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  1. 2023 OCE Annual Report

Oncology Research and Development 2023

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Project Catalyst

In 2023, OCE’s Project Catalyst provided guidance and expanded the educational resources available to small pharmaceutical companies and academic life science incubators to support informed anticancer therapy development, including:

  • Discussions with Academic Accelerator Life Science Hubs to include the Pennsylvania Vagelos Life Science & Management Program, and Harvard University.
  • New Bench to Bedside Chats included discussions on: Expedited Programs for Serious Conditions––Drugs and Biologics; and currently in development First in Human-Chemistry Manufacturing and Controls (CMC) considerations for Phase 1 CMC Guidance.
  • Developed plans for a 2024 workshop with FDA CDER SBIA and NCI SBIR to produce a webinar aimed at companies involved in the SBIR CARES program as well as academic accelerators and small start-up companies regarding FDA Interactions and functions.
  • Collaborated with the Foundation for the National Institutes of Health, National Cancer Institute, and FDA Oncology Pediatric and Rare Tumors Programs to hold a public meeting, “Development of a Public Private Partnership Developing Anti-Cancer Therapies for Ultra-Rare Tumor Indications.” This virtual meeting on August 24, 2023, discussed plans for the creation of a public-private partnership to guide and support development of new drugs for the treatment of patients with ultra-rare cancers. Discussions included an envisioned framework for a collaborative open-science, open-drug development process designed to develop candidate targeted drugs for specific rare cancer indications. FNIH solicited feedback from the scientific community and patient advocate groups on the proposed plans.
  • Educational initiatives included the following conferences, presentations and working groups: DIA China Presentation, FDA’s Rare Disease Day Presentation, the Parker Institute for Cancer Immunotherapy and Friends of Cancer Research regarding Potential CRIPSR Screens as Phase 1 studies for CAR-T and Synthetic TCR approaches, An AACR Annual Meeting Entrepreneurial track presentation, a Convergence Accelerator Conference Roundtable discussion in Tucson, and The Society for Immunotherapy annual meeting.

Project Catalyst welcomes questions regarding oncology drug development plans that are premature for a pre-IND submission, and values input regarding other efforts that would be useful to early-stage oncology drug development programs. 

OCE Scientific Collaborative

The OCE Scientific Collaborative supports FDA oncology staff who participate in regulatory science research, including internal research projects and collaborations with external experts. OCE research focuses on applied (rather than basic) research questions to address specific challenges encountered during the IND and NDA/BLA review process. 

Program highlights from 2023 include:

  • Launched the OCE-Funded Active Extramural Research Projects web page.
  • Released two Requests for Applications in the areas of ultra rare cancers and health equity in oncology trials (RFA-FD-23-006 and RFA-FD-23-008).
  • Worked with the OCE Real World Evidence Program to execute multiple Research Collaboration Agreements (under “Selected Currently Active Oncology RWE Collaborations”).
  • Presented an overview of OCE scientific priorities at 2023 FDA BAA Day.
  • Funded five new applied research projects:
    • Leveraging Real World Data to Investigate the National History of Rare Cancers Treated in the US Community Oncology Setting to Provide Clinical Context to Inform Future Research, Ontada.
    • Development of neuroblastoma tissue diagnostic utility through fusion of deep learning-based image analytics and targeted multiplex proteomics, Stanford University.
    • SMARTCORE Technology: Using AI and Patient Tissue to Identify Potential Cancer Therapies for Ultra-rare Cancers, Fred Hutchinson Cancer Research Center.
    • Using the UNC Clinical Data Warehouse to Evaluate the Benefit/Risk Ratio and Optimal Doses of Molecularly Targeted Therapies and Novel Biologics in Real World Patients, Triangle CERSI.
    • Principal stratification methods and software for intercurrent events in clinical trials, Triangle CERSI.

Project Significant

Project Significant (Statistics in Cancer Trials) holds discussions with the Biopharmaceutical Section of the American Statistical Association, the LUNGevity Foundation and other oncology stakeholders to further the design and analysis of cancer clinical trials with the goal to advance cancer therapies. Visit our web page for links to the latest publications.

Seven key topics in 2023 included:

  • January 10, “Non-inferiority Cancer Clinical Trial Design Considerations when Data from a Single Foreign Country is Available.” This discussion examined the potential non-inferiority cancer clinical trial designs (innovative/Bayesian) using available information from clinical trials conducted outside of the regulatory region for marketing authorization.
  • March 9, “Statistical Considerations in the Early Interim Overall Survival Analysis in Indolent Cancers for Evaluation of Risk.” This discussion explored the possibility of pre-specifying criteria for unacceptable risk for early OS analysis in indolent cancer trials. 
  • April 13, “Consideration of Criteria for Evaluation of Surrogate Endpoint.” This discussion focused on whether the criteria used previously by Shi Q et al, or a modified criteria may be needed for evaluation of future surrogate endpoints.
  • May 11, “Cancer Clinical Trial Design and Analysis Considerations in Evaluating Treatment Effect in Marker Negative Population–Part 2.” This event discussed cancer clinical trial design and analysis considerations and understanding and measuring uncertainties in the evaluation of treatment effect in the marker negative subgroup.
  • August 10, “Design Considerations in the Evaluation of Contribution of Effect of Combination of Two New Investigational Drugs in Randomized Cancer Clinical Trials." This forum focused on trial designs and analysis methods in a randomized trial setting that can provide the requisite information to establish safety and efficacy of new drug combinations while exposing the least number of patients to a potentially less effective monotherapy.
  • October 12, “Impact of Cross-over in the Evaluation of Overall Survival in Randomized Cancer Clinical Trials.” This discussion examined the different statistical methods to understand the impact of cross-over in the evaluation of overall survival in randomized cancer clinical trials where some patients in the control arm receive investigational drug after disease progression.                        
  • December 12, “Considerations in requiring blinded independent central review of radiological scans in randomized cancer clinical trials when progression-free survival is the primary endpoint.” This open forum focused on when or if blinded independent central review of all radiographs from all patients in a randomized cancer clinical trial is necessary. 

 

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