr Bergström noted. Using digital technology to compare the DNA of patients with confirmed diagnoses will make it possible to pinpoint relevant genes. This highlights the value of EU-supported biobanks, which provide access to consented DNA samples from patients in different member states.
In general, powering phase III trials in common chronic diseases requires large numbers of patients to be treated and monitored over long time periods. Getting a better handle on the genetics could change this, Mr Bergström believes. “Armed with genetic markers, it will be possible to select those patients that will respond and it will also be possible to identify patients at higher risk of suffering a sudden worsening of their condition – a heart attack, or renal failure for example – making it possible to show efficacy in smaller and shorter trials.”
Pharmaceutical companies are increasingly taking a personalised healthcare approach from the beginning of drug discovery and across development. There are also examples of a personalised approach being retrofitted, when subgroup analyses of clinical trial results pointed to a group of patients that benefited, even though the trial as a whole did not show statistical significance. In fact most drugs that have been approved to date for such targeted patient groups, have been developed this way.
The digital platforms underpinning personalisation are also being applied to boost productivity in ongoing clinical programmes, to provide evidence to support regulatory and payer decision making.
“This is not something that the industry can do unilaterally,” Mr Bergström noted. “EFPIA members are involved in a number of projects and pilots, both at a national level, and through the EU’s Innovative Medicines Initiative, to develop new methodologies, digital tools and processes, that are laying the foundation for adaptive pathways.”
The aim is to move from the current approach of approving products only after large, expensive and lengthy phase III trials, to granting early approvals in a subgroup of patients and allowing the label to be expanded if the real world evidence warrants it.
Real world evidence
EFPIA has set up a Medicines Adaptive Pathways to Patients special interest group as a forum for member companies to share ideas, discuss progress and consider how to implement the post-approval monitoring and collection of outcomes data that adaptive pathways call for.
One IMI project of relevance here is the €16.3 million GetReal, which is working to develop new approaches for incorporating real world evidence. “It is hoped this will inform decision making in development, and provide evidence for regulators and payers, to speed market access,” says Mr Bergström.
EFPIA is also the driving force behind one of the largest and most ambitious digital health platforms, the European Medicines Verification System, which is designed to prevent counterfeiting and theft, and promote patient safety, by verifying each pack of medicine across the supply chain from manufacturing to dispensing.
“The system is a huge investment and it will generate an enormous volume of information. We think it may be possible to use this in support of a real world evidence approach,” Mr Bergström says.
Patients own their health data
The most disruptive digital platform of all, and the one that perhaps most clearly has the potential to increase accessibility and affordability, is mobile health. This bottom-up movement has seen users across Europe downloading thousands of health and wellness apps. Mobile phones are doubling up as devices for diagnosing and monitoring disease.
“Undoubtedly, the potential is enormous,” says Mr Bergström. “Patients can have ownership of their health data, promoting self-management. It will be possible to link adherence to outcomes, plug the gaps in formal health records, and to truly put patients at the centre of our healthcare systems.”
The EU has estimated that there could be data-fuelled health savings of close to €100 billion. EFPIA is contributing to the EU’s work to establish an industry-led code of conduct and provide a framework for the deployment of mHealth, in order to help capture this potential.