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Big opportunities in big data, but EMA and industry just getting started

250m genomes sequenced, but data trapped in silos

big data

The potential of big data to transform drug development and regulation is now established in biopharma, but Europe’s regulators and industry agree there is a long way to go to make this vision a reality.

That’s because while there has been an explosion in volumes and types of data being generated, a lack of common standards and regulations means these aren’t yet being fully exploited to streamline drug approval or monitoring.

The heads of all the national EU medicines agencies (HMA) and the centralised European Medicines Agency (EMA) have just released a report from a joint big data taskforce, which sets out a roadmap to unlock the potential of big data.

Big data encompasses a broad range of non-clinical trial sources, from emerging ‘omics’ (genomics, proteomics etc) as well as electronic patient records, real world studies and patient-generated data from wearables and mobile health apps.

However, there are lots of barriers to overcome if big data is to meet its potential: there are currently no standards for how to agree data quality, or how to harmonise datasets or work within data privacy regulations.

The report gives one eye-catching example: around 250m genomes of individuals have been sequenced, but few are well structured, and "much of the data is siloed by disease, institution and country, generated with different methodologies, analysed by non-standardised software, and often stored in incompatible file formats and consequently only a small percentage is linked".

The HMA and EMA are looking for a way for the European Union to move forward on these issues with pharma, biotech and medical devices industries, although their report (two years in the making) also acknowledges there is a current lack of expertise within national agencies to handle and analyse big data.

Nikolai Brun

Co-chair Nikolai Brun

The task force is made up of representatives from the national medical agencies from Germany, Denmark, Estonia, Finland, Hungary, Ireland the Netherlands and the UK, and the wider EMA. Its co-chairs are Nikolai Brun, chief medical officer from Denmark’s DKMA regulator, and the EMA’s principal scientific administrator Alison Cave.

The report, published on Friday, sets out the scale of the challenge:

“Much may be gained from the rational use of Big Data in a regulatory context for approval and monitoring of efficacy/effectiveness and safety of medicines, medical devices and combinations thereof.

“AI technologies offer particularly promising advances in these fields. It is however clear that without a systematic, co-ordinated and integrated European approach many of these advantages may not be gained."

It continues: “Challenges of great complexity remain to be solved particularly regarding data access, transfer, interoperability and data quality as outlined by the respective subgroups.”

The report adds that the timescale required for this change will be long, and will need “continual iteration and reconsideration” as new technology and other considerations emerge.

The report says big data represents a huge shift away from when only pharma companies and regulators held data relevant to medicines and patient health. Relations will have to broaden out to national institutions which hold genomic data, big data giants such as Google and Apple, as well as individuals generating and owning their own health data.

A survey undertaken by the working group only received 37 responses from industry, and these were evenly split between big pharma companies and SMEs.

big data roadmap

The taskforce's visual representation of the Big Data roadmap

Respondents from large pharma tended to highlight personalised medicine, understanding current clinical care and informing clinical trial design as areas where big data could have the greatest impact, while SMEs emphasised patient reported outcomes, outcome identification and signal validation as the most important areas.  However, the issues of data access, data integration, data validation and data reproducibility were key concerns shared across the sector.

The roadmap sets out the direction of travel, and subgroups have created a set of recommendations for action, such as agreeing on data formats and standards for regulatory submissions of raw patient data, and granting Europe’s regulatory network direct access to individual patient data (IPD) during assessment of a marketing authorisation.

Europe’s efforts mirror similar announcements from the FDA, where commissioner Scott Gottlieb has unveiled numerous initiatives, including on real world data, integrating health records with clinical trial data, and a Digital Health Innovation Action Plan.

Read the summary report of the HMA-EMA Joint Big Data Taskforce

Article by
Andrew McConaghie

18th February 2019

From: Regulatory

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