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Ever decreasing patient populations... an opportunity for Real World Evidence

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Date: 3/8/2016


Medicines that are aimed at more and more specific targets, resulting in smaller and smaller groups of patients who can benefit from new drugs, are becoming a familiar story in cancer treatment. Rare diseases by definition also each affect small numbers of patients. But as Professor Richard Barker, Director of CASMI reminded the audience in his opening address to the 2016 pH Summit, even diabetes (a disease affecting almost 3.5 million people in the UK) is being subdivided beyond the traditional type I and type II classification. As research discovers further sub-types such as monogenic neonatal diabetes which respond differently to different anti-diabetic drugs, these patient populations too are becoming smaller and smaller.

While this presents a challenge for researchers looking for eligible patients to recruit efficiently into clinical trials, it does offer an opportunity for the UK to capitalise and build on Real World data (RWD) assets such as the UK Biobank, SACT and the 100,000 Genomes Project, and Big Data informatics collaborations such as the Farr Institute. But how to make this happen?


‘Following Winds’

Professor Barker highlighted the work of the Precision Medicine Catapult, a recent initiative which aims to make the UK the go-to location for development of targeted medicines. He also suggested some ‘Following Winds’ which are supporting this drive:

·       Policy drivers such as:

o   The European Medicines Agency Adaptive Pathways to Licensing scheme which allows early conditional approval with RWD collection for safety, efficacy and value evidence generation; the Cancer Drugs Fund #2 also builds on this value model.

o   Outcomes-based reimbursement, which takes a risk-sharing approach, relying on RWD to provide the evidence of outcomes

o   The Accelerated Access Review, now expected to report in July 2016 following the EU referendum.

·       Technology drivers increasing our ability to easily collect or analyse RWD:

o   Wearable technologies such as the FitBit, Apple watch and the Hexoskin biometric vest

o   Machine learning




Professor Barker turned to the audience to identify the challenges of collecting and using RWD to support access to medicines and suggestions included:

·       Lack of PROs (Patient reported outcomes) in medical notes

·       Missing/inconsistently recorded/inaccurate data recorded in medical notes/databases

·       Absence of comparators in the analysis

·       Potential for bias, hence reliability of conclusions

·       Need for adjustment to deal with potential confounders

·       Fear of contradicting RCT results

·       Capacity and willingness of health care organisations to work with Pharma to collect data – lack of incentive

·       Systems integration to link/extract datasets is costly and time consuming

·       Data linkage between sectors such as primary and secondary care is still difficult

·       Early use of a medicine may not be in a typical population, but RWE often must be generated from this early use group

·       It is not yet clear whether policy makers will accept RWD



Value of Real Word Evidence (RWE)

In the light of these challenges, Professor Barker again turned to the audience to identify the value of RWE:

·       RWE is more representative of the whole population, as opposed to a clinical trial population

·       RWE allows the study of populations who would be excluded from RCTs

·       RWE is relatively quick to collect

·       RWE can supply different outcomes to a RCT

·       RWE gives a better indication of value of a medicine, not just safety and efficacy

·       RWE can show the resource implications of managing adverse events

·       RWE can reflect the effect of real life, such as a busy schedule, polypharmacy or co-morbidities on compliance and outcomes


Summarising, although there are challenges in both technological and organisational terms related to capacity, capability and culture, RWE has the potential to provide valuable information on the practical context of drug use, which must be further developed to support the drive to bring targeted medicine development to the UK.

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