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Patients The Key to Real World Data?. Alan G. Wade. Real World Data Sources. What is real world data?. Reflects the average patient in a real-world setting Demographics Sex Age Social status Education Diagnosis Co-morbidities Co-medications Medical system Social impact

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what is real world data
What is real world data?

Reflects the average patient in a real-world setting

  • Demographics
    • Sex
    • Age
    • Social status
    • Education
  • Diagnosis
  • Co-morbidities
  • Co-medications
  • Medical system
  • Social impact
  • Work impact
  • Family impact
  • Impact on Quality of Life
hierarchies of evidence
Hierarchies of evidence
  • Systematic reviews of randomised controlled trials (RCTs).
  • RCTs.
  • Controlled observational studies - cohort and case control studies.
  • Uncontrolled observational studies - case reports.
  • Expert opinion
    • ?KoL
randomised controlled trials
Randomised Controlled Trials
  • “the gold standard” for demonstrating (or refuting) the benefits of a particular intervention.
  • Important limitations
limitations of rct s
Limitations of RCT’s


  • Age - Effectiveness in younger or older patients
  • Sex
  • Severity - effectiveness in mild or severe
  • Risk factors - effectiveness in patients with risk factors for the condition (eg, smokers)
  • Co morbidities - Influence of other conditions
  • Ethnicity - effectiveness in other ethnic groups
  • Socioeconomic status - effectiveness in disadvantaged patients


  • Dose - high dose used in RCTs?
  • Timing of administration Influence on adherence (compliance) to treatment regimens
  • Duration of therapy - effectiveness during long-term use
  • Co medication - adverse interactions
  • Comparative effectiveness - in comparison with other products used for the same indication


  • Quality of care
  • Prescription and monitoring by less specialist (expert) healthcare providers
  • Care pathway
effectiveness and efficiency
Effectiveness and efficiency




Does it work in

Does it work in

Does it contribute to a

clinical trials?

real life?

more efficient use of



post marketing surveillance
Post Marketing Surveillance

To assess performance of drug in real world setting

  • Large numbers
  • Off-label prescribing
  • Comorbidities
  • Concomitant medications
  • Speed of reporting

Do we achieve this with formal PMS?

hierarchies of evidence1
Hierarchies of evidence
  • Systematic reviews of randomised controlled trials (RCTs).
  • RCTs.
  • Controlled observational studies - cohort and case control studies.
  • Uncontrolled observational studies - case reports.
  • Expert opinion
    • ?KoL

6. Patient reports ?

focus groups
Focus groups
  • Small numbers
  • Representative?
  • Skill of co-ordinator/observers
who in the world most influences the pharmaceutical industry
Who in the world most influences the pharmaceutical industry?
  • Barack Obama

2. Michael Rawlins

harveian oration
Harveian Oration

Hierarchies of evidence should be replaced by accepting—indeed embracing—a diversity of approaches.

The Lancet Vol 372 December 20/27, 2008


Patient and public involvement

The views of patients, carers and the public matter to NICE. We want to involve them, as well as doctors, nurses, other healthcare professionals and managers in our work. – accessed 22 06 09


The assessment of the benefit-risk balance should be based on the available tests and trials, which are designed to determine the efficacy and safety of the product under normal conditions of use (Directive 2001/83), and which are generally performed under ideal conditions.

It is important to be explicit about the perspectives of different stakeholders that are taken into account in the assessment of the benefit-risk balance,in particular the perspectives of patients and treating physicians.

Considerations about how the treatment is expected to perform under real conditions of use are relevant in the context of pharmacovigilance activities, for example, to take into account any available information on misuse and abuse of medicinal products which may have an impact on the evaluation

of their benefits and risks (Directive 2001/83).

patient groups the patient
Patient groups - the patient?

EPF is the umbrella organisation of pan-European patient organisations active in the field of European public health and health advocacy.

