Inequities in cancer drug development in terms of unmet medical need

Barrenho, Eliana; Halmai, Réka; Miraldo, Marisa; Tzintzun, Iván; Ali, Setti Rais; Toulemon, Léa; Dupont, Jean-Claude K.; Rochaix, Lise

doi:10.1016/j.socscimed.2022.114953

Cited by 4 publications

(2 citation statements)

References 36 publications

(27 reference statements)

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“…While this approach narrowly focuses on patient eligibility, certain orphan cancer drugs may also be used in non-rare or multiple rare conditions, shifting the market dynamics to resemble conventional medical therapies [38][39][40][41][42]. Yet, numerous rare cancers, especially in children, continue to face significant unmet health needs [43].…”

Section: Rare Cancer Policiesmentioning

confidence: 99%

Availability and Access to Orphan Drugs for Rare Cancers in Bulgaria: Analysis of Delays and Public Expenditures

Kostadinov,

Popova-Sotirova,

Marinova

et al. 2024

Cancers

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Rare cancers are defined by an annual incidence of fewer than 6 per 100,000. Bearing similarities to rare diseases, they are associated with substantial health inequalities due to diagnostic complexity and delayed access to innovative therapies. This situation is further aggravated in Southeastern European countries like Bulgaria, where limited public resources and expertise underscore the need for additional policy and translational research on rare cancers. This study aimed to explore the availability and access to orphan drugs for rare cancers in Bulgaria for the period of 2020–2023. We cross-compared data from both the European Union and national public sources to evaluate the number of available and accessible orphan drugs for rare cancers, the delay from market authorization to reimbursement, the dynamics of public expenditures, and regional disparities in access across the country. We juxtaposed the main characteristics of oncological and non-oncological orphan drugs as well. Only 15 out of 50 oncological orphan drugs that were authorized by the European Medicine Agency were accessible for rare cancer patients in Bulgaria. The median delay between market authorization and inclusion in the Bulgarian Positive Drug List was 760 days. The total expenditures for all orphan drugs for rare cancers amounted to EUR 74,353,493 from 2020 to 2023. The budgetary impact of this group rose from 0.24% to 3.77% of total public medicinal product expenditures for the study period. Rare cancer patients represent a vulnerable population that often faces limited to no access to treatment. We call for targeted European and national policies to address this major inequality.

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Section: Rare Cancer Policiesmentioning

confidence: 99%

Availability and Access to Orphan Drugs for Rare Cancers in Bulgaria: Analysis of Delays and Public Expenditures

Kostadinov,

Popova-Sotirova,

Marinova

et al. 2024

Cancers

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“…Inequities related to clinical trial participation are widespread and well documented. 19 – 21 Access via an Expanded Access Program (EAP) or SAP exacerbates these concerns, 22 and often, only a motivated, informed, and well-connected subset of the patient population will achieve access through these programs. When access requires private pay or travel out of province or country, equity concerns are exacerbated even further.…”

Section: Drug Development Approval Reimbursement and Accessmentioning

confidence: 99%

Ethical challenges and opportunities in the development and approval of novel therapeutics for rare diseases

Djordjevic

McFadyen²,

Anderson

2023

The Journal of Medicine Access

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The development of novel therapeutics for rare “orphan” diseases has brought a growing tension between the desire to accelerate access to these breakthrough therapies and the need to generate quality evidence regarding their safety and efficacy. Accelerating the pace of drug development and approval may facilitate the rapid delivery of benefits to patients and cost savings for research and development, which theoretically improves affordability of drugs for the health system. However, several ethical challenges arise with expedited approval, compassionate release of drugs, and subsequent study of drugs in “real-world” settings. In this article, we explore the changing landscape of drug approval and the ethical challenges expedited approval creates for patients, caregivers, clinicians, and institutions, and propose tangible strategies to maximize the benefits of “real-world” data acquisition while mitigating risks to patients, clinicians, and institutions.

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The role of patient organisations in research and development: Evidence from rare diseases

Gentilini,

Miraldo

2023

Social Science & Medicine

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Inequities in cancer drug development in terms of unmet medical need

Cited by 4 publications

References 36 publications

Availability and Access to Orphan Drugs for Rare Cancers in Bulgaria: Analysis of Delays and Public Expenditures

Availability and Access to Orphan Drugs for Rare Cancers in Bulgaria: Analysis of Delays and Public Expenditures

Ethical challenges and opportunities in the development and approval of novel therapeutics for rare diseases

The role of patient organisations in research and development: Evidence from rare diseases

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