Unveiling the disparity in access to cancer therapies: A cross-sectional study on FDA-approved drugs in India

INTRODUCTION

India stands out as one of the most significant contributors to the global cancer burden, ranking third in terms of new incidences and second in mortalities, which significantly burdens its healthcare system.^[1] Approximately three out of every five individuals in India succumb to death following a cancer diagnosis, as per the GLOBOCAN 2022 incidence (1,413,316) to mortality (916,827) ratio.^[1] As the population of India transitions from the reproductive age group to the middle age and geriatric age group, India is anticipated to witness the highest cancer burden of all time in the near future, potentially exacerbated by the looming epidemic. Over the past five years, the U.S. Food and Drug Administration (FDA) has approved approximately 289 cancer therapies for various indications, including new drug approvals and dose modifications for both solid and haematological malignancies, demonstrating significant progress in oncology. However, a striking number of these advancements have yet to reach Indian patients. This disparity highlights an urgent need to assess the clinical value of these drugs and address the barriers that hinder their accessibility in India. Access to these therapies for cancer in India can be limited by several factors, including lack of insurance coverage, financial toxicity, and low accessibility and availability. A significant hindrance remains the lack of availability of the drug in the Indian market, explained by several possible reasons from the drug manufacturing companies,which thus makes a huge impact on the clinical outcomes of our patients. The approval of the new drug for medical use is closely regulated by a central authority. To regulate every aspect of the drugs from research to approval, manufacture, and marketing, there are specific regulatory rules and laws implemented by the government and regulatory authorities. Every country has its own authority to regulate the manufacturing and marketing of drugs. The FDA and the European Medicines Agency (EMA) are responsible for the approval and licensing of medicines in the USA and the European Union, respectively. The Central Drugs Standard Control Organization (CDSCO) is the corresponding agency in India.^[2] The CDSCO is a division in the Ministry of Health and Family Welfare and is headed by the Drugs Controller General of India (DCGI). It generates/sets the standards for efficacy, quality, and ensuring the safety of drugs, cosmetics, devices, and diagnostics in India.^[3]

Access to effective therapies is critical in improving outcomes, yet the availability of FDA-approved chemotherapy and targeted drugs with high ESMO-MCBS scores remains limited. This disparity affects patient survival rates and quality of life, underscoring the need for an in-depth analysis of factors that hinder access. We aimed to explore the proportion of FDA drug approvals in solid and haematological malignancies in the past five years, having a high clinical implication as calculated by ESMO-MCBS SCORE, and their availability in India.

MATERIAL AND METHODS

This cross-sectional study was conducted to evaluate the availability and clinical value of FDA-approved cancer drugs in India from January 2020 to May 2025. A comprehensive list of cancer drugs approved by the U.S. Food and Drug Administration (FDA) between January 8, 2020, and May 31, 2025, was obtained through systematic queries on the FDA’s official website. We searched the FDA’s “Drugs@ FDA” database, using relevant keywords such as “oncology,” “haematology,” and “cancer indications” to filter the results. For each drug identified, detailed information was extracted, including the name of the drug, approved indications, year of approval, and pivotal clinical trial data. Only oncology drugs granted full approval by the US FDA were included, while those withdrawn from the market were excluded. We also excluded the investigational new drugs, diagnostic products, the different dosage forms of already approved drugs, other routes of administration of a single drug, vaccines, and drugs available in India. We then searched for these drugs in the EMA database by using their generic names. If the anticancer drugs were not found on searching these databases, they were considered as not approved. The status of drugs (approved/not approved), the indication of approval, and the date of approval or authorization were identified for all the drugs that are approved in India by CDSCO during the study period and that are approved by the FDA and EMA. The approved drugs were cross-checked for approval for the same indication. Two investigators searched these databases independently for solid and haematological indications, and drugs were confirmed as not approved if both investigators were unable to obtain the drug in the search. Any discrepancies between the findings of the first two investigators were resolved by the senior investigator. The search was conducted in June 2025, and approval of drugs after the date of the search is not reflected in this manuscript. Descriptive statistical analysis was done. The ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS) scores v1.1 assigned to these therapies based on clinical trial data, focusing on outcomes such as overall survival, progression-free survival, and quality-of-life benefits, were searched and recorded. The ESMO-MCBS scores were extracted from the published ESMO evaluation reports or, when unavailable, calculated using published clinical trial data following ESMO’s standardized framework. Drugs were categorized into two major groups:

1. Curative setting: Drugs were considered to have a clinically meaningful benefit if they achieved a score of ‘A’ or ‘B’ on the ESMO-MCBS.

