In recent years, the oncology sector has undergone a fundamental shift away from chemotherapy-dominant treatment towards the use of regimens incorporating targeted therapies. This trend has seen several new drugs become embedded into treatment paradigms across haematological cancers and solid tumours.
This shift is, in part, driven by the cytotoxicity associated with chemotherapy, which can have a significant impact on a patient’s quality of life (QoL), as well as their ability to tolerate further treatment if they were to relapse.
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In terms of sales and uptake of targeted therapies, immune checkpoint inhibitors (ICIs) are arguably one of the greatest success stories. This is evidenced by the broad use of MSD’s Keytruda (pembrolizumab) across more than 40 cancer indications, as well as its 2025 sales of $31.7bn – making it one of the best-selling drugs currently on the market. Antibody-drug conjugates (ADCs), monoclonal antibodies (mAbs), kinase inhibitors, chimeric antigen receptor T-cell (CAR-T) therapies and radiopharmaceuticals are also making their mark on the space.
More targeted approaches are also being accepted by regulators, as the US Food and Drug Administration (FDA) awarded 63 review designations to ADCs in 2024 – nearly double the previous year’s peak of 35.
As targeted therapies continue to alter the oncology treatment paradigm, experts tell Pharmaceutical Technology that several factors will influence the market uptake of these treatment options, with chemotherapy set to remain a workhorse in oncology.
Progression to targeted therapies varies on indication
While the use cases of targeted therapies in oncology are generally growing across the board, this effect can particularly be seen in non-small cell lung cancer (NSCLC) and melanoma, says Tatiana Kolesnikova, director of oncology research and analysis at GlobalData.
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By GlobalData“NSCLC therapies targeting driver mutations like EGFR, KRAS G12C or ALK achieve overall survival (OS) well beyond platinum-based chemotherapy, and in no-actionable-mutation disease, checkpoint inhibitors have become preferred first-line options – particularly in populations with high PD-L1 expression,” Kolesnivoka notes.
Paul Peter, CEO of immune oncology-focused biotech, Candel Therapeutics, echoes Kolesnikova’s sentiments, noting that checkpoint inhibitors have been shown to significantly improve patient outcomes in frontline stage 3 or 4 NSCLC. However, in the relapsed/refractory setting, he believes that different targeted treatments like viral immunotherapies will offer further outcome benefits. Peter adds that immunotherapies may also play a larger role in earlier disease stages moving forward, as these patients tend to be “less immunologically exhausted” due to prior treatment experience – meaning they could gain more benefit from this drug class.
Despite good progress across certain indications, this trend is not unanimous across oncology, as the industry has traditionally struggled to successfully treat cold tumours, which are characterised by an immunosuppressive microenvironment. “Indications like pancreatic cancer, glioblastoma and soft tissue sarcomas are still heavily dependent on chemotherapy,” Kolesnikova comments.
“Future progress to develop targeted therapies in these areas will depend on personalised treatments based around multi-omic profiling and multi-modal combinations, as well as therapeutic sequencing to combat emerging resistance,” she adds.
Meanwhile, Christina Coughlin, oncologist and CEO of biotech Avacta Therapeutics, adds that rare cancers tend to see a lack of progress in terms of targeted therapies due to the reduced economic return a company may make as a result of the small nature of the treatable population. She specifically names salivary gland and rarer subsets of lung cancer as examples.
Combination is king
As targeted therapies become increasingly important in oncology, Coughlin notes that the industry should continue to focus on developing combination therapeutics – whether that be with chemotherapy or other targeted agents. “Administering a single drug to a tumour often breeds resistance, which you always want to avoid when treating a patient,” she says. According to Coughlin, this is particularly true in acute myeloid leukaemia (AML), which is why physicians employ a highly sequenced treatment approach to prevent acquired resistance.
Kolesnikova adds that multimodal combinations will increasingly replace monotherapies to overcome resistance – naming ICI-ADC and bi/multi-specific T-cell engager-cytokine pairs as examples.
Meanwhile, Peter predicts that physicians will likely use more combination therapies where it makes sense, but believes the “real progress” lies in identifying where targeted approaches can deliver durable benefit, with less cumulative toxicity, alongside chemo, which he says will play “a more supportive role”.
Payers influence shift to targeted therapy
Though payers could theoretically prioritise chemotherapy over the use of targeted treatments due to their lower price, Jason Shafrin, senior managing director of FTI’s Center for Healthcare Economics and Policy, notes that reducing cost is not a payer’s whole goal. “It’s also about using resources in the best way to improve patient health outcomes; payer decisions will depend on a therapy’s benefit-to-cost ratio,” he says.
In the US, Shafrin believes that private payers are generally more likely to allocate funds to high-cost oncology therapies – including medicines given the greenlight through accelerated approval pathways – over drugs in other areas like cardiovascular disease. This is mostly due to the acute severity of many cancer indications, as well as their “immediate and life-threatening consequences”.
However, Shafrin does acknowledge a discrepancy between the risk appetite of patients and payers. “Many patients will take the risk on a treatment with a potential upside, but payers will often want to know a drug works for sure before reimbursing,” he says.
Shafrin also notes the importance of considering a therapy’s cost on both the patient and system-wide levels. “A lot of targeted cancer therapies are high cost, but they are often only suitable for a smaller patient population who express a specific biomarker,” he says. “As you only use these therapies in a smaller group of patients, the total cost to the system is not necessarily as much as, for example, a cardiovascular drug you’d use across a large population,” Shafrin adds.
According to Shafrin, the level of conservatism demonstrated in payer decisions can also depend on geography. “In Germany, payers are quite strict with cancer therapy reimbursement, as these drugs will often have to show OS benefits to gain coverage.” He contrasts this with the approach of US payers, who he says are increasingly open to accepting established surrogate endpoints as proof of efficacy.
As companies increasingly shift their focus to create oncology drugs that are more convenient for patients, Shafrin does not believe that the administration route will significantly impact payer decisions moving forward.
Chemotherapy still set to play a key role
While the industry has set the scene for the increased use of targeted therapies, Peter notes that the goal should not be to abolish chemotherapy. “There will still be an important place for it [chemotherapy]. The paradigm shifts we’re seeing are more centred around selective replacement,” he says.
Coughlin mirrors this take, adding: “Chemotherapy combines well, meaning it’s a good tool to weaken the tumour before coming in for the kill with targeted therapies. What companies and clinical oncologists are looking for now is better combinations and smarter ways to use chemotherapy,” she explains. According to Coughlin, this includes both ADCs and peptide-drug conjugates (PDCs), which deliver chemotherapy straight to the tumour.
Meanwhile, Kolesnikova believes that chemotherapy will likely continue to lose its position as a backbone for cancer treatment over the next 5-10 years, while being “completely phased out in some indications”, including chronic lymphocytic leukaemia (CLL), selected forms of NSCLC, and melanoma. However, she notes that chemotherapy will remain a minor component in a subset of cancer indications. “The pace of this change will depend on how much the field invests, the regulatory environment and the ability to address the fundamental challenges of resistance, tumour heterogeneity and equitable treatment access,” Kolesnikova concludes.
