Table 1 Checklist: what type of information should be included in a RDR to inform clinical trial design

Basic characterization [12]

Patient characteristics

Once, baseline

For (coded) identification of individual patients

Specific id per person is needed for all analyses

Patient id, date of birth

Demographic characteristics

Once, baseline

To classify patient, information for patient retrieval, for trial feasibility assessment and/or to be aware of other factors possibly associated with outcome and to be able to adjust for it

For general ascertainment, historical controls, and post-marketing phase

Gender, age, ethnicity, country

Disease aspects [12]

Diagnosis

Once, baseline

To classify genotype and/or phenotype, for trial feasibility assessment and/or to be aware of other factors possibly associated with outcome and to be able to adjust for it

For general ascertainment, historical controls, and post-marketing phase

Date of first symptoms/diagnosis, genetic test results, type/staging of disease

Co-morbidities

Once, baseline or when it is diagnosed, and if severity is worsening

For trial feasibility assessment and/or to be aware of other diagnoses possibly associated with disease and/or treatment effect,

For general ascertainment

Concurrent diagnoses, e.g. renal, cardiovascular or psychiatric problems

Treatment

Repeated

For trial feasibility assessment and/or to be aware of other factors possibly associated with outcome and to be able to adjust for it

For general ascertainment, historical controls, post-marketing phase

Off-label treatment, surgery, dosage

Outcome variables (either for efficacy or safety) [14, 54]

Mortality

Once, when it occurs

To assess change in disease course over time (relevance depends on disease)/ To collect information on (un)expected, possible side-effects of treatment

For (Bayesian) sample size calculation, historical controls, and post-marketing phase

Survival time, age of death

Life impact

repeated

To assess change in disease course over time at the personal level/ To collect information on (un)expected, possible side-effects of treatment

For (Bayesian) sample size calculation, historical controls, and post-marketing phase

Symptom status, functional status, general health perceptions, quality of life, cognitive functioning, hospitalization

Pathophysiological manifestations

repeated

To assess change in disease course over time and phenotype /To collect information on (un)expected, possible side-effects of treatment

For (Bayesian) sample size calculation, historical controls, and post-marketing phase

Organ function, biomarkers, allergic reaction