2. Non-curative setting: A score of ‘4’ or ‘5’ on the ESMOMCBS was considered indicative of meaningful clinical benefit.

To validate our data, the availability status of these drugs in India was confirmed through searches in public databases, regulatory documents from the CDSCO, and market research reports. Cross-references were made with major pharmaceutical distributors and hospital formularies to verify market presence. Descriptive statistical methods were used to analyse trends in the clinical benefit scores of drugs unavailable in India. All data collection and analysis adhered to ethical guidelines, ensuring transparency and confidentiality. Approvals were not needed from the institutional ethics committee, as it did not involve patient information. This helped us to understand the accessibility challenges and clinical value of FDA-approved cancer drugs in the Indian context.

RESULTS

We identified the 109 oncological indications, including haematological and solid tumours, for which the US FDA has approved 84 anticancer drugs from 2019 to 2024. These drugs have 104 approvals in non-curative and 5 in curative settings, respectively [Table 1]. The ESMO MCBS score was found for each of these approvals. The list of drugs, approval status, and ESMO MCBS is provided as Supplementary File 1. All the drugs except camrelizumab have FDA approval; however, 26 (31%) out of 84 drugs do not have an EMA approval, with one drug, belantamab, having expired approval.

For the management of advanced non-small cell lung cancer alone, there are more than 10 indications, including drugs like Lazertinib, which is approved in frontline indications and is not available in India. For gastrointestinal malignancies like metastatic colorectal and cholangiocarcinoma, we observed 10 indications and two clinically relevant drugs (Avapritinib, Repotrectinib not available for gastrointestinal stromal tumours. Tumour agnostic approvals like BRAF and MEK inhibitors (Binimetinib, Cobimetinib ) for various malignancies like thyroid, melanoma, and lung cancer, and Encorafenib for colorectal cancer, Dostarlimab for MMR-deficient solid tumours, NTRK fusion inhibitors like Entrectinib have been in practice for many years now, but are not readily available in the Indian setup. Similarly, drugs with overall survival benefit in the second-line therapy and beyond in common cancers like ovarian cancer (Mirvetuximab) need to be made available for our patients. The ESMO-MCBS score was assessed for each approval to gauge clinical benefit. Total Number of Indications: 109

DISCUSSION

India is grappling with a rising cancer burden, a challenge compounded by the limited availability of cutting-edge cancer treatments. Worldwide, in terms of absolute incidence number, following China and the United States (ranked first and second, respectively), India ranks third, contributing 7.5% (1.38 million) of all new cancer incidences. Furthermore, India ranked second following China for cancer-related mortalities, accounting for 10.3% (0.89 million) of all cancer-related mortalities. The five most prevalent cancer types in India affecting both genders account for over 44% of all new incidences and cancer-related mortalities. With a 5-year prevalence of 16.5 per 100,000 people, female breast cancer remains the most prevalent cancer among females, contributing to 13.8% of all new cases, followed by oral (10.3%), cervical (9.2%), respiratory (5.8%), and oesophagus (5%) cancers, with 5-year prevalence of 11.4, 10.4, 3.5, and 3.4 per 100,000 people.^[1] With cutting-edge technologies like artificial intelligence and precision medicine, researchers are uncovering novel molecular targets that have led to the development of targeted therapies, immunotherapies, and next-generation drugs like antibody drug conjugates that improve survival rates and quality of life for patients.

India is the third-largest in terms of volume and fourteenth in terms of value.^[4] India's drug approval rate has significantly increased over the last two decades. The drug approval and regulatory authority in India, CDSCO, works in close liaison with international organizations such as the World Health Organization (WHO) and other regulatory organizations.^[5,6] The drug-approving authorities around the world investigate the integrity of the research data, assess the safety, efficacy, and quality of the new drugs they approve to safeguard . The CDSCO, headed by DCGI, ensures that medicines comply with all laws and regulations.