The European Patients’ Forum (EPF) currently represents 57 patient organisations and an estimated 150 million patients across the 27 Member States throughout Europe.

patient organisations
Patient organisations

EU drugs agency working with patient groups bankrolled by big pharma

23.04.10 @ 19:17

the lancet vol 372 december 20 27 2008

Harveian Oration

But equally, we have a right and responsibility to look at the interests of other patients who use the healthcare system. What I am critical of, however, is patient organisations that are acting on behalf of pharma companies. I am not alone in complaining about them.

The Lancet Vol 372 December 20/27, 2008

big data definition
Big Data - definition

No single agreed definition


The bottom line: whatever the disagreements over the definition, everybody agrees on one thing: big data is a big deal, and will lead to huge new opportunities in the coming years. Accessed 27 01 14

big data pharma
Big Data – Pharma???


Claims Databases (US)

e - Medical Records


Pharmacy databases

Specific hospital databases

Specific disease or procedure databases

what do you want from a registry
What do you want from a registry?
  • Large numbers
  • Patient level data
  • Immediacy of data
  • Longitudinal data
  • Representative population
  • Presence of YOUR required data
  • Linkage of data fields of interest
  • Confirmation of diagnosis
  • Standardised measurement
  • Validated PRO’s
existing registries
Existing registries


  • Large numbers
  • Immediate access
  • Longitudinal data
  • Prescribing data


  • Inherent biases
  • Representative population
  • Diagnostic drift
  • Patient level data
  • Surrogate outcomes
  • Completeness of data
  • Family social and work history

To effectively use any registry it is important to understand how it has been developed and its strengths and weaknesses

patient reported outcomes why how
Patient Reported Outcomes Why & How?
  • Some treatment effects only known to patient
  • Pts provide unique perspective on treatment
  • Provide information on QoL, work, social and family
  • Formal assessment may be more reliable than informal interview

Addressing registry weaknesses

What the Patient Knows

What the Patient Shares

Ask the patient – but how?

What the physician understands

What the Physician records

Big Data



Collect data directly from patients

Patient Reported Outcomes


patient reported outcomes definition
Patient Reported Outcomes - Definition
  • “any aspect of patients health status that comes directly from patient” - FDA
  • “insight into way patients perceive their health & impact treatments or adjustments to lifestyle have on their quality of life” –DH
patient reported outcomes concerns
Patient Reported Outcomes – concerns
  • Pt recording versus doctor
  • Pt understanding of question/form
  • Validity of question (in that format, pt popn)
  • Reliability of question
  • Ability of question to detect change
  • For licensing – need set as per RCT
definition of real data from real patients
Definition of “Real Data from Real Patients”
  • Collecting data from patients receiving “routine care”


  • Not affecting their prescribers/ healthcare professionals behaviour


Patient Reported Outcome Based Evaluation

process set up
Process set-up
  • Define question - protocol
  • Define patient group of interest
  • Determine location of group e.g. Specialist unit, community setting, geography
  • Review options for accessing patient group
    • Orphan indications
  • Design questionnaires and reports
    • development and testing
  • Structure customised database
structured patient registries
Structured patient registries
  • Bespoke – ask required questions
  • Innovative
  • Prospective
  • Hosted on Patients Direct site
  • Interactive
  • Global Coverage
  • Is on-line collection satisfactory?
  • Will patients cooperate
  • How do you recruit?
    • Methodology
    • Achieving a representative population
on line reporting
On-line reporting
  • Age?
  • Education?
  • Social class?
  • Carer reporting?
    • AlzDis
    • children

Generally require alternative data collection routes

Nurse manned telephone

why do people participate
Why do people participate?

...the benefits and attraction to each individual will differ but we believe the main reasons are :

  • Outlet for their feelings and views – might be a threat to their relationship if they report problems to their healthcare professional – we’re neutral
  • Altruistic - Assist in developing new and better treatments
  • Obtain better information and knowledge through participation
  • Feel valued through regular contact/ interaction
  • Desire to make sure the patients voice is heard
will patients cooperate
Will patients cooperate?


The above report has shown the public’s enthusiasm towards a system that would let

consumers report adverse drug reactions through the Internet. The findings of the

survey carried out by us revealed to us this enthusiasm. The report has further shown

that health professionals have a positive opinion towards such a system, which


in favour of the overall mission of Patients Direct. Not only has the report articulated

the publicity campaign that Patients Direct can carry out to raise its awareness, but

also examples of different medicine inserts has been provided with reasoning behind

them that could be used by the company to make itself different from its competition.

