Table 4 Mapping COS onto RWD sources in selected European countries: an exercise in feasibility
Outcome classification/Outcomes | Linked administrative databases (Finland, Norway, Sweden, Hungary, Italy) | Prostate Cancer data Base Sweden (PCBaSe): National Prostate Cancer Register (NPCR) linked to administrative databases (Sweden) | ||||||
|---|---|---|---|---|---|---|---|---|
High-quality study | COS for practice | Hospital discharges (H) | Mortality (M) | Purchased medication (PM) | Notes | Notes | ||
Mortality and survival | ||||||||
Survival | √ | √ | Requires identifying patients in H and measuring mortality in M | √ | NPCR D plus Mortality PCBaSe | |||
√ | √ | Overall survival | √ | √ | Requires identifying patients in H and measuring mortality in M | √ | NPCR D plus Mortality; PCBaSe | |
√ | √ | Cause (disease) specific survival | √ | √ | Requires identifying patients in H and measuring cause of death in M | √ | NPCR D plus Mortality; PCBaSe | |
Relative survival | √ | √ | Requires comparing the above measures to a similar population (by age, sex) without the disease | √ | NPCR D plus Mortality; PCBaSe | |||
√ | √ | Metastasis-free survival | √ | √ | Requires measuring survival in patients for whom no metastatic carcinoma codes (codes 196–199) or secondary cancers are discernible over time | √ | NPCR 5yrf-up; PCBaSe | |
Progression-free survival | √ | √ | Requires stratifying patients for evidence of progression compared to those without, using codes identified as indicating disease progression (e.g., castration-resistant disease, metastases, recurrence) | √ | NPCR 5yrf-up; NCPR Tx; PCBaSe | |||
√ | √ | Biochemical recurrence-free survival | No clinical test data is available | √ | NPCR 5yrf-up includes PSA levels at diagnosis and over time (changes), some indications in NPCR RT or RP | |||
Outcomes relating to neoplasms | ||||||||
(Change in) prostate-specific antigen (PSA) levels | Clinical test data are not available but the code for elevated prostate specific antigen (PSA) (e.g., 790.93) could be checked over time in the H | √ | NPCR 5yrf-up includes PSA levels at diagnosis and over time (changes), some indications in NPCR RT or RP | |||||
Measurable disease response | Clinical data are not available | Might be possible using NPCR, but only RT and RP forms collect imaging data | ||||||
Time to progression | √ | Requires identifying codes for indicating progression (e.g., castrationresistant disease, metastases, recurrence) | √ | NPCR 5yrf-up; NPCR Tx; PCBaSe | ||||
√ | Disease progression/Progression rate | √ | Requires identifying codes for indicating progression (e.g., castrationresistant disease, metastases, recurrence) | √ | NPCR 5yrf-up; NPCR Tx; PCBaSe | |||
Progression-free probability | √ | Requires measuring probability using the outcome above | √ | NPCR 5yrf-up; PCBaSe | ||||
√ | √ | Development of metastases | √ | Requires identifying codes for metastatic carcinoma (196–199) or secondary cancers | √ | NPCR 5yrf-up; PCBaSe | ||
Metastases-free probability | √ | Requires measuring probability using the outcome above | √ | NPCR 5yrf-up; PCBaSe | ||||
√ | √ | Symptomatic skeletal event | √ | Requires identifying codes for pathologic fracture (e.g., 733.1), surgery or radiation to bone (e.g., ICD9 procedure codes indicating bone surgery, 79*) or radiation (e.g., ICD9 V58.0) combined with bone diagnoses, or spinal cord compression (e.g., 336.9) | √ | NPCR 5yrf-up; PCBaSe | ||
√ | Local disease | Local disease recurrence would be difficult to discern, except the case where patients with established diagnoses (e.g., 185) over time, present later with an in situ diagnosis (233.4) | Difficult to discern but could follow technique at left; also may be helpful to stratify patients according to NPCR Tx. | |||||
√ | Positive surgical margins | √ | NPCR 5yrf-up; NPCR RP | |||||
Response duration | ||||||||
