A Short Call with the FDA

Srinivas Karri
Director of Product Strategy

Did you know that over 15,000 clinical trials are currently active around the world, with another 45,000 trials actively recruiting?  This was the statistic provided to me by a representative from the U.S. Food and Drug Administration (FDA). I will share some of the interesting aspects of this conversation which directly influences how we view clinical data today, and the technological direction we need to move towards for the future.

Clinical Data Volume

45,000 clinical trials generate an enormous amount of data.  Not all of the data is useful. Much of it is discarded or archived, as neither the clinical value of the information cannot be established, nor is patient consent provided for secondary use.  A quick calculation on the back of an envelope has suggested that over $1 trillion worth of data is generated and never used again. 

 A fundamental constraint with obtaining patient consent for secondary use means that there is a huge opportunity to collect data differently and to maximise its utility. In this regard the FDA is piloting a program to create a ‘data market’ designed to empower patients, healthcare providers, and insurance companies with the right balance of incentives to optimize healthcare outcomes.  A key consideration is the balance of incentives within a well-defined ecosystem that empowers each of the stakeholders to contribute and collaborate.

Historical approaches that have tried to aggregate and share patient data using personalized portals and information vaults have not gained widespread traction.  Part of the reason for this failure has been the lack of incentives for the patient to contribute data, the lack of incentives for insurance providers to use the data to make meaningful and effective healthcare decisions for the patient, and the lack of incentives for technology providers to create a secure and robust data management ecosystem. 

Ultimately, all of the stakeholders need to collaborate through sharing of data, interpreting/predicting outcomes, and identifying optimal treatment pathways that maximize patient safety and make economic sense.  On a side note, it turns out that the vast majority of health care costs associated with an individual are incurred within the last few months compared to the whole of life. This point is dramatically illustrated with the cost of oncology care.

The Rising Cost of Healthcare

Even when the very best care is provided -- and it is estimated that the cost of oncology care spend is expected to increase to over $150 billion per year by 2020 -- much of this expenditure is off target or ineffective.  Health care providers are unable to determine the efficacy of treatment due to a lack of data that correlates the indication, the genomic profile of the individual, and the treatment.  It is also apparent that this is leading clinical research development organizations to look at more ‘personalized treatments’ that clearly demonstrate value for money.

So how can we make headway against these strong economic and scientific headwinds?

Ready, Steady, Sequence

One suggestion is to assist everyone to have their genome sequenced.  The current cost of sequencing of the human genome is a few hundred dollars. If everyone was sequenced irrespective of any medical condition and the data was made available this would be a step toward a more collaborative healthcare ecosystem. The total cost of sequencing all of the individuals in the US may reach up to $70 billion.  However, this is a small cost when you consider that between 50 to 70% of oncology treatment is off target.  Increasingly, the use of biomarker data is gaining momentum with a significant number of research organizations routinely bio banking clinical samples.

Figure 1. May 2017 - Global Oncology Trends 2017.  Advances, Complexity and Cost

By maintaining a centralized repository of sequencing data and associating the clinical healthcare record of the individual, it will be possible to make better clinical decisions across the entire patient population more effectively than ever before. Not only will an individual be able to understand whether a suggested course of treatment is predicted to be effective, using empirical data, the health care provider and the insurance payer will be able to optimize constrained financial and scientific resources.


Figure 2.  Biomarkers are increasing being used to segment Cancer in selected tumors

Furthermore, an attractive side-effect with a centralized repository of clinical and scientific data is the possibility of fewer medical negligence and malpractice suites due to new levels of data transparency being offered.  By introducing new capabilities to broker and share data in a secure way the potential to use this data for secondary use can also be explored.  In this regard, the FDA is looking at Blockchain to support patient consent use cases throughout the clinical data generation, analysis, and submission lifecycle.  This also, of course, has extraordinary implications as clinical trials would routinely generate significant volumes of genomic data that would be in orders of magnitude larger than what is collected and analyzed today.

Next Steps

The FDA is preparing for changes.  It is anticipated that genomic data will become routine as part of the clinical dataset.  Sequencing data will be generated for each individual participating in the trial, and this will be associated with the resulting clinical dataset which will ultimately belong to the patient.  The patient will have control over all of his/her data and will be able to provide consent for secondary use across an ecosystem of clinical data consumers.  The FDA wants to build a marketplace in which patients will be able to share data using a common data platform that incorporates balanced incentives across the participating stakeholders.

The platform would allow patients complete mobility to move their healthcare records from one healthcare provider to another.  It is envisaged that a patient should be able to have a complete physical geographic mobility, yet maintain a single clinical and healthcare data profile using one technology platform.  Using new technology, such as smart contracts and Blockchain, clinical data could be brokered to improve clinical and healthcare decision-making for the wider population. 



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