Rethinking the reimbursement of innovative medicines in oncology: Looking beyond overall survival

BJMO - volume 17, issue 6, october 2023

M. Piccart MD, PhD, J. Vansteenkiste MD, PhD, H. Prenen MD, PhD, F. Duhoux MD, PhD, A. Janssens MD, PhD, M. Delforge MD, PhD, A. Awada MD, PhD, P. Neven MD, PhD, A. Sibille MD, B. Neyns MD, PhD


The oncological treatment landscape is evolving at a very rapid pace with a continuous stream of novel treatment options. To fully leverage these therapeutic advances in clinical practice it is important to facilitate a rapid access to innovative anticancer drugs for patients. Specifically for Belgium, the delay from EMA approval to reimbursement for anticancer drugs is very long, with an average of almost 600 days, and a substantial proportion of innovative drugs never make it to the patient. The stringent focus of the Belgian Commission for Reimbursement of Medicines (CRM) on overall survival (OS) data in reimbursement decisions is believed to be an important contributor to this situation. However, the ever-increasing pace at which new anticancer therapies are being developed in combination with an earlier detection of cancer will make it increasingly difficult to present mature OS data at the time of regulatory approval in the years to come. As such, there is an urgent need for a debate with the regulators to consider more readily available endpoints in their reimbursement assessments. In fact, when a strong treatment effect is seen on an intermediate endpoint such as disease-free or progression-free survival, a benefit in OS is quite likely. As such, our reimbursement system needs to be adapted to better align with the scientific progress in oncology. In this, a temporary reimbursement decision based on intermediate endpoints could give Belgian patients earlier access to promising lifesaving medicines. In this, we as oncologists, including specialists in haematology, respiratory oncology, and gastrointestinal cancer, strongly encourage the CRM to use the grading provided by the ESMO magnitude of clinical benefit scale to evaluate the clinical added value of future cancer treatments. This will not only facilitate a faster patient access to innovative therapies, but will also help policy-makers in advancing ‘accountability for reasonableness’ in their resource allocation deliberations.

(BELG J MED ONCOL 2023;17(6):211–5)

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Cancer, chronic kidney disease and thrombosis

BJMO - volume 17, issue 2, march 2023

C. Isnard-Bagnis MD, PhD, J. Nortier MD, PhD, C. Vulsteke MD, PhD, C. Hermans MD, PhD, S. Treille De Grandsaigne MD, P. Clement MD, PhD, A. Awada MD, PhD


Chronic kidney disease (CKD) and cancer are intertwined in many ways. In fact, cancer can cause CKD either directly or indirectly through the treatment adverse effects, while CKD may conversely be a risk factor for cancer. According to the Belgian Renal Insufficiency and Anticancer Medications (BIRMA) study, 64% of patients with cancer had a glomerular filtration rate (GFR) <90 ml/min/1.73m2 and 16% of them presented with a mildly to severely decreased GFR (i.e.; <30 ml/min/1.73m2 or 30–60 ml/min/1.73m2). As many anticancer drugs are predominantly excreted in the urine, tailoring the drug dose to the renal function of the individual patient is a crucial consideration. Furthermore, patients with cancer and CKD are also at an increased risk of thrombosis. Therefore, safe and effective drugs for the treatment and prevention of thrombotic events are necessary.

(BELG J MED ONCOL 2023;17(2):46–51)

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Belgian clinical practice guidelines for the treatment of patients with HER2-positive advanced breast cancer

BJMO - volume 16, issue 6, october 2022

G. Nader-Marta MD, F.P. Duhoux MD, PhD, D. Taylor MD, T. Van den Mooter MD, H. Denys MD, PhD, J-L. Canon MD, J. Mebis MD, PhD, A. Awada MD, PhD, H. Wildiers MD, PhD, K. Punie MD, E. de Azambuja MD, PhD


HER2-targeted agents are the central component of HER2-positive metastatic breast cancer (MBC) treatment. The combination of trastuzumab, pertuzumab and a taxane is the preferred first-line regimen in most settings. For patients with disease relapse after adjuvant therapy, treatment decisions in the first-line are influenced by the treatment-free interval and the regimens used in the (neo)adjuvant setting. T-DXd has been recently established as the preferred second-line therapy. T-DM1, or the combination of tucatinib, trastuzumab and capecitabine, are reasonable third-line options, although efficacy and safety data of these regimens after prior exposure to T-DXd are lacking. In fourth and later lines, trastuzumab duocarmazine, neratinib plus capecitabine, margetuximab plus chemotherapy, lapatinib-based combinations or the continuation of trastuzumab with different chemotherapy partners are valid alternatives.