Patients Directs corporate identity is important to begin the process of publicity. IT

has been noted that establishing a user friendly website that is easily navigable is

fundamentalto setting the pace for a strong brand and image that will appeal to

Patient’s Direct customers.

EDGE Survey - Market Research, 2008


...tailored to attract patients of interest with a conscious effort to reduce bias and population selection issues

Core recruitment methods include :

  • social networking sites
  • search engine and website optimisation
  • public affairs articles and traditional methods of publicity e.g. Newsprint/TV
  • Healthcare professionals –Doctors, pharmacists
  • wholesalers, distributors
  • Clinical trial participants
  • patient groups

Special focus can be given to groups of interest such as children if appropriate and recruitment monitored to ensure sufficient numbers in each cohort.

recruitment examples
Recruitment Examples
  • General – statins
    • Pharmacy/wholesale distributor
    • Advertising - Google
  • Vaccines
    • Direct at vaccination
  • Families and children
    • Appropriate web-sites
  • Specialist product – home delivery
    • Invitation with delivery
  • OTC – Strepsils
    • General advertising
    • Pharmacy
    • Package wrap
  • Recruitment Method - Facebook only
  • First Participant Recruited – 22nd Dec 2013
  • Data Protection/ Confidentiality
  • Ethics
    • NRES response
  • Industry Code of Practice
  • Safety Reporting systems – MHRA
    • Automated A/E reporting
    • A/E cascade
  • Medical considerations/standards
    • No interference with prescribing/treatment
process management
Process - management
  • Patient response handling and back up
  • Review of data as study progresses
  • Design of reports
    • A/E reporting in agreement with sponsors
  • Statistical interpretation and reporting
  • Publications
  • If appropriate, patient education or further action e.g. Adherence schemes
  • Medical interventions are now being assessed on the basis of “real world” data
  • Current collection methods are inadequate
  • Novel systems for collection are required
  • Patients are increasingly being involved in medical decision making
  • Collecting “real world” data directly from patients may be one possible method
www patients direct org


Patients Direct

3 Todd Campus

G20 0 XA

United Kingdom

output examples

Output Examples


Cover all 4 areas of use

pv flu vaccination
PV - Flu Vaccination
  • 102 leaflets distributed
  • 73 PIN numbers have been entered on the database (72%)
  • 40 male and 33 female aged between 21 and 99 years.
  • They were asked about pain and discomfort from the vaccination
flu vaccination day 2
Flu Vaccination - Day 2
  • 70 responses were entered for Day 2
  • 14 having side-effects.

Reported Side Effects

In response to “What did you do

about these side effects?”

hive demographics
HiVE - Demographics

Male – 449

Female - 663

hive side effects
HiVE – Side effects


p <0.001

H1N1 only


Subject participates

  • “Buy-in”
  • Enrols “Buddy”
  • Subject + Buddies + “Virals”

The Virtuous Cycle

  • Feedback on outcome
  • “Viral Transmission”
  • Engaged for next year
enbrel project
Enbrel Project
  • Delivery at home
  • Compliance with drug still low
  • Understand agency effectiveness, reasons for drop outs
  • Understand real life prescribing vs guidelines
  • Evaluate and then implement ways of improving outcomes
  • Measures – AEs, compliance, QOL – disease specific and generic EQ5D
enbrel process
Enbrel - Process
  • Patient group recruited through leaflets with hospital clinic or HaH nurse visit to train on injection
  • Initially 6 month follow up at 1 month intervals
  • Recently extended to 2 year follow up at 3 month intervals
enbrel first 6 months
Enbrel – First 6 months
  • Enrolled 344 patients
  • Out of ~1000 leaflets distributed
  • 284 patients by web site
  • 60 patients by telephone
  • 93-100% completion of questions at baseline
  • 140 patients have reached month 6

