The cost of taking a drug to market – estimated to be approximately $2.6 billion – combined with increasingly tight healthcare budgets, has put intense pressure on R&D teams.
Genomics, or more widely, ‘omics’ (genomics, proteomics, and metabolomics, etc), might be the solution. By using patients’ genetic profiles, scientists should be able to narrow down patient groups into subsets with a higher chance of responding to a specific treatment. Surrogate endpoints are also speeding up trials and eliminating ineffective therapies at an earlier stage, getting products into the market earlier, at lower cost.
From the perspective of Jo(e) Bloggs, there is the personal benefit from having your DNA analysed to determine which conditions you or your blood relatives may be genetically predisposed to. But it also benefits the health of the wider population. On top of this, the economic benefit of greater investment in prevention is huge, and has a measureable (positive) impact on GDP (gross domestic product).
In a previous life I was an oncology consultant in the NHS, and I have first-hand experience of cancer. This is one area where genomics has enormous potential. Take genetic sequencing company Adaptive Biotechnologies, for example. The company is partnering with Microsoft to map the genetics of the human immune system, or immunome, in a bid to help detect early stages of cancers and other diseases. The future is far brighter for cancer patients, thanks to ‘omics’.
Data, data everywhere
A recent KPMG survey carried out among 2,000 people in the UK found that 53% of people would be willing to share their DNA with a pharmaceutical company to help research into new drugs and treatments.
I spend a lot of time in the US, and I strongly believe that if the survey was carried out there, the results wouldn’t be anywhere near as positive. We trust in our NHS, and the UK is making waves when it comes to progress in genomics.
A great example is Inclisiran – the new cholesterol drug (developed by The Medicines Company, acquired by Novartis) that’s expected to save 30,000 lives over ten years by giving patients accelerated access to the drug. The clinical trial will use NHS data to identify patients across the country who are at risk of heart disease, and who haven’t benefited from conventional treatments. The reason this world-first trial is possible in the UK is thanks to the vast amount of data the NHS holds. There is no other health system in the world that follows people from cradle to grave with a unique identifier.
We also have the benefit of resources such as Biobank that are enabling life sciences companies to have access to the same data as the public sector. And of course, UK Research and Innovation has recently announced its Whole Genome Sequencing project, where 500,000 people’s genetic codes will be sequenced to better understand, diagnose, treat and prevent life-changing diseases.
What’s stunting the UK’s progress in ‘omics’, however, is the lack of joined-up electronic health records. Let’s be honest, this isn’t the NHS’ strongpoint. But some of this is attributable to the nervousness that still persists among patients around the security of personal data. The fear that personal information will be used without permission by pharma companies, especially for corporate gain, was a concern among more than half (54%) of our survey respondents. If genomic data falls into the wrong hands, it is worth far more on the black market than, for instance, credit card data.
This fear will and is already having a major impact on medical research. In my personal view, the risks here are hugely overplayed. The theft of trade secrets by hackers and company insiders is actually a far greater threat to the industry than to individuals.
Other challenges
Although justified, regulation is holding the industry back as it struggles to keep pace with scientific progress. The FDA is moving as fast as it can, but access to drugs is still too slow. The industry needs to think about the shift away from blockbusters for many and a move towards niche busters, where only small numbers of patients will be eligible for therapy but because of its precision it will be more effective.
Another hurdle is the GDPR. Meeting the specific requirements isn’t easy, particularly when it comes to collecting data from wearable tech. There also needs to be a suitable legal basis to justify the data processing. And while the GDPR allows individuals to give their consent only to certain areas of research, it is extremely difficult to manage a full audit trail of consent for each area of research and for each individual taking part. Again many NHS organisations have mitigated this problem by seeking consent at the point at which patients
Moving away from the data challenge, the industry and healthcare systems need to address patient numbers for trials, particularly among BAME (Black, Asian and minority ethnic) groups which have been historically under-represented. As genomic targeting gets more precise, the volume of potential trial participants continues to fall, to the point where it can be very difficult to reach an adequate sample size.
We need to see more innovative trial design and ways of comparing groups of patients that move beyond the ‘old’ randomised controlled Phase III trial which uses endpoints which take years to reach. Data sharing between companies that are traditionally competitors could open the door to faster trials and subsequent product approval. This is where third parties come in. Companies can act as an ‘intermediary’ hosting platforms which enable a range of data sources to be used and remain compliant with regulations and privacy standards. Developing secure platforms so that organisations like Google, Microsoft and the NHS can gain insights from each other’s information, without sharing or exchanging personal data or IT systems, has huge potential.
Genomics data strategy
Yes, there are challenges. But it’s time for life sciences companies to jump on the omics bandwagon, or risk being left behind. Quite simply, pharmaceutical companies can’t afford not to.
If any life sciences organisation is going to derive value from genomics, it absolutely has to have a robust genomic data strategy that consists of:
1) Standardised sequencing and analysis
2) Privacy
3) Security
4) Storage and data transfer
5) Harnessing analytical technology.
There is much more to be done to raise awareness and make it clear that the benefits of sharing personal data far outweigh the risks.
Professor Hilary Thomas is director and chief medical adviser at KPMG in the UK
Source: Pharma Times
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