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The Ministry of Food and Drug Safety (MFDS) has issued comprehensive guidelines for conducting clinical trials of anticancer drugs, applicable to all cancers targeting specific molecular alterations. According to these recent guidelines, the focus is on ensuring scientific evidence, safety, efficacy, and Regulatory compliance throughout the trial process.

Introduction 

This guideline is applicable to the development of anticancer drugs that target specific molecular alterations across multiple cancer types—such as colon, thyroid, cervical, and breast cancers.

  • It defines anticancer agents as those targeting molecular markers across various types of cancer.
  • The Regulatory framework for these drugs remains the same as that for cancer-type-specific drugs.
  • Safety and efficacy must be assessed, and consultations with the MFDS are required.

Background on the Development of Anticancer Drugs 

  • Traditionally, cancers have been classified based on pathology and organ involvement. Anticancer drugs, however, focus on molecular-level changes.
  • Development is influenced by internal factors (e.g., NTRK gene fusion) and external factors (e.g., tumor microenvironment, immune system response).
  • These molecular targets provide broader potential for patient benefit beyond a single cancer type.

Determining the Suitability of Clinical Trials Across Cancer Types

Biology

  • A deep understanding of molecular mechanisms and drug responses is essential, as well as evidence showing the drug's efficacy across cancer types. This should be backed by scientific data, not just molecular theory.
  • The drug's efficacy across various cancer types depends on scientific validation, not just on molecular target similarity.
  • Early trial data is required to confirm that efficacy is consistent across cancers.

Test Subject Population 

  • Rare molecular alterations might make such trials more feasible.
  • In cases of common molecular alterations (like RET-positive lung or thyroid cancer), the drug may be effective across various types.

Clinical Pharmacology and Clinical Safety/Efficacy 

  • Pharmacology: Involves studying pharmacokinetics (PK) and pharmacodynamics (PD), including sample collection, tumor burden, patient characteristics, and site of the tumor.
  • Safety and Efficacy: While early data show consistent response rates across cancer types, there's insufficient scientific basis. Thus, focusing on cancers meeting a defined threshold response level is more appropriate.

Key Considerations in Clinical Trials Across Cancer Types

Non-Clinical, Population, and Cancer-Specific Data

  • Trial planning should be based on a combination of nonclinical studies, population data, and cancer-specific insights.
  • Factors like sample size of subgroups and development strategies must be evaluated.

Non-Clinical Evaluation 

  • Trials for indications that span multiple cancer types must follow existing guidelines like:
    • Guidelines for Clinical Trials
    • Q&A on Guidelines for Nonclinical Trials of Anticancer Drugs

Subject Selection  

  • Patients are selected based on disease, population needs, and unmet medical requirements.
  • The drug's indication can be defined through subgroup characteristics or by including/excluding certain populations.

Clinical Trial Design 

  • The design is shaped by treatment options, unmet needs, expected outcomes, commercial goals, and the size of the targeted patient population.
  • Early development focuses on evaluating the drug’s activity across cancer types.

Conclusion
 Successful anticancer drug trials require a deep understanding of molecular biology, careful subject selection, and strict adherence to Regulatory expectations.

South Korea's MFDS guidelines emphasize early regulatory consultations, scientific evidence, and patient safety throughout the trial process.

Contact Freyr today to streamline your anticancer drug submissions and accelerate market entry in South Korea.