We could be entering a very dangerous chapter in public health across the globe, writes Simon Tilley.
Pathogenic bacteria are becoming resistant to the drugs that used to kill them. Each year, an estimated 400,000 patients across the EU, Iceland and Norway present a resistant strain, leading to 25,000 deaths. Not only is this a pressing medical issue, it also has huge economic implications. The World Health Organisation reports that multi-drug-resistant bacteria in the EU are estimated to cause an economic loss of more than Euro 1.5 billion each year.
The scary fact is no new drugs in this area have been released for more than 25 years, and no new class of antibiotics developed. Infections that are currently treatable could very soon kill once again.
One of the biggest barriers to developing new drugs, improving patient care and curing diseases are the cost and time required to bring new drugs to market. Many argue that this is the biggest challenge facing the healthcare industry. It will take years and an estimated Euro 3.79 billion to create a new drug. In an effort to help clinicians and the healthcare industry find cures quicker, data transparency is being seen as a way to speed up drug development, save on duplicated effort and get quicker breakthrough therapies to those in need.
Key industry bodies, including regulatory authorities like the European Medicines Agency (EMA), medical researchers and other leading industry influencers are also placing pressure on the biopharmaceutical sector to provide complete transparency into de-identified patient-level data and results from clinical trials.
Consensus is building on the benefits of greater access to such patient-level data. Greater transparency in clinical cancer research, for example, has been recognised by the EU as being essential to helping scientists and doctors find more effective ways to diagnose and treat cancer in its Clinical Trials Regulation on 27 May 2014. Project Data Sphere, an initiative led by the CEO Roundtable on Cancer’s Life Sciences Consortium, was an early step towards this vision. The true power of this new data-sharing platform will come from an ever-increasing volume of data and the continuing engagement of a diverse global community focused on finding solutions for cancer patients.
From a pharmaceutical perspective, pooling data from different trials in a consistent way can significantly reduce the cost of drug development, while improving the efficiency of clinical trials. Indeed, big pharma companies including GSK, Sanofi, Johnson & Johnson and Pfizer are already involved in data transparency projects that really augers well for health.
From a medical perspective, aggregating information from multiple trials and sponsors can lead to deeper medical understanding and new scientific breakthroughs. Data sharing also enables objective, third-party review and validation of study results, thereby building public trust in biopharmaceutical treatments. The medical knowledge gained through access to clinical study data results means better treatments can get to market faster.
These benefits, coupled with regulatory pressures such as the European Medicines Agency’s March 2014 recommendation that pharmaceutical companies should share clinical trial data, has resulted in the biopharmaceutical industry shifting its mindset towards allowing access to historically proprietary, patient-level clinical trial data. However, there are many implementation factors to consider.
For clinical trial transparency to take hold, patient privacy is paramount. Industry leaders have legitimate concerns about issues of patient privacy, potential misuse of data and loss of proprietary advantage. The crux of this solution is how data transparency is operationalised – such as giving researchers secure access to studies, and ensuring patient privacy without unduly limiting the research value of the data.
To preserve data integrity and prevent users from e-mailing it to unauthorised people or surreptitiously altering it in any way, data transparency initiatives must be underpinned by a secure clinical trial information platform.
Medical researchers fundamentally want an environment in which to create, run and save their own analyses across data sets from multiple organisations, while having the ability to securely collaborate on a project with other researchers. The ability to analyse clinical trial data in this way means researchers and organisations can utilise statistical routines to reconfirm the analyses performed, combine data across a series of studies, and explore unresolved medical questions. Researchers can confidently generate new medical knowledge, improve the efficiency of clinical trials, verify trial results and improve healthcare.
As such, a clinical trial data transparency platform must allow medical researchers to analyse, model and draw insights from the huge data sets, while protecting the data in a ‘castle and moat’ environment. This means that authorised researchers can access de-identified, patient-level data while also being limited to just the data needed for the approved project. Organisations can also limit extracts from the system, and from a data transparency systems perspective, researchers must be able to request access to study data and track the approval process.
Ultimately, data has the power to uncover answers and bring cures to market much quicker. Sharing and collaboration is critical to success – the opportunity afforded to the EU and across the globe by sharing de-identified patient level data is enormous.
It is clear that in order for the biopharmaceutical industry to productively move towards making cost-effective speedy developments, data transparency initiatives must be pushed forwards. Building on the initial success of early data transparency adopters, the EU has already made a lot of progress in a short time, but there is more to be done. There is now a clear path to success with clinical trial data transparency that will benefit patients in the near future, and hopefully head off the worrying threat from the new breed of pathogenic bacteria.
Simon Tilley is Head of Pharmaceuticals at SAS UK & Ireland