Failure-free probability | ||||||||
Development of castration-resistant disease20 | √ | √ | Requires identifying patients with evidence of surgical castration (ICD9 procedure codes 62.3, 62.41, 62.42) or medical castration using ADT (e.g., ATC code L02BB03), noting also abiraterone (ATC L02BX03), and disease progression (e.g., metastases codes 196–199) subsequent to ADT or abiraterone; check also for elevated PSA (e.g., 790.93). | √ | NPCR 5yrf-up; NPCR RP subgroup; PCBaSe | |||
√ | Treatment failure | √ | √ | √ | Requires measuring mortality and/or evidence of disease progression (e.g., metastases) for groups of patients stratified for various treatments (e.g., radical prostatectomy, ADT) | √ | NPCR 5yrf-up for failure of conservative therapy; PCBaSe | |
Renal and urinary outcomes | ||||||||
√ | √ | Urinary incontinence | √ | Requires identifying relevant codes (e.g., 788.30) | √ | NPCR PROMs; PCBaSe | ||
√ | √ | Urinary obstruction/irritation | √ | Requires identifying relevant codes (e.g., 599.60) | √ | NPCR PROMs; PCBaSe | ||
√ | √ | Urinary symptoms | √ | Requires identifying relevant codes (e.g., 788*) | √ | NPCR PROMs; NPCR 5yrf-up; PCBaSe | ||
Voiding behaviour | √ | Requires identifying relevant codes (e.g., 596.59, 788.69) | √ | NPCR PROMs; NPCR 5yrf-up; PCBaSe | ||||
Haematuria | √ | Requires identifying relevant codes (e.g., 599.7) | √ | PCBaSe | ||||
Pelvic pain | √ | Requires identifying relevant codes (e.g., 608.9) | √ | PCBaSe | ||||
Lymphedema | √ | Requires identifying relevant codes (e.g., 457.1) | √ | PCBaSe | ||||
√ | Urinary functioning | √ | Requires identifying relevant codes (e.g., 788*) | √ | NPCR PROMs; NPCR 5yrf-up; PCBaSe | |||
Gastrointestinal outcomes | ||||||||
√ | √ | Bowel symptoms | √ | Requires identifying relevant codes (e.g., 787.99) | √ | NPCR PROMs; NPCR 5yrf-up; PCBaSe | ||
√ | Faecal incontinence | √ | Requires identifying relevant codes (e.g., 787.6) | √ | NPCR PROMs; PCBaSe | |||
√ | Bowel functioning | √ | Requires identifying relevant codes (e.g., 787.99) | √ | NPCR PROMs; NPCR 5yrf-up; PCBaSe | |||
Diarrhoea | √ | Requires identifying relevant codes (e.g., 787.91, 564.5) | √ | PCBaSe | ||||
Endocrine outcomes | ||||||||
√ | √ | Hormonal symptoms | √ | √ | Requires identifying relevant codes related to side-effects from hormonal treatments (e.g., fatigue 780.79, weight loss 783.21) in patients with evidence of ADT (e.g., ATC code L02BB03), checking for chemotherapy with docetaxel (ATC code L01CD02) in PM. | √ | NPCR 5yrf-up; NPCR Tx; PCBaSe | |
Reproductive system outcomes | ||||||||
√ | Erectile/sexual function | √ | Requires identifying relevant codes (e.g., 607.84 and/or procedure codes 60.94, 60.95, 60.96, 60.97) | √ | NPCR PROMs; PCBaSe | |||
√ | √ | Erectile/sexual dysfunction (impotence) | √ | Requires identifying relevant codes (e.g., 607.84 and/or procedure codes 60.94, 60.95, 60.96, 60.97) | √ | NPCR PROMs; PCBaSe | ||
√ | √ | Sexual symptoms | √ | Requires identifying relevant codes (e.g., 607.84 and/or procedure codes 60.94, 60.95, 60.96, 60.97) | √ | NPCR PROMs; PCBaSe | ||
General outcomes | ||||||||
√ | √ | Pain | √ | √ | Requires identifying relevant codes (e.g., 338*, 780.96, various) in H, ATC codes (N02*) in PM | √ | PCBaSe | |
√ | √ | Fatigue | √ | Requires identifying relevant codes (e.g., 780.79) | √ | PCBaSe | ||
Weight loss | √ | Requires identifying relevant codes (e.g., 783.21) | √ | PCBaSe | ||||
Bone pain | √ | √ | Requires identifying relevant codes (e.g., 733.90), though the code is not specific only to bone pain, suggesting a need for further identification of codes for pain (e.g., 388*) in H and ATC codes for pain medications (e.g., N02*) in PM | √ | PCBaSe | |||
Anaemia | √ | Requires identifying relevant codes (e.g., 285.9) | √ | PCBaSe | ||||
√ | √ | Performance status | ||||||
Physical functioning | ||||||||
√ | √ | Physical wellbeing/functioning | √ | NPCR PROMs | ||||
