Secured Access to innovative medicines for CHildren, adolescents and young adults with cAncer

First published 01/02/2024

Last updated 10/06/2024

Data source

Drug registry

Spontaneous reporting of adverse drug reactions

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The data source contains the following information

Disease information Does the data source collect information with a focus on a specific disease? This might be a patient registry or other similar initiatives.: Yes

Disease details, other: Collection of real-world safety and efficacy data of compassionate and off label used innovative anti-cancer therapies, with adequate pharmacovigilance declaration. For off-label therapies, they are only collected if first approved in adults in Europe after the implementation in 2017 of the EU Pediatric Regulation (1901/2006/EC)

Rare diseases Are rare diseases captured? In the European Union a rare disease is one that affects no more than 5 people in 10,000.: Yes

Pregnancy and/or neonates Does the data source collect information on pregnant women and/or neonatal subpopulation (under 28 days of age)?: No

Hospital admission and/or discharge: No

ICU admission Is information on intensive care unit admission available?: No

Cause of death: Captured

Cause of death vocabulary: Other

Cause of death vocabulary, other: Cancer, AE related to drug of interest, other reason (and then free text)

Prescriptions of medicines: Captured

Prescriptions vocabulary: DrugBank

Dispensing of medicines: Captured

Dispensing vocabulary: DrugBank

Advance therapy medicinal products (ATMP) Is information on advanced therapy medicinal products included? A medicinal product for human use that is either a gene therapy medicinal product, a somatic cell therapy product or a tissue engineered products as defined in Regulation (EC) No 1394/2007 [Reg (EC) No 1394/2007 Art 1(1)].: No

Contraception Is information on the use of any type of contraception (oral, injectable, devices etc.) available?: No

Indication for use Does the data source capture information on the therapeutic indication for the use of medicinal products?: Captured

Indication vocabulary: Other

Indication vocabulary, other: ICCC-3

Medical devices Is information on medicinal devices (e.g., pens, syringes, inhalers) available?: No

Administration of vaccines: No

Procedures Does the data source capture information on procedures (e.g., diagnostic tests, therapeutic, surgical interventions)?: Captured

Procedures vocabulary: Not coded (Free text)

Clinical measurements Is information on clinical measurements (e.g., BMI, blood pressure, height) available?: Yes

Healthcare provider Is information on the person providing healthcare (e.g., physician, pharmacist, specialist) available? The healthcare provider refers to individual health professionals or a health facility organisation licensed to provide health care diagnosis and treatment services including medication, surgery and medical devices.: Yes

Genetic data Are data related to genotyping, genome sequencing available?: Captured

Genetic data vocabulary: Other

Genetic data vocabulary, other: Drop-down box values

Biomarker data Does the data source capture biomarker information? The term “biomarker” refers to a broad subcategory of medical signs ( objective indications of medical state observed from outside the patient), which can be measured accurately and reproducibly. For example, haematological assays, infectious disease markers or metabolomic biomarkers.: Captured

Biomarker data vocabulary: Other

Biomarker vocabulary, other: Drop-down box values

Patient-reported outcomes Is information on patient-reported outcomes (e.g., quality of life) available?: No

Patient-generated data Is patient-generated information (e.g., from wearable devices) available?: No

Units of healthcare utilisation Are units of healthcare utilisation (e.g., number of visits to GP per year, number of hospital days) available or can they be derived? Units of healthcare utilisation refer to the quantification of the use of services for the purpose of preventing or curing health problems.: Yes

Unique identifier for persons Are patients uniquely identified in the data source?: No

Diagnostic codes: Captured

Diagnosis / medical event vocabulary: Other

Diagnosis / medical event vocabulary, other: ICCC-3

Medicinal product information: Captured

Medicinal product information collected: Brand name
Dose
Formulation
Route of administration

Medicinal product vocabulary: Other

If 'other,' what vocabulary is used?: Drop-down box values

Quality of life measurements: Captured

Quality of life measurements vocabulary: Not coded (Free text)

Lifestyle factors: Not Captured

Sociodemographic information: Captured

Sociodemographic information collected: Age
Country of origin
Gender