Real World Evidence

Our services in this area include:

 

Pharmacoepidemiology

An indisputable leader in Pharmacoepidemiology

Founded by one of the world’s pioneers and leaders in pharmacoepidemiology and public health, Professor Lucien Abenhaim, who directed the famous McGill Pharmacoepidemiology Education Program for several years, LASER ANALYTICA offers unique consulting capabilities in these domains.

These consulting services are provided both internally to the LASER ANALYTICA teams conducting pharmacoepidemiology research and externally to customers who require support in the design and supervision of studies. We also provide statistical analysis and data management services for observational data.

  • Study design: protocol, scientific boards
  • Research tools: Development of e-CRF & questionnaires
  • Supervision of studies
  • Statistical analysis plan
  • Programming (most packages)
  • Statistical analysis
  • Medical writing

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Study Design and Control

LASER ANALYTICA scientists pioneered a series of innovative designs in clinical pharmacoepidemiology, to respond to complex comparative effectiveness or relative risk questions (mostly at the request of health authorities in Europe, either for regulatory or for coverage with evidence development purposes).

Our teams are expert in the following designs, which can be applied to the analysis of existing databases (eHCD, eMR, PGRx) or to de novo collections of data planned by clients (and realized by them or other teams):

  • Cohorts with fixed or dynamic exposure
  • Mirror image studies historic-prospective designs
  • Case-referent designs (case-control, case-cross over, case-cohort)
  • Pragmatic trials
  • Multiple purpose data platforms

When appropriate, our sister company LASER C.R.O. (Cohorts, Registries & Operations) can ensure the collection of clinical data for the conduct of these studies.

Identifying and interpreting biases

The identification of biases stemming from observation is a cornerstone of epidemiological training and experience. While the infamous confounding bias, including indication bias, and exposure information bias, including recall bias, are the subject of most attention, the distortions of selection biases and non-differential misclassification, both on exposure and on case/non case status are too often neglected. All require the utmost experience. Contrary to common belief, all of these biases may affect both clinical and electronic database and medical record studies alike. Experience lies in the ability to minimize biases but also in the ability to draw meaningful conclusions from observational results nonetheless.

Data Management & Analysis

Ensuring Validity of Observational Data

LASER ANALYTICA ensures the data management for numerous studies conducted by clients. Its systems and programs are particularly adapted to the management of observational data, including data collected for pragmatic trials. The data collected for pharmacoepidemiology studies and pragmatic trials require an element of variability and adaptation to the ‘dynamic’ nature of the exposure and outcomes measurement that is often lacking in those systems and processes usually developed for clinical trials or fixed registries.

LASER ANALYTICA data management SOPs and processes as well as its IT security and privacy protection meet the highest standards in the industry, including all requirements of regulatory agencies, a quality rare for observational studies.

Analysing dynamic exposure data

LASER epidemiologists and statisticians are expert in the most sophisticated and advanced statistical analysis techniques, including all regression models and splines, control for confounding, propensity scores analysis, risk score analysis, time-varying models, SMS, varying time-window designs, depletion of susceptibles assessment, survival analysis and multiple others. They have unique experience in the analysis of ‘dynamic’ data in pharmacoepidemiology.

Methodological Programmes

Thanks to its leading participation in several international methodological initiatives, the IMI and ENCePP programs, LASER ANALYTICA provides its clients with the full benefit of the newest developments applicable to the evaluation of medicines.

Innovative Medicines Initiatives (IMI)

Two major IMI programs have recently been developed to address methodological issues relating to the study of drugs in real world settings: the PROTECT program and the GetReal program. LASER is the sole SME playing a leading role in both:

  • Our teams chair the WP6 and participate in the WP2 and WP5 of PROTECT, a consortium coordinated by the European Medicines Agency which aims to develop innovative methods for the assessment of the risk of medicines
  • We chair the WP2 of GetReal, a consortium established to propose new approaches for the evaluation of the efficacy of medicines in current medical practice: we lead the methodological component of the consortium.

ENCePP

LASER ANALYTICA was one of the pioneer members of ENCePP (European Network of Centres in Pharmacoepidemiology and Pharmacovigilance), a program of the European Medicines Agency.

 

Outcomes Research

We provide cutting edge outcomes research designed to demonstrate the benefit to patients that products and health technologies provide. Our Outcomes Research is applied patient- and population-based research that seeks:

  • To study the end results of healthcare in “real life” conditions
  • To study healthcare pathways and resource use in the real world
  • To study the burden of illness of a specific disease or group of patients
  • To target drugs to unmet needs and identify niches

Outcomes Research uses not only medical measures, but also Patient-Reported Outcomes – (PROs) – e.g. Health Related Quality of Life, adherence to treatment, satisfaction with treatment and symptom questionnaires – and other related measures such as utility measurement.

