What is real-world evidence?
Real-world evidence (RWE) comprises data generated from real‑world clinical settings, derived from sources other than prospective, interventional clinical trials.
What is RWE & why does it matter?
RWE is important for determining whether survival improvements seen in randomised controlled trials are reflected in routine practice.1,2 In the real world, 40% of patients with newly diagnosed multiple myeloma may not be eligible for randomised controlled trials (RCTs) based on standard eligibility criteria.2
RWE can be derived from several different sources; these can include:1
- Electronic Health Records
- Chart review or manual data extraction
- Technology-enabled abstraction
- Claims Systems
- Claims data from billing
- Cancer Registries
- Retrospective data
- Prospective data
- Patient Reported Data
- Patient health surveys or questionnaires
- Sourced from patients directly (eg., wearable devices)
- Genomic databases
- Genome sequencing linked with real-world clinical data to create a clinico-genomic database

Therapies may have limitations in the community setting, associated with toxicity and patient burden.3 Despite demonstrating efficacy within rigorously controlled clinical trials, the same results are not always achieved in real-world practice.3
What is the difference between RWE and RCTs?
RCTs are the gold standard for therapeutic evaluation and regulatory decision-making but RWE demonstrates broader generalisability compared with RCTs1,4
Strengths and limitations of RCTs4

Approximately 40% of people living with MM are ineligible for RCTs.5
Patient populations typically under-represented in RCTs include those with comorbidities or advanced disease, elderly and frail patients.2,3-6
Strengths and limitations of RWE4

RWE complements clinical trial data
RWE investigates the performance of an intervention under real-world conditions.7
RWE can complement clinical data in several ways, including:
- Providing insights into patient characteristics, treatment outcomes and treatment practices in the real-world setting6,8,9
- Providing additional insights in evidence sometimes not addressed by RCTs, such as:
- Long-term effectiveness and safety data9
- Treatment outcomes in typically under-represented MM patient populations2-6