(BELG J MED ONCOL 2022;16(6): 287–92)

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Current treatment patterns of advanced hormone receptor-positive, HER2-negative breast cancer in Belgium

BJMO - volume 16, issue 4, june 2022

G. Jerusalem MD, PhD, A. Awada MD, PhD, K. Detournay DVM , K. Punie MD


Several treatment guidelines exist for hormone receptor-positive/HER2-negative advanced breast cancer (HR+/HER2- aBC), but various factors influence their local implementation. We performed a 3-round Delphi methodbased study in search of a consensus regarding HR+/HER2- aBC management in Belgian practice. Panel questionnaires included questions related to treatment patterns, drug-drug interactions (DDIs) and side-effect management (SEM). A consensus threshold of 75% was applied. The results were evaluated for concordance with the ABC5 guidelines. Treatment patterns in HR+/HER2- aBC reached moderate to high consensus among Belgian oncologists and showed high concordance with ABC5 guidelines. A CDK4/6 inhibitor is the preferred first-line treatment, combined with an aromatase inhibitor or with fulvestrant in the endocrine-sensitive and -resistant setting, respectively. Alpelisib-fulvestrant is the preferred second-line treatment in presence of a PIK3CA-activating mutation. Some practices regarding DDI and SEM needed further discussion before reaching consensus highlighting the need for additional training and incorporation of these topics in guidelines.

(BELG J MED ONCOL 2022;16(4):176–86)

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Is there a place for thromboprophylaxis in cancer patients?

BJMO - volume 16, issue 2, march 2022

A. Awada MD, PhD, K. Jochmans MD, C. Vulsteke MD, PhD, T. Vanassche MD, J. Mebis MD, PhD, V. Mathieux MD, J-F. Baurain MD, PhD, P. Hainaut MD, P. Verhamme MD


Venous thromboembolism (VTE) is common in cancer patients. It is associated with poor outcomes and increased mortality. In fact, VTE is known as the second most common cause of mortality in cancer patients. Although the benefit of thromboprophylaxis is clear for acutely ill hospitalised cancer patients, routine prophylaxis is not recommended for all ambulatory cancer patients. The reason is the risk to treat a high proportion of patients who do not need treatment and an increased risk of major bleeding. Here we highlight the importance of adequate risk assessment models to select patients at an increased VTE risk and present pivotal trial results that form the basis for the latest international treatment guidelines related to thromboprophylaxis in cancer patients.

(BELG J MED ONCOL 2022;16(2):53–9)

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Future perspectives and opportunities to improve the treatment of brain metastases in solid tumours: Brainstorm program – an initiative from the Oncodistinct network

BJMO - volume 15, issue 7, november 2021

N. Kotecki MD, MA. Franzoi MD, A. Awada MD, PhD


Patients with central nervous system (CNS) metastases have a poor prognosis, which is generally worse than in those with disease only outside the CNS. Treatment options for CNS metastases are still limited and suboptimal. New systemic therapies such as targeted therapies and immunotherapy have emerged for different cancers and differences in survival of patients with CNS metastases by tumour subtype have been observed. A better knowledge on the evolving epidemiology and biology of CNS metastases are key elements in the development of new treatment strategies whereby the identification of promising therapeutic targets for new compounds may play an important role in improving patient outcome. This article will provide a general overview of the recent improvement in systemic therapies for CNS metastases, highlighting perspectives to improve the management of CNS metastases and introduce the BrainStorm program- an innovative research program from the Oncodistinct network aiming to overcome the challenges of CNS metastases.

(BELG J MED ONCOL 2021;15(7):357-61)

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Clinical management of first-line advanced triple-negative breast cancer patients

BJMO - volume 14, issue 7, november 2020

M. Rediti MD, K. Punie MD, E. de Azambuja MD, PhD, E. Naert MD, D. Taylor MD, FP. Duhoux MD, PhD, H. Denys MD, PhD, A. Awada MD, PhD, H. Wildiers MD, PhD, M. Ignatiadis MD, PhD


Chemotherapy has represented the main treatment option for patients with advanced triple-negative breast cancer for a long time. However, due to our better understanding of tumour biology, recent clinical trials led to a change in the treatment paradigm of this disease, identifying clinically relevant subgroups with different therapeutic options. Both clinical and biological factors have become relevant and need to be considered in the treatment decision algorithm of this heterogeneous disease.

(BELG J MED ONCOL 2020;14(7):333-38)

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