Navigating the complexities of AML drug development

The webinar, "Navigating the Complexities of AML Drug Development," is moderated by Scott Smith, senior vice president and global franchise head of hematology at Parexel. The main speakers are Sinan Sarac, senior vice president, regulatory strategy, Europe at Parexel, and Maria Ahmad, senior medical director at Parexel. The discussion covers various AML (Acute Myeloid Leukemia) drug development aspects, including historical treatment methods, current trends, and regulatory challenges.

Key Points Discussed:

1. Historical treatment of AML:
   Traditional use of induction chemotherapy, specifically the 7+3 regimen, which has been the standard of care for nearly 30 years. This regimen involves a combination of cytarabine and an anthracycline, requiring extended hospital stays due to high toxicity.
2. Current trends in AML treatment:
   Renaissance of Antibody-Drug Conjugates (ADCs) in AML treatment. Improved linker technology has made these treatments more effective and less toxic. The number of clinical trials involving ADCs is increasing exponentially.
3. Urgency and complexity of AML treatment:
   AML treatment is urgent and complex due to the disease's heterogeneous nature and rapid progression. Certain subtypes, like acute promyelocytic leukemia (APML), are considered oncologic emergencies.
4. Regulatory challenges:
   Differences between FDA and EMA policies. The FDA is more accepting of complete remission (CR) and duration of CR as endpoints for accelerated approval, while the EMA requires additional supporting data. A global development strategy is essential to navigate these regulatory differences.
5. Novel therapies:
   Insights into novel therapies such as CAR-T cell therapy, bispecific antibodies, and small molecule inhibitors. Challenges of managing the unique toxicities associated with these therapies were also addressed.
6. Logistical challenges in clinical trials:
   Challenges in data collection, site training, and protocol amendments during clinical trials. Emphasis on timely data collection, staff training, and maintaining up-to-date site-facing documents for optimal protocol execution.
7. Patient-centric approach:
   Importance of maintaining a patient-centric approach throughout the drug development process. Ensuring patient comfort, understanding their options, and providing thorough information about the clinical trial and its assessments.

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