Clival Database: Mastering the Drug Development Process

1. Breakthrough Approval miracle in clinical trials

2. Introduction The term “breakthrough approval” has become one of the most frequently used terms while discussing new medicines and the role of regulating bodies. This status represents change in the assessment and approval of new therapy to promote the application of innovative treatment for critical and life-threatening diseases.

3. What do BTD stands for? The FDASIA enacted the breakthrough therapy designation in 2012 to support the developement as well as the investor. Its purpose is to help catalyze the development and speed up the evaluation of drugs which demonstrate potential in clinical trials. This designation provides several benefits, including: • • Intensive Guidance: Sponsors get more frequent communication with the FDA on the drug’s development plan and proper trial design. • • Organizational Commitment: To ensure that an adequate and harmonized review process is done for a project that has input from various faculties, the FDA offer cross-disciplinary project lead. • • Rolling Review: Sponsors are allowed to submit the completed portions of the NDA or BLA instead of applying when all parts are ready.

4. Criteria for Breakthrough Therapy Designation 1. Serious or Life-Threatening Condition: The therapy must be directed towards a serious and/or life threatening chronic condition. 2. Preliminary Clinical Evidence: Some level of basic clinical data which show that the drug or the biologic may perform better than current treatments has to exist. 3. Significant Benefit Over Existing Treatments: The therapy should be much more effective than the existing treatments which are used for the management of the disorder.

5. Breakthrough Therapy Designation Process 1. Submission: The proposed breakthrough therapy is identified by the sponsor, which is often the pharmaceutical or biotechnology company that will receive the designation, and submits to the FDA a request for the designation with preliminary clinical data and a description of the therapy’s prospect. 2. FDA Review: The FDA goes through the submission process, which establishes the severity of the disease being treated, and the initial evidence on how the therapy under review can significantly improve the outcome. 3. Designation Decision: When the FDA provides the breakthrough therapy designation, the therapy has more assistance and cooperation at every stage of development.

6. List of drugs that has been approved under the act Keytruda (pembrolizumab): • Indication: Non-small cell lung cancer abbreviated as NSCLC is a type of lung cancer that falls under the category of, Lung cancer. • Approval: This was awarded breakthrough therapy designation because Keytruda had improved the overall survival compared to chemo in patients with stage IV NSCLC. Ukoniq(umbralisib): • Indication: Lymphoma • Approval: This oral medication was granted by the FDA breakthrough therapy for its efficacy as a treatment for specific forms of lymphoma as evidenced by a positive outcome on the tumor size.

7. Conclusion Breakthrough approval is another innovation of the regulatory systems with the aim of moving potentially beneficial therapies to patients with serious and life-threatening diseases more quickly. In essence, this designation enables the enhancement of the development and review process to address the unmet medical needs and bring hope where there is limited alternatives.

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