Global Myeloma Trial Participation and Drug Access in the Era of Novel Therapies

Abstract

PURPOSE The globalization of clinical trials has accelerated recent advances in multiple myeloma (MM). However, it is unclear whether trial enrollment locations are reflective of the global burden of MM and whether access to novel therapies is timely and equitable for countries that participate in those trials. METHODS To assess this, we characterized where MM trials that led to US Food and Drug Administration (FDA) approvals were conducted and determined how often and quickly these drug regimens received approval in their participating trial countries on the basis of country income level and geographic region. RESULTS A systematic review was conducted to identify all MM clinical trials that met their primary endpoint, enrolled patients outside the United States, and resulted in FDA approval from 2005 to 2019. A total of 18 pivotal MM clinical trials were identified. High-income countries enrolled patients in 100% (18/18) of the trials identified, whereas upper-middle and lower-middle-income countries were represented in 61% (11/18) and 28% (5/18) of trials, respectively. No patients from low-income countries were enrolled. One trial enrolled patients in sub-Saharan Africa, and no trials enrolled patients in South Asia/Caribbean. For drugs/regimens that were approved in their participating countries, the median time from FDA approval to approval was 10.9 months. There were no drugs approved in lower-middle-income trial countries. MM trials leading to FDA approval are generally run in high-income, European, and Central Asian countries. CONCLUSION There are substantial disparities in where novel therapies are evaluated and where they are ultimately approved for use on the basis of income level and geography.

Global, regional, and national burden and quality of care of multiple myeloma, 1990–2019

“…In fact, inequality has been exacerbated with the emergence of new, expensive therapy [ 49 ], resulting in concerns on the limited availability and applicability of treatment. A recent review synthesised 18 pivotal MM clinical trials from 2005 to 2019 and reported a lag for treatment approval in LMICs, which were likewise under-represented in clinical trials [ 50 ]. Europea and Central Asia had the highest trial approval rates for MM (with a time lag of 8.3 months), while Latin America and sub-Saharan Africa did not even have a regimen approval for the trials in which they participated [ 50 ].…”

“…A recent review synthesised 18 pivotal MM clinical trials from 2005 to 2019 and reported a lag for treatment approval in LMICs, which were likewise under-represented in clinical trials [50]. Europea and Central Asia had the highest trial approval rates for MM (with a time lag of 8.3 months), while Latin America and sub-Saharan Africa did not even have a regimen approval for the trials in which they participated [50]. For example, lenalidomide and bortezomib, listed as the standard care of MM are still not approved in some LMICs located in Africa and Mid-eastern The data presented in this study can be found in the GBD repository (https://vizhub.healthdata.org/ gbd-results/).…”

Optimizing autologous stem cell transplantation for multiple myeloma: early step-down ward vs. HEPA-filtered room settings – a practical approach for resource-constrained settings

“…Multiple myeloma (MM) remains an incurable malignancy, accentuating the necessity for therapeutic strategies that augment treatment response and prolong survival [1,2]. Despite the advent of novel pharmacological classes that have improved outcomes, the availability of these treatments is often restricted, especially in resource-limited contexts [3]. Autologous stem cell transplantation (ASCT) continues to be a pivotal treatment for eligible patients.…”