If you are experiencing problems with the website or any of the questions please contact the following number and we will endeavour te help you: 0800 731 2647

intermittent events data gathering
Intermittent events- data gathering
  • Examples – MS, epilepsy, gout, infectious illness, depression
  • Pre programmed questionnaire – timing ??
  • Baseline then every month/ 2 weeks - recall?
  • Simple email – yes or no
  • Rely on people saying when they have “event”
intermittent condition problem periods
Intermittent Condition – Problem Periods
  • Capture QoL changes when worst time of cycle
  • Variation within and between women
  • Compare to “normal” time
results screening survey
Results – Screening survey
  • 2699 respondents
  • Age – good range from < 20 to > 49 (61% < 30)
  • Absenteeism – 3+ days/mth 6%

1-2 days/mth 16%

results main outcomes
Results – Main Outcomes
  • Significant impact on ALL Quality of Life Scales
  • Significant change between the different times of period cycle
resource utilisation
Resource utilisation
  • Routinely gather PRO on
  • All Medication related to condition
  • Doctor visits (Primary & Secondary care)
  • Nurse visits
  • Pharmacy visits
  • Telephone calls (if relevant)
  • Hospital In/ Out Patient visits and number of days

A real world study using Patient Reported Outcomes to assess the consequences associated with the forced switching of asthma medication/device in stable adult asthma patients

  • Data to be captured from Patients themselves:
  • baseline patient reported level of Asthma control/satisfaction with device
  • Reasons for switch (if known) (prospectively)
  • Outcomes of switch: Clinical (FEV1, Control) Health resource use (GP visits, hospitalisations) lifestyle impact (days off work) (prospective)
  • The questionnaires would be administered monthly (prospectively) so that any changes are captured
cystic fibrosis
Cystic Fibrosis
  • Unmet need
  • Gathering information on children/ adolescents/ parents
  • Adherence, satisfaction, burden of illness
  • If you need real world PRO data in Europe/US
  • Databases don’t collect info you need
  • Patients Direct can collect the data, directly from the patient

Quick, efficient, cost effective solution

market access
Market Access
  • Burden Of Illness Study – Depression Management
  • Understanding Patients ability to monitor their own condition – Heart Rate survey
  • Disease treatment pathway mapping – CVA Study
  • Mapping new EQ5D
  • Are QALYs appropriate across EU ?
burden of illness 300 pts depression
Burden Of Illness - 300 pts depression
  • Socio-demographics,
  • PMHx,
  • Resources use – Client Service Receipt Inventory
  • QOL - EuroQoL
  • Productivity – WHO Health & Productivity Questionnaire
  • Depression – HAM-D, MADRS
burden of illness
Burden Of Illness
  • Utility weights UK population data
  • and EQ5D
  • → QALYs
burden of illness1
Burden Of Illness
  • Depression severity - cost
  • Depression severity - QALYs
disease pathway and management
Disease Pathway and Management
  • Medical pathway of ischaemic stroke until 1 year acute episode
  • Cost of stroke management
  • Cost drivers
  • Comparison between UK, France, Germany
disease pathway and management1
Disease Pathway and Management
  • Socio-demographic
  • Pre hospital management – PMHx, 1st contact, transport
  • Hospital management – treatments, investigations
  • Post hospital management - rehabilitation
eq 5d mapping
EQ 5D Mapping
  • New 5 level questionnaire (from 3 level)
  • 500 pts UK
  • Different levels disability
  • CV disease, Respiratory, Neurological, RA
use of qaly across eu
Use of QALY across EU
  • FP 7 grant
  • Pan European – UK arm (with A Walker)
  • University of Lyon
  • Identification of different methods in HTA
  • Review of different methods
  • Alternatives to QALY
he and or
HE and OR
  • Involved in questionnaire mapping
  • Gathered data via various QoL scales
  • Gathered data on inputs and outputs -costs, diseases, outcome
  • Individuals involved in SMC and NICE submissions and Advisory Boards
  • Access and use of UK experts – Robertson Centre
  • Bespoke Innovative solutions
  • Professional Quality Assurance /control
  • History of delivering results
    • On time
    • Value for Money
  • Tailored to sponsor brief
  • Multiple Applications