197
Volume 5, Issue 10: Special Issue
(EJAR)
ISSN: 2181-2020
MPHAPP
THE 6TH INTERNATIONAL SCIENTIFIC AND PRACTICAL
CONFERENCE
“
MODERN PHARMACEUTICS: ACTUAL
PROBLEMS AND PROSPECTS
”
TASHKENT, OCTOBER 17, 2025
in-academy.uz
HIGH-COST ANTICANCER DRUGS: CLINICAL BENEFIT OR FINANCIAL
BURDEN FOR THE HEALTHCARE SYSTEM?
Rikhsieva O.G.
Rajabova N.Kh.
Tashkent Pharmaceutical Institute, Tashkent city, Republic of Uzbekistan
e-mail: odina.rixsiyeva2001@gmail.com
https://doi.org/10.5281/zenodo.17332721
Relevance:
the growing number of innovative but expensive anticancer drugs imposes a
significant burden on healthcare systems. Many of these medicines are approved under accelerated
procedures with a limited evidence base and often fail to demonstrate a clinically meaningful
additional effect, raising doubts about the rationality of their use.
Objective of the study: to conduct a market analysis of anticancer drugs approved by the
European Medicines Agency between 1995 and 2020, focusing on the assessment of their proven
clinical effectiveness, comparison with market costs, and determination of payback periods.
Materials and methods:
a review of anticancer drugs approved during the specified period
was carried out, with analysis of the level of evidence for their clinical benefit and the timelines for
recouping research and development costs. Additionally, examples of rational use of high-cost drugs
were considered (in particular, the SONIA study on HR+/HER2– breast cancer).
Results:
the analysis showed that a significant proportion of new anticancer drugs approved by
the European Medicines Agency between 1995 and 2020 did not demonstrate a substantial additional
clinical benefit compared to existing standards of therapy. This was especially characteristic of
molecules registered under accelerated or conditional approval procedures: their effectiveness was
often confirmed on the basis of surrogate endpoints (e.g., tumor shrinkage or disease progression)
rather than survival data or improvement in quality of life. From an economic perspective, more than
90% of such drugs recoup development costs within 8 years, with a median payback period of only
3–4 years, reflecting the high profitability of this market segment. However, this does not always
correspond to proportional patient benefit. An example is the SONIA study (Sequential versus
Optimal strategy of treatment with New generation aromatase Inhibitors in Advanced breast cancer),
which demonstrated that optimizing the timing of expensive drug use (e.g., deferring their
administration to later lines of therapy) can result in significant cost savings without worsening
clinical outcomes.
Conclusions:
there is a gap between regulatory approval procedures and the actual clinical
benefit for patients. Policies are needed to encourage the development of genuinely meaningful
medicines and the more rational use of high-cost drugs. Otherwise, healthcare systems risk being
overwhelmed by the financial burden of anticancer drugs without proven additional benefit.
