New Horizons from Clinical Research to Cancer Treatments

The pharmaceutical industry is significantly increasing its investments and R&D efforts in the field of oncology. By 2030, oncology, immunology, and metabolic diseases are expected to be among the most dominant areas in the market. (1) Given the high risks involved, there is growing demand for technologies and methodologies such as artificial intelligence (AI), adaptive trials, and real-world evidence (RWE), which promise to significantly reduce drug development timelines and costs. While these dynamics drive competition, they also deepen challenges related to pricing and access.

In 2023, AI- and machine learning-related business deals reportedly exceeded $12 billion in total. Molecules discovered by AI have shown Phase I success rates of 80–90%, creating a significant difference compared to the 40–65% success rate typically seen with molecules discovered by scientists. (2)

Recent Developments in Clinical Research

In recent years, significant changes have been observed in the design and execution of clinical research. Regulatory bodies like EMA and FDA emphasize the critical importance of ensuring diversity in trial populations. Several global cancer research and development initiatives led by the FDA’s Oncology Center of Excellence are significantly shaping the field. Key initiatives include Project FrontRunner, which promotes testing new drugs in early-stage patients; Project Optimus, which focuses on dose optimization; Project Orbis, which facilitates simultaneous international review; Project Patient Voice, which integrates patient experiences into the process; and Project Confirm, which aims to increase transparency in accelerated approval pathways.

Alongside regulatory initiatives, newly developed methods in clinical research are providing numerous benefits in terms of rapid access to innovative therapies. Adaptive clinical trials allow data-driven decision-making during the study, increasing efficiency in drug development. This flexibility enables changes in design details such as sample size, treatment regimens, and patient allocation. Decentralized clinical trials (DCTs) have improved accessibility to studies and reduced the participation burden. DCTs enable continuous, real-time data collection through technologies like wearable sensors and mobile health applications.

The high success rates of 80–90% observed in Phase I trials for molecules discovered by artificial intelligence represent a significant difference compared to the average 40–65% success rates for molecules discovered by scientists.

Türkiye ranks 26th globally in terms of the annual number of clinical trials conducted. However, when ranked by population, GDP, and pharmaceutical market size, its position drops. This highlights Türkiye’s potential to increase its share in global clinical research investments. As of June 2019, the annual value of clinical trials in Türkiye was estimated at $327.7 million. This figure translated into $46.4 million in medical cost savings for the Social Security Institution, $23.4 million in additional income for the healthcare system, $44.4 million in employment-related value, and approximately $18.6 million in other direct economic contributions. (3)

In 2023, R&D-based pharmaceutical companies invested $425 million in clinical trials in Türkiye. (4) To enhance Türkiye’s attractiveness in clinical research, there are opportunities for improvement in eight areas: easier patient access to trials, increased patient awareness, process simplification, development of new investigators, physician incentives, dedicated clinical research staff, predictable clinical trial costs, and sponsor incentives. By leveraging these opportunities, Türkiye could become one of the top 10 countries globally in clinical research in the short term. In an ambitious growth scenario, the number of clinical trials could be tripled, raising the annual total value to $1.13 billion and enabling the participation of approximately 70,000 patients each year. (3)

Recent Advances in Cancer Therapies

Cancer treatment has undergone a major transformation over the past decade. While traditional approaches like chemotherapy and radiotherapy once dominated, precision oncology and targeted therapies have now come to the forefront. The number of new cancer drugs approved annually by the EMA has increased significantly since 1995—for example, 14 new drugs were approved in 2023. (5)

Among the newly approved and developed therapies are tyrosine kinase inhibitors, immune checkpoint inhibitors, antibody-drug conjugates, bispecific antibodies, CAR T-cell therapies, radioligand therapies, gene and RNAi therapies, oncolytic virus therapies, personalized neoantigen vaccines, KRAS inhibitors, and p53-targeting agents. Combination therapies have become a key strategy in cancer treatment, offering potential to overcome drug resistance and create synergistic effects by targeting multiple pathways. For example, the combination of dabrafenib and trametinib has demonstrated superior efficacy compared to monotherapy in advanced melanoma. Likewise, the nivolumab and ipilimumab combination has significantly improved overall survival (OS) and treatment-free survival (TFS) in patients with advanced melanoma. (6)

Cancer treatment has undergone a significant transformation over the past decade. While traditional approaches such as chemotherapy and radiotherapy once dominated, precision oncology and targeted therapies have now come to the forefront.

Significant progress has also been made in diagnosis and early detection. Molecular diagnostic techniques such as next-generation sequencing (NGS) play a vital role in identifying cancer-specific genetic alterations. Non-invasive methods like liquid biopsy—which involves detecting biomarkers such as circulating tumor DNA or proteins—offer great potential for cancer screening, diagnosis, treatment monitoring, and detection of minimal residual disease.

The ASCO Congress is a key platform for presenting the latest scientific developments and clinical practices in oncology. The 2024 and 2025 meetings emphasized the importance of personalized and targeted approaches in cancer treatment, the expansion of immunotherapy in solid tumors, and the significance of early detection. Emerging research areas such as improving post-treatment quality of life and the effects of unexpected factors like circadian rhythm on treatment effectiveness also garnered attention.

These developments offer great hope for treating cancer more effectively and improving patients’ quality of life. The biopharmaceutical industry continues to make breakthroughs in the fight against cancer through innovation and collaboration. Countries like Türkiye can gain significant economic and healthcare benefits by keeping pace with these global changes and maximizing their own potential.

References:

  1. Marante T., The Future of Pharma in 2030. (Accessed 19.06.2025) https://pharmaoffer.com/blog/the-future-of-pharma-therapyareas-ai-supply-chains/
  2. Alcacer T., Evolution of pharmaceutical R&D: three key trends to follow. 16.05.2025 (Accessed 19.06.2025) https://www.alcimed.com/en/insights/pharma-rd-trends/
  3. Benefits of a Clinical Research Strategy for Türkiye, IQVIA, 2020.
  4. Türkiye Pharmaceutical Industry Report, IQVIA, 2024.
  5. Manzano A, Svedman C, Hofmarcher T, Wilking N. Comparator Report on Cancer in Europe 2025 – Disease Burden, Costs and Access to Medicines and Molecular Diagnostics. IHE REPORT 2025:2. IHE: Lund, Sweden.
  6. Medical Rationale Supporting Patient Access to Novel Oncology Combination Therapies, EFPIA, 03.2024.