Preferences studies are also included in Outcomes Research. In the last few years, the Patient-Centered Outcomes Research has become more and more important. It helps people and their caregivers communicate and make informed healthcare decisions, allowing their voices to be heard in assessing the value of healthcare options.

For this purpose, we use a series of methods:

We rely on the resources of our sister company LASER C.R.O. (Cohorts, Registries & Operations) to ensure the optimal collection of clinical data for the conduct of these studies.

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For operations please see:

LASER C.R.O.
(Cohorts, Registries & Operations)

Surveys & Health Services Research

Burden of Disease Studies

LASER ANALYTICA has in-depth experience in the conduct of burden of disease studies, both from a patient- and a population- perspective. We combine physician and patient surveys to quantify the burden of illness associated with a specific disease, including estimations of incidence and prevalence, morbidity and mortality outcomes, patient quality of life and also associated economic aspects (e.g. indirect medical costs, non-medical costs…).

Health Services Research

Targeting drugs to patients’ and physicians’ needs, as well as to public health systems, requires a sound knowledge of treatment pathways, patterns of drug use, and health services utilisation. We conduct various types of health services research to inform these decisions, through patient surveys, physician surveys and chart reviews.

Impact on drug and resource use

Benefit-risk management plans often rely on the evaluation of the use of drugs and of health technologies.  So-called ‘risk sharing agreements’ and ‘managed entry schemes’ often entail the commitment by industry to assess the actual use of resources after launch, in the real life practice.

LASER ANALYTICA has extensive experience in conductung impact studies with direct contact and follow-up of patients by telephone interviews. When necessary, the operational services of LASER C.R.O. are utilized to conduct these studies (however, other resources and external CROs may be used as well)

Qualitative Research

To get a complete appreciation of the unmet/uncovered needs of patients with a particular disease, to define the most important needs, the best market access roads and the commonalities and differences between groups, we also use qualitative research methods like focus groups, Delphi panels and semi-structured interviews in addition to surveys. We use CAQDAS programs (“Computer Assisted Qualitative Data Analysis Systems”) to improve the processes of storage, organization, analysis and presentation of qualitative data.

Patient-Reported Outcome Measures

The formulation of an overall PRO strategy for a development compound is a critical step in the development of a high-quality product value proposition package. The added value of PROs to the product development strategy is based on the use of high quality scales that address constructs of interest to the target audience.

LASER ANALYTICA helps customers choose the best tools for their needs (HRQoL, satisfaction with treatment, adherence or symptom measures) and to solve any problems or questions related to these instruments. We perform cultural adaptations of PROs across different countries and we design and carry out validation studies to assess their psychometric properties (reliability, validity and sensitivity to change). To address diseases for which there are no available robust tools to use,  we offer to develop a new one.

We apply Classic Theory, Rasch Model and Item Response Theory (IRT) in developing and validating PROs. Moreover, we have experience in developing Computer Adaptive Test (CAT) based on IRT.

Patient-Reported Outcome Measures & Quality of Life
  • Development and validation of specific QoL questionnaires
  • Incorporating QoL in pragmatic trials and observational studies
  • Questionnaires based on item response theory
  • Patient satisfaction with medication & patient preferences

 

Utility measurement & preferences studies

There’s a growing interest in developing specific utility measurements that include the range of health states associated with each specific disease. LASER ANALYTICA applies modern approaches to assess the preferences of public or patients about the specific health states of such a measure in order quantify the associated utility value.

We also carry out observational studies to discover patients’ preferences about different treatment alternatives, allowing the voice of patients to be heard in assessing the value of healthcare options.

Utilities
  • Development of specific utility measures
  • Preferences studies to weigh utility measures

 

LASER ANALYTICA applies classic quantitative methods in preference elicitation games: ranking, rating and choice- based methods, using Conjoint Analysis, Standard Gamble, Time Trade-off or willingness to pay. We also use our deep understanding of qualitative methodology to study patient preferences in order to include the patient’s point of view in assessing the value of new products. Another application of our knowledge on preference elicitation is the valuation of disease-specific health states in order to obtain specific utility measurements to be included in health economic analysis.

Data Solutions

LASER ANALYTICA has cutting-edge knowledge and experience in the development and analysis of observation systems, using electronic health care databases (eHCD), electronic medical records (eMR) and its own unique resource (PGRx), to investigate:

  • Impact of medicines and health technologies on drug and resource utilization
  • Comparative/relative effectiveness of medicines & health technologies
  • Relative risk and continuous benefit-risk of pharmaceuticals

These studies are conducted in a variety of contexts to inform decisions such as:

  • Post-authorization efficacy/effectiveness studies (EMA’s PAES & FDA Post marketing commitments)
  • Post-authorization safety studies (EMA’s RMPs & FDA REMS)
  • Continuous benefit-risk assessment
  • EU HTA dossiers and US payers
  • Coverage with evidence development
  • Market access evaluations
  • Rapid response to alerts and crisis management

One of our unique services consists in linking these studies of observation systems to advanced modeling in so-called access data platforms built for bridging-to-real life studies.