Adapted from Katkade VB, et al. 2018.10
Ixazomib FAQs
Ixazomib is an oral, highly selective and reversible proteasome inhibitor. Each capsule of Ixazomib contains Ixazomib citrate, a prodrug that rapidly hydrolyses under physiological conditions to its biologically active form, Ixazomib.1
1. Ixazomib. Summary of Product Characteristics.
Ixazomib in combination with lenalidomide and dexamethasone is indicated for the treatment of adult patients with multiple myeloma who have received at least one prior therapy.1
1. Ixazomib. Summary of Product Characteristics.
In England, IRd is recommended by NICE as an option for treating multiple myeloma in adults if they have already had two or three previous lines of therapy and the company provides Ixazomib according to the commercial arrangement.1 Ixazomib is also available to appropriate patients in Wales and Northern Ireland.2
1. NICE. Ixazomib with lenalidomide and dexamethasone for treating relapsed or refractory multiple myeloma. Available from www.nice.org.uk/guidance/TA870. Accessed March 2026
2. All Wales Therapeutics and Toxicology Centre. Ixazomib citrate. Available at: https://awttc.nhs.wales/accessing-medicines/medicine-recommendations/Ixazomib-citrate-ninlaro.
Ixazomib preferentially binds and inhibits the chymotrypsin-like activity of the beta 5 subunit of the 20S proteasome.1 Ixazomib induced apoptosis of several tumour cell types in vitro. Ixazomib demonstrated in vitro cytotoxicity against myeloma cells from patients who had relapsed after multiple prior therapies, including bortezomib, lenalidomide, and dexamethasone.1 The combination of Ixazomib and lenalidomide demonstrated synergistic cytotoxic effects in multiple myeloma cell lines. In vivo, Ixazomib demonstrated antitumour activity in various tumour xenograft models, including models of multiple myeloma. In vitro, Ixazomib affected cell types found in the bone marrow microenvironment including vascular endothelial cells, osteoclasts and osteoblasts.1
1. Ixazomib. Summary of Product Characteristics.
Ixazomib should be taken in combination with lenalidomide and dexamethasone.1
- The recommended starting dose of Ixazomib is 4 mg administered orally on Days 1, 8, and 15 of a 28-day treatment cycle. Ixazomib should be swallowed whole with water at least 1 hour before or at least 2 hours after food
- The recommended starting dose of lenalidomide is 25 mg administered daily on Days 1 to 21 of a 28-day treatment cycle
- The recommended starting dose of dexamethasone is 40 mg administered on Days 1, 8, 15, and 22 of a 28-day treatment cycle
Prior to initiating a new cycle of therapy:1
- Absolute neutrophil count should be ≥1000/mm3
- Platelet count should be ≥75000/mm3
- Non‑haematologic toxicities should, at the physician’s discretion, generally be recovered to patient’s baseline condition or ≤Grade 1
Antiviral prophylaxis should be considered in patients being treated with Ixazomib to decrease the risk of herpes zoster reactivation. Patients included in studies with Ixazomib who received antiviral prophylaxis had a lower incidence of herpes zoster infection compared to patients who did not receive prophylaxis.1
Thromboprophylaxis is recommended in patients being treated with Ixazomib in combination with lenalidomide and dexamethasone, and should be based on an assessment of the patient's underlying risks and clinical status.1
Ixazomib should be taken at approximately the same time on Days 1, 8, and 15 of each treatment cycle at least 1 hour before or at least 2 hours after food.1 The capsule should be swallowed whole with water. It should not be crushed, chewed, or opened.1
Treatment should be continued until disease progression or unacceptable toxicity.1 Treatment with Ixazomib in combination with lenalidomide and dexamethasone for longer than 24 cycles should be based on an individual benefit risk assessment, as the data on the tolerability and toxicity beyond 24 cycles are limited.1
Refer to the Ixazomib SmPC for full dosing guidelines.
1. Ixazomib. Summary of Product Characteristics.
Elderly1
- No dose adjustment of Ixazomib is required for patients over 65 years of age
Hepatic impairment1
- No dose adjustment of Ixazomib is required for patients with mild hepatic impairment (total bilirubin ≤ upper limit of normal (ULN) and aspartate aminotransferase (AST) > ULN or total bilirubin > 1-1.5 x ULN and any AST). The reduced dose of 3 mg is recommended in patients with moderate (total bilirubin > 1.5-3 x ULN) or severe (total bilirubin > 3 x ULN) hepatic impairment
Renal impairment1
- No dose adjustment of Ixazomib is required for patients with mild or moderate renal impairment (creatinine clearance ≥ 30 mL/min). The reduced dose of 3 mg is recommended in patients with severe renal impairment (creatinine clearance < 30 mL/min) or end-stage renal disease (ESRD) requiring dialysis. Ixazomib is not dialyzable and, therefore, can be administered without regard to the timing of dialysis
Paediatric population1
- The safety and efficacy of Ixazomib in children below 18 years of age have not been established. No data are available
Refer to the Ixazomib SmPC for the full dose modification guidelines. Refer to the Ixazomib SmPC for an overview of all Ixazomib side effects. For additional information regarding lenalidomide or dexamethasone, please refer to each respective SmPC.
1. Ixazomib. Summary of Product Characteristics.
Clinical studies have shown the most frequently reported adverse reactions (≥ 20%) in 418 patients treated with IRd to be diarrhoea (47%), thrombocytopenia (41%), neutropenia (37%), constipation (31%), upper respiratory tract infection (28%), peripheral neuropathy (28%), nausea (28%), back pain (25%), rash (25%), peripheral oedema (24%), vomiting (23%) and bronchitis (20%). Serious adverse reactions reported in ≥ 2% of patients included diarrhoea (3%), thrombocytopenia (2%) and bronchitis (2%).1
Please refer to the SmPC for further information on the Ixazomib tolerability profile.
1. Ixazomib. Summary of Product Characteristics.

Request a meeting
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MM, multiple myeloma; RCT, randomised control trial; RWE, real world evidence.
1. Derman BA, et al. Blood Rev. 2022;53:100913. 2. Shah JJ, et al. Clin Lymphoma Myeloma Leuk. 2017;17:575–583. 3. Richardson PG, et al. Blood Cancer J. 2018;8:109. 4. Visvanathan K, et al. J Clin Oncol. 2017;35:1845–1854. 5. Terpos E, et al. Blood Cancer J. 2021;11:40. 6. Chodankar D. Perspect Clin Res. 2021;12:171–174. 7. Blonde L, et al. Adv Ther. 2018;35:1763–1774. 8. Camm AJ and Fox KAA. Open Heart. 2018;5:e000788. 9. Roberts MH and Ferguson GT. Pharmacoecon Open. 2021;5:3–11. 10. Katkade VB, et al. J Multidiscip Health. 2018;11:295–304.
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