A company in India must apply for authorization from the licensing authority (DCGI) to manufacture or import a new medicine by submitting Form 44, along with the information provided in Schedule Y of the Drugs and Cosmetics Act, 1940, and the Rules, 1945. It must conduct clinical trials in compliance with the parameters outlined in Schedule Y and submit the results of these trials in the manner required to demonstrate its efficacy and safety in the Indian population. However, Rule 122A of the Drugs and Cosmetics Act, 1940, and Rule 1945 provide that the licensing authority may waive some of the risks if it determines that allowing the import of novel drugs based on evidence from trials conducted in other nations is in the public interest. Similarly, Rule 122A contains another clause that states that clinical trials may be omitted for newly licensed medications that have been in use for several years in other nations.^[7,8]

There are several potential reasons why pharmaceutical companies might not approach the CDSCO for marketing drugs in India. These reasons can be categorized into regulatory, economic, and strategic factors. CDSCO may have strict guidelines and documentation requirements, making the approval process time-consuming and resource-intensive. India often mandates local clinical trials for new drugs, which can increase costs and delay market entry. Konwar et al.^[9] In their 15-year analysis report, they evaluated drug lag for new drugs approved by the Indian regulator relative to the United States, European Union, and Japanese regulatory agencies. Optimizing time to approval is a challenge for any regulator, regardless of the country that they serve. We presume that economic factors also play a significant role when it comes to making a drug available in an low- and middle-income country (LMIC) like India. India’s focus on affordable healthcare often leads to price caps and profit margins that may not be attractive to multinational companies. The target population for certain drugs may be too small in India to justify the cost of obtaining approval and launching the product, combined with the cost of market penetration, like setting up distribution channels and marketing a new drug in a vast and diverse market like India, can be expensive.

There are some proposed strategic decisions, such as prioritizing regions with higher profitability, like North America and Europe, over India, which may contribute to delays in drug availability in the Indian market. Additionally, we have a competitive landscape due to the presence of generics and biosimilars, which may reduce the commercial viability of certain innovator drugs.

All these potential causes for delay, along with the obligation for strict post-marketing studies and pharmacovigilance, can be seen as an additional burden.

Over the past decade, the therapeutic landscape of haematolymphoid malignancies has undergone a remarkable transformation globally, driven by the advent of targeted therapies, bispecific antibodies, and cellular therapies. These innovations have significantly improved survival and quality of life in relapsed/refractory settings where conventional treatments have limited benefit. However, access to many of these novel agents remains restricted in India, despite their approval by major international regulatory bodies such as the U.S. FDA and the EMA. This analysis of recently FDA-approved agents for hematologic and lymphoid cancers reveals that over a dozen essential drugs are not yet approved in India. These include not only highly innovative therapies, such as bispecific antibodies, but also targeted small molecules and agents for transplant-related complications. Some of the most notable examples include Belantamab mafodotin, a BCMA-targeted antibody–drug conjugate (ADC) that has shown promise in heavily pretreated multiple myeloma. Belumosudil is a selective ROCK2 inhibitor used for chronic graft-versus-host disease (GVHD). Chronic GVHD is a growing post-transplant complication in India, yet there are limited systemic treatment options beyond steroids and calcineurin inhibitors. Bispecific antibodies such as Glofitamab, Epcoritamab, and Mosunetuzumab represent an important new class of agents in relapsed/refractory B-cell lymphomas, offering an off-the-shelf alternative to chimeric antigen receptor T-cell therapy (CAR-T). These agents have demonstrated high response rates even in heavily pretreated populations and are approved in the U.S. and Europe. Yet, none are currently accessible in India. Pacritinib, an agent approved for myelofibrosis with severe thrombocytopenia, addresses a key unmet need in a subset of patients often ineligible for ruxolitinib due to cytopenias. However, it remains unavailable in India despite FDA approval.

The limited availability of these drugs creates inequities in care delivery. In specific clinical scenarios, we are compelled to use available immunotherapy agents off-label for indications where another drug from the same class is FDA-approved but unavailable in India. For example, we may use pembrolizumab in settings where the originally approved agent (such as Dostarlimab or Cemiplimab) is indicated but not accessible in the Indian market. Through a conservative analysis of FDA-approved drugs that remain unavailable in India, we sought to identify and understand the underlying reasons for these gaps. This delay compromises India’s ability to achieve global benchmarks in cancer treatment. Addressing these barriers is paramount for ensuring equitable cancer care in India. Policymakers, healthcare professionals, and pharmaceutical companies must collaborate to facilitate the accessibility and affordability of innovative therapies. By addressing these challenges, India can enhance cancer outcomes and mitigate the burden of this disease.

TAKE HOME MESSAGE

Cancer drugs with a high ESMO-MCBS score represent treatments with proven, meaningful clinical benefit. Ensuring their availability and accessibility in low- and middle-income countries like India is imperative to bridge the global inequity in cancer care and to deliver value-based oncology at par with international standards.