These services are served by the reliance on our advanced pharmacoepidemiology, data management and statistical teams.

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e-HCD & eMR

LASER staff pioneered the use of electronic health care databases (eHCD) and electronic Medical Records (eMR) for the conduct of pharmacoepidemiology studies (from Saskatchewan & VAMP Research data back in the late 80s to CPRD and now large US databases). Studies of comparative effectiveness and relative risk using advanced methods are conducted with these databases. Also used for market access studies and bridging-to-real life studies, these databases are used more and more for advanced pharmacoepidemiological research.

Selected experience with eMRs & eHCDs

  • US – Medstat, Premiere, Ingenix, Ford Health System, LabRx, others
  • UK – CPRD, THIN
  • France – EDB
  • Canada – RAMQ, Saskatchewan, Ontario
  • Germany – BKK sickness funds databases
  • Netherlands – PHARMO
  • Denmark – National eHCD (psychiatry)
  • Sweden – National eHCD
  • Multiple disease specific registries

Pharmacoepidemiologic General Research eXtension (PGRx)

Real world datasets for multiple purposes

No. 1 datasets for continuous benefit-risk assessment now available for HEOR studies & performance assessment

First developed for the study of the safety and risk of medicines, PGRx datasets were rapidly applied to the study of the comparative effectiveness of drugs in the prevention of morbidity and mortality outcomes. They now also incorporate information of special interest for health economics and payers’ decision purposes.

Case-based data for risk & effectiveness assessment

PGRx datasets allow in-depth analysis and regulatory-grade studies, as part of RMP, REMS, Continuous Benefit/Risk assessment, Post-Authorisation Safety Studies (PASS), Post-Authorisation Efficacy Studies (PAES), Coverage with Evidence Development (CED) or rapid response to alerts. PGRx data were used for pioneer continuous benefit-risk assessments and are the No1 primary data collections cited for that purpose on the ENCePP site.

PGRX1

New data for HEOR studies and decisions

Data collected by PGRx also provides in-depth information for the study of treatment pathways, clinical decisions, and patient journeys useful in predictive modeling, bridging studies, population impact assessment, cost-effectiveness studies, budget impact and other economic appraisals. PGRx data were used for pioneer outcome- based performance plans and provide a unique opportunity to expedite market access

Both medical & patient data

The only datasets to offer a possibility of follow-up and linkage

Using standardised data collection tools that have been validated in multiple studies, the PGRx Observation System offers a unique opportunity for obtaining extremely high quality data in real time, combining both medical information and patient reported outcomes

Medical Information

Data are collected from medical records and/or directly from physicians, providing detailed information on diagnoses and associated subconditions, laboratory tests, imaging and other diagnostic procedures, medication use and other healthcare resources.

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Patient Voice

A unique feature of PGRx data is that a linked subsample of patients are surveyed directly through autoquestionnaires or interviews on their drug use, adherence to treatment, including OTC drug use, personal history, risk factors, quality of life and outcomes. Patient consent allows follow-up and linkage to electronic records from other sources.

5 of the top 8 major pharma manufacturers and 15 mid-size companies and biotechs already use PGRx

Datasets in multiple therapy areas

PGRx licenses data from our networks of centers across Europe and North America, maintaining data collection in multiple therapy areas. More than 20 disorders are presently available in PGRx, from a dozen therapy areas. The list is constantly up-dated and increased in response to need.

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The PGRx deliverables

With PGRx Real World Datasets we commit to deliver:

  • Fast, real time data: the PGRx System provides data in a few weeks on almost any disorder or clinical sub-population, thereby allowing rapid responses to any information need
  • Credible data: PGRx provides high specificity of disease diagnosis and uses only validated questionnaires on patient reported outcomes and drug use
  • Ethical data: PGRx data collection being approved by ethics committees, and samples collected in respect of epidemiological principles, the data can be used for peer-review publications and reports to health authorities
  • Powerful data: because only cases of interest are collected, and with a high positive predictive value on all parameters considered, the PGRx datasets are as powerful as very large databases or surveys that eventually provide only a few really relevant sets of data.
  • Efficient data: the costs of PGRx data is fixed and includes all expenses and fees, including investigators’ fees. The multiple users of the system, and sometimes of the same data, allows very significant savings.

*The PGRx trade name belongs to Lucien Abenhaim and Lamiae Grimaldi. The use of the systematic case-referent methodology is on free access.