Study Type/s

Patient-level characterisations are classified as ‘off the shelf’ 

Study Design

Cohort analysis.

Participant/s

Patient-level characterisation studies will include one or more cohort/s of people newly diagnosed with 1 or more pre-specified condition/s and with some amount of data visibility before diagnosis, and with no record of the same condition/s in the previous year (or in all previous history).

Additional eligibility criteria could apply as follows, to be incorporated as sensitivity analyses:

• Additional restriction/s could apply based on socio-demographics, e.g., people aged 18 or older at the time of diagnosis
• Additionally, people with a competing (differential) diagnosis could also be excluded (e.g., people with rheumatoid arthritis with a history of psoriatic arthritis could be excluded to minimise misclassification)

Follow-up

Participants will be followed up from their date of new diagnosis (index date) until the earliest of the following: loss to follow-up, end of data availability, a pre-specified time period (e.g. 1 year after index date) or death.

Analyses

Details will be discussed during programming of pipelines, but it is likely that patient-level characterisation will include:

• Automated large-scale characterisation, including all recorded baseline characteristics available in the data before or on index date, based on code/s, and classified into conditions (medical history), medicine/s use, and procedure/s
• Pre-specified patient-level characteristics on and/or before index date, based on pre-existing code lists or definitions (e.g., history of type 2 diabetes, or Charlson comorbidity index)
• Pre-specified patient-level characteristics on and/or before index date, based on concepts and descendants where no previously validated algorithms are available
• Incidence rate/s of pre-specified outcome/s within a pre-specified time period (e.g. 1 year)
• Prognosis / progression to a pre-specified outcome within a pre-specified time, e.g., cumulative incidence of certain events or mortality within 1- or 5-years after diagnosis
• Standard care description, including n (%) receiving each of a pre-specified list of medicine/s, device/s or procedure/s, and combinations within a pre-specified time window after diagnosis

Study Type/s

Patient-level DUS analyses are classified as ‘off the shelf’ studies. Patient-level DUS will offer the possibility to include population-level DUS analyses as part of the same analysis.

Study Design

New drug/s user cohort

Participants

Patient-level DUS analyses will include one or more cohort/s of incident drug users with at least 1 year of data visibility, and no use of that same drug/drug class in that previous year.

Additional eligibility criteria could apply as follows:

• Source population could be restricted to a specific subpopulation with certain socio-demographic or clinical feature/s, e.g., people with a diagnosis of rheumatoid arthritis who then start to take a disease-modifying anti-rheumatic drug (DMARD)
• Additional restriction/s could apply as per product label, indication, or study aim/s, e.g., people aged 18 or older at the time of therapy initiation

Follow-up

Participants will be followed up from the date of therapy initiation (index date) until the earliest of loss to follow-up, end of data availability, or death. Patients might be censored at the time they discontinue treatment or switch to an alternative therapy.

Outcome/s

The following outcome/s will be obtained, potentially stratified by pre-specified criteria (age bands, sex, calendar year or month), and other pre-specified criteria:

• New drug user cohort/s patient-level characteristics on or before index date
• Indication (where available)
• Initial dose/strength (as prescribed/dispensed at therapy initiation, where available)
• Cumulative use within a pre-specified time period (e.g. 1 year) based on number of prescriptions and dose/strength
• Treatment duration
• Count of repeated prescriptions during a pre-specified time period (e.g. 1 year)

Analyses

Patient-level DUS analytics will include:

• Large-scale characterisation of patient-level features based on code/concept and descendants, including socio-demographics, comorbidity, and previous medicine/s use any time in history, and in the year, and/or in the month previous to index date
• Frequency and % of indication/s, based on pre-specified list of diagnoses recorded before therapy initiation (where available)
• Reporting of minimum, p25, median, p75, and maximum initially prescribed or dispensed dose/strength (where available)
• Reporting of minimum, p25, median, p75, and maximum cumulative use within a pre-specified time period (e.g. 1 year)
• Reporting of minimum, p25, median, p75, and maximum treatment duration
• Reporting of minimum, p25, median, p75, and maximum number of repeated prescriptions of the index drug during a pre-specified time period (e.g. 1 year)

Study Type

Population-level DUS analyses are classified as ‘off the shelf’ studies.

Study Design

Population-level cohort.

Participants

Population-level analyses will include the entire source population with at least some time (typically 1 year) of data visibility available before start of study period.

Additional eligibility criteria will apply as follows:

• Analyses of incidence of drug use will exclude prevalent users of the same drug/drug class on index date and/or in the previous (washout) year
• The study population could be restricted to a specific subpopulation with certain socio-demographic e.g., age 18 or older, or with a history of a pre-specified clinical feature/s, e.g., people with a prior diagnosis of rheumatoid arthritis
• In some cases, a minimum follow-up will be requested e.g., for treatment pattern analyses

Outcome/s

The following outcome/s will be obtained, potentially stratified by pre-specified criteria (age bands, sex, calendar year or month):

• Population-based incidence rates of use of a drug/drug class over calendar time. Periods could be calendar  days, weeks, months, quarters or years.
• Population-based prevalence of use of a drug/drug class on a given time point (point prevalence) or within a given time period (period prevalence). Periods could be calendar days, weeks, months, quarters or years.

Follow-up

Follow-up will start on a pre-specified calendar time point pre-defined as index date , e.g., 1^st January or 1^st of each month, and continue for a pre-specified time period, typically week, month, quarter or year.

Analyses

Population-level DUS analyses use the same analytical pipeline as Population-level descriptive epidemiology studies (see separate subsection). Incidence rates will have number of new users (with a pre-specified washout) in the numerator, and total population as person-years (except prevalent users) in the denominator. Prevalence will be calculated as number of users (prevalent or new) over whole source population at a specific time point (i.e., point prevalence) or over a specific time window (i.e., period prevalence). Both may be stratified by socio-demographics (e.g., age bands or sex) and/or calendar period. Additional criteria (e.g. disease severity/duration) may need to be considered and integrated as pre-specified in future studies.

Study Type/s

Population-level descriptive epidemiology are classified as ‘off the shelf’ studies.

Study Design

Population-level cohort

Participant/s

Population-level analyses will include the entire source population with at least some time (typically 1 year) of data visibility available before index date.

Additional eligibility criteria will apply as follows:

• Analyses of disease incidence will exclude patients with a previous/prevalent history of the same disease on index date and/or in the previous (washout) year and/or in all previous history
• The source population could be restricted to a specific subpopulation with certain socio-demographic or clinical feature/s, e.g., people aged 50+ on index date

Outcome/s

The following outcome/s will be obtained, potentially stratified by pre-specified criteria (age bands, sex, calendar year or month), and other pre-specified criteria:

• Population-based incidence of a disease/condition (or group of diseases/conditions) on a given time point or over time (stratified by calendar period)
• Population-based prevalence of disease (or group of diseases/conditions) on a given time point (e.g., a pre-specified date), and/or over time (e.g., stratified by month or year)

Follow-up

Follow-up will start on a pre-specified calendar time point pre-defined as index date , e.g., 1^st January or 1^st of a given month, and continue for a pre-specified time period, typically a week, a month or a year

Analyses

Incidence rates will have number of newly diagnosed people in the numerator, and total population (satisfying the study eligibility criteria) in the denominator. Prevalence will be calculated as number of people with the diagnosis (prevalent or new) over whole source population on a specific date (point prevalence) or over a window of time (period prevalence). Both can be stratified by socio-demographics (e.g., age bands or sex) and/or calendar period. Other criteria (e.g. disease severity/duration) may need to be considered and integrated in future studies.