We read with interest the article by de Wilde et al that explores partnerships between academia and industry in conducting trials for pediatric cancer drug approval.[1] Cancers in the pediatric group have high cure rates and offer the possibility of significant gains in terms of years of life lived in the event of sustained remission. The pace of cancer drug development has traditionally been slower in children, with reported median lag periods of over 5 years for approvals in comparison to adult use.[2] Legislation mandating pediatric studies for drugs being tested in adults has helped accelerate this process over the last decade.[1] However, the global reach of such approvals is limited. Childhood cancer survival rates are reported to be as high as 80%-in high-income countries (HICs), while low- and middle-income countries (LMICs) lag behind.[1] [3] Treatment abandonment and social barriers to obtaining treatment are significant issues in LMICs, which may be further exacerbated by high costs of care.[4] Thus, drug approval is only an initial step in the process of translating drug development into survival benefits in childhood cancer. The role of academia–pharmaceutical collaborations in enhancing drug access remains unexplored.

Traditionally, the degree of academic involvement in drug trials, as opposed to pharmaceutical company involvement, is higher in pediatric cancers than in adult cancers. The article by de Wilde et al has emphasized the vital role of partnerships between academia and industry in conducting trials for pediatric cancer drug approval.[1] Clinicians can access large hospital databases that provide real-world patient data about drug use patterns, efficacy, and toxicity. They also have more opportunities to understand the patient's perspective holistically regarding reasons for satisfaction with treatment, nonadherence, treatment refusal, and abandonment. Thus, they may be better placed to identify routine clinical problems and design patient-friendly and cost-effective solutions. On the other hand, pharmaceutical companies could provide funding and rigor to trial conduct and may be better equipped to steer trial protocols in a direction that facilitates approval. In India and other LMICs, drug trials are predominantly pharmaceutical driven.[5] The lack of academic involvement in clinical trials may be due to financial, technical, and regulatory constraints. It has been seen that randomized trials of cancer drugs conducted in LMICs are more likely to identify larger effect sizes and identify effective therapies in comparison to those in HICs.[6] Thus, collaborative ventures in LMICs are likely to reap rewards for both academicians and clinicians. [Fig. 1] shows how such collaborations may enable better drug access and benefit the partners involved.

| Fig 1 : Academia–drug manufacturer partnerships for enhancing cancer drug access: the contributions of and benefits to each partner.

Tannock et al found in their review that drug doses used in adult clinical trials of new drugs are often higher than the effective doses identified in earlier phases.[7] Many investigator-initiated trials have explored the efficacy of lower doses of approved drugs ([Table 1]).[8] [9] [10] [11] [12] While some of these trials may have been conducted with the aim of reducing drug-related toxicities with the support of government funding sources, they have the collateral benefit of reducing treatment-related costs and health care resource utilization.[10] In general, cost-reduction strategies are perceived as not being in line with the commercial interests of pharmaceutical companies. However, a few pharmaceutical-funded trials have also attempted to seek out pharmacoeconomic strategies. A pharmaceutical-funded phase III study of low-dose nivolumab in India demonstrated significant improvement in advanced head and neck carcinoma outcomes.[8] This critical finding has opened up the possibility of expanding the use of immunotherapy for cancer patients in resource-challenged settings

References

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Publication History

Article published online:
10 February 2025

© 2025. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution License, permitting unrestricted use, distribution, and reproduction so long as the original work is properly cited. (https://creativecommons.org/licenses/by/4.0/)

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| Fig 1 : Academia–drug manufacturer partnerships for enhancing cancer drug access: the contributions of and benefits to each partner.

References