| Compound |
Area of Interest and Areas of Non-Interest |
| enzalutamide |
- New approaches for treatment of prostate cancer, including drug and non-drug (modality) combinations
- Research in early stages of prostate cancer
- Adverse event management under standard enzalutamide dosing
- Biomarkers to inform response, resistance, and treatment decisions
- Patient reported outcomes and quality of life in prostate cancer
- New screening, artificial intelligence, & diagnosis technology in conjunction with prostate cancer treatment
- Understanding mechanisms of androgen receptor inhibitor action and resistance
- Treatment of oligometastatic disease
The following proposal types will not be considered at this time:
All tumor types other than prostate cancer, country-specific studies where there is no rationale for data generation in sub-populations, healthcare resource utilization (cost analysis), and unapproved enzalutamide dosing
|
| gilteritinib |
- FLT3 mutation positive (FLT3 m+) malignancies, acute myeloid leukemia (AML) and other
- Targeted drug combinations with gilteritinib in FLT3 mutation positive (FLT3 m+) acute myeloid leukemia (AML)
- Maintenance therapy in FLT3 mutation positive (FLT3 m+) acute myeloid leukemia (AML)
- Minimal residual disease and impact on treatment outcomes in gilteritinib treated acute myeloid leukemia (AML) patients
The following proposal types will not be considered at this time:
Wild type FLT3 acute myeloid leukemia (AML), paediatric studies, mechanisms of resistance, allelic ratio, bridge to transplant, and combination with other FLT3 inhibitors, sequencing with FLT3 inhibitors and head-to-head studies with other FLT3 inhibitors
|
| enfortumab vedotin (EV) |
Practice Informing:
- Retreatment
- In patients who previously responded to EV (MIBC, la/mUC)
- Biology of retreatment (e.g. changes in nectin-4 expression)
- AE prevention, treatment /mitigation (aligned to labeled recommendations)
- Optimizing initiation of treatment and sequencing
Proof of Concept:
- la/mUC
- Combinations/ sequences with targeted therapies
- Nectin-4*** expressing tumors with scientific rationale/ unmet need
- Of interest but not limited to: Gyn tumors, Thyroid, GI not included in EV-202 Trial**, others as appropriate^
Biomarkers/Health Economics Outcomes Research (HEOR):
- Biology of Resistance pathway/ overcoming resistance pathways
- Biology of Nectin-4: expression dynamics***
- HEOR
- Non-muscle-invasive bladder cancer (NMIBC) claims data identification of high risk/low risk patients
- Uptake of novel therapies and impact on NMIBC treatment patterns
- Support new innovative modeling methods for combination therapy
- Patterns and outcomes of cycling (reuse) of treatment
The following proposal types will not be considered at this time:
Duplicative with development program/ISRs, Evaluation of altered route, dose or schedule, Combinations with unapproved therapies*, RCT (may be exceptions), 1L maintenance in la/mUC, Tumors in EV-202 trial**, NMIBC (clinical investigation), PD markers/response predictors, Economic modeling, Health resource, cost and outcomes with EV.
* For evaluation by business development.
**Any stage, all tumor associated subtypes and combination until tumor development plan is implemented. Tumors included in EV-202 Trial are: HR+/HER2- Breast Cancer, Triple Negative Breast Cancer (TNBC), Squamous Non-Small Cell Lung Cancer (SNSCLC), Non-Squamous Non-Small Cell Lung Cancer (NSNSCLC), Head and Neck Cancer and Gastric, Gastroesophageal Junction or Esophageal Cancer.
*** all nectin-4 testing to be done by Q2;
^ For tumors without Nectin-4 expression data, evaluation of Nectin-4 should be done.
|
| isavuconazonium sulfate |
- Real world experience, including pharmacokinetic data, regarding the use of isavuconazonium sulfate with new molecules used in oncology and immunology
- Real-world evidence/experience, including incidence and outcome, of invasive pulmonary aspergillosis and other invasive fungal infections in non-traditional hosts, such as post-severe influenza/ARDS, COVID-associated secondary infections, etc.
- Real-world evidence/experience regarding management of complicated/serious invasive fungal infections with combination antifungal therapy
- Use of isavuconazonium sulfate for prophylaxis
- Use of isavuconazonium sulfate in the treatment of endemic fungi
- Paediatric studies
|
| mirabegron |
- Real World Evidence research to confirm clinical trial results (Databases, registries, networks of care analysis for treatment patterns, clinical outcomes, interventional prospective, non-interventional and retrospective data analysis)
- Studies to support personalized medicine in overactive bladder (OAB), including clinical phenotyping, biomarkers, prediction tool, etc.
- Mirabegron in patients with comorbidities (e.g. subgroups of patients in regular practice, overweight overactive bladder patients (OAB), male patients with benign prostatic hyperplasia (BPH), male or female overactive bladder OAB patients with impaired sexual function)
- Patient’s self-administered overactive bladder (OAB) management tool
- New Patient Reported Outcomes
- Combination treatment with mirabegron (e. g. with botulinum toxin A (Botox), percutaneous tibial nerve stimulation (PTNS) and sacral nerve stimulation (SNS))
- Impact of the timing of mirabegron dose on nocturia (e.g. morning vs. evening dosing)
- Epidemiological studies in the evolution of overactive bladder (OAB) prior to diagnosis and treatment
The following proposal types are not being considered at this time:
Clinical trials in the current indication or paediatric overactive bladder and neurogenic detrusor over-activity.
|
| ipragliflozin |
- Clinical and mechanistic studies regarding the effects of sodium-glucose co-transporter 2 inhibition with ipragliflozin associated type 1 diabetes mellitus.
- Real World Evidence research Observational research regarding the effects of SGLT-2 inhibition on micro / macro vascular complications in patients with T2DM and T1DM. (Databases, registries, clinical outcomes, non-interventional and retrospective data analysis).
The following proposal types are not being considered at this time:
Comparator studies with other sodium-glucose co-transporter 2 inhibitors
|
| roxadustat |
- Clinical efficacy outcomes
- Special populations
- patients with CKD anemia with a functioning renal transplant
- HIV+ patients with anaemia of CKD (South Africa)
- Iron metabolism
- Treatment patterns
- Patient Reported Outcomes (PROs): adherence and persistence
- Mechanistic and biomarker studies
- Renal anemia/anemia of chronic kidney disease epidemiology
The following proposal types are not being considered at this time:
Off-label use studies, studies with roxadustat with safety as the primary endpoint (due to required size and length of such study), chronic kidney disease progression, and single-arm interventional studies
|
| tacrolimus |
ADVAGRAF: Real World Evidence research including:
- Database, registries, networks of treatment pattern of long-term clinical outcomes in liver/kidney transplantation (non-interventional and retrospective data)
- Conversion from Prograf to Advagraf, de novo use of Advagraf (e.g. dose, trough level, renal function)
- Risk factors evaluation related to treatment outcomes, (e.g. intrapatient variability, adherence)
- COVID-19 related
PROGRAF:
- Epidemiological studies in those countries who have launched/pre-launch autoimmune disease indication
- Efficacy and safety outcome of Prograf treatment on auto-immune patients.
The following proposal types are not being considered at this time:
Very low dose tacrolimus regimens (e.g. <3ng/ml), comparison studies between Prograf & Advagraf.
Please note: Caution with very low dose tacrolimus regimens (e.g. <3ng/ml) due to concerns with immunological injury or in studies with switch to Advagraf when tacrolimus levels are already low (e.g. <5ng/ml).
|