Emotional functioning/wellbeing | ||||||||
√ | √ | Mental/emotional wellbeing/functioning | Not possible, although some measures of well-being, such as depression codes (e.g., 311, 2962 or 2963) in H or anti-depressives (ATC code N06A) in PM could be identified. | √ | NPCR PROMs | |||
Social functioning | ||||||||
Social functioning | ||||||||
Role functioning | ||||||||
Role functioning | ||||||||
Global quality of life | ||||||||
√ | Quality of life | √ | NPCR PROMs | |||||
Economic outcomes | ||||||||
Cost-effectiveness | √ | √ | Requires measuring costs in H, e.g., using DRG tariffs, and medication costs in PM, and constructing cost-effectiveness measures for various treatments and outcomes | √ | PCBaSe | |||
Costs | √ | √ | Requires measuring costs in H, e.g., using DRG tariffs, and medication costs in PM | √ | PCBaSe | |||
Need for intervention | ||||||||
√ | Need for salvage therapy | Salvage therapies might be partially identified by stratifying patients for therapies, e.g., radical prostatectomy (60.5 procedure code) followed by external beam radiotherapy (92.29 procedure code) or ADT (e.g., ATC L02BB03) | √ | NPCR 5yrf-up, to some extent; NPCR Tx; PCBaSe, to some extent | ||||
√ | Need for curative treatment | This outcome seems to refer specifically to patients under active surveillance, difficult to ascertain, other than by observing a total lack of treatment upon and after diagnosis, followed by eventual treatment after some time. | √ | NPCR 5yrf-up, related to questions regarding reasons for terminating conservative therapy | ||||
√ | √ | Need for pain medication | √ | √ | Requires identifying relevant codes for pain (e.g., 338*, 780.96) in H, or pain medication (ATC codes N02*) in PM | √ | PCBaSe | |
√ | √ | Procedures need for local progression | As described for “need for salvage therapy” or “local disease”, such measures could follow a similar logic. | PCBaSe, to some extent | ||||
Delivery of care | ||||||||
Time to treatment failure | ||||||||
Adverse events/effects | ||||||||
√ | √ | Adverse events | √ | √ | Requires stratifying patients by therapy and then measuring any adverse events, i.e., complications of surgical and medical care, not elsewhere classified (996-999), urinary complications (997.5), bowel obstruction (560.9) or effects of radiation, unspecified (990). See also “major systemic therapy effects”. | √ | NPCR 5yrf-up; NPCR Tx; NPCR RP; NPCR RT; NPCR PROMs; PCBaSe | |
√ | Perioperative deaths | √ | √ | Requires measuring in-hospital deaths for surgical admissions and deaths within a certain timeframe from surgery (M) to give a (potentially incomplete) indication of this outcome | √ | PCBaSe | ||
√ | Thromboembolic disease | √ | Requires identifying relevant codes (e.g., 451*, 453*), pulmonary embolism (415.1), peripheral arterial occlusion disease (443.9), deep vein thrombosis codes, especially for ADT patients. Possibly also related procedure codes (e.g., venography, 886*). | √ | PCBaSe | |||
√ | Bothersome or symptomatic urethral or anastomotic stricture | √ | Requires identifying relevant codes (e.g., 598.9, 997.49). | √ | NPCR 5yrf-up; NPCR PROMs; PCBaSe | |||
√ | Side-effects of hormonal therapy | √ | √ | Requires identifying relevant codes related to side-effects from hormonal treatments (e.g., fatigue 780.79, weight loss 783.21) in ADT patients (e.g., ATC code L02BB03). Also, see “Hormonal symptoms”. | √ | NPCR 5yrf-up; NPCR Tx; PCBaSe | ||
√ | √ | Major systemic therapy effects | √ | √ | Requires stratifying patients into groups with evidence of systemic therapy, i.e., hormonal therapy (e.g., ATC L02BB04 in PM), chemotherapy (e.g., docetaxel L01CD02 or cabazitaxel L01CD04 in PM), immunotherapy (e.g., sipuleucel-T L03AX17), treatments for bone metastases (e.g., denosumab M05BX53 in PM). Evidence of side effects are then sought in H and/or PM. | √ | NPCR 5yrf-up; NPCR Tx; PCBaSe | |