• L.J. Wei (Harvard University) - Utilizing Multiple Endpoints and Multiple Studies for Assessing Totality of Evidence of Treatment Effect
• Robert Beckman (Georgetown University) - Clinical Trial Designs for Rare Diseases: The Confirmatory Basket Trial and the Informational Design
  

  </li>
  <li>Discussant: Kun Chen (University of Connecticut)</li>
</ul>   
  </td>
</tr>
<tr>
  <td>9:55 AM - 10:15 AM</td>
  <td>Coffee Break</td>
</tr>
<!-- Session 2 -->
<tr>
  <td>10:15 AM - 12:00 PM</td>
  <td>
<ul class="agenda">
  <li class="mb-2">
    Session II: Review of Statistical Approaches for Developing Rare Disease Medicines: Regulatory, Industry and Academic Perspectives
  </li>
  <li class="mb-2">Chair: Charlie Cao (Biogen)</li>
  <li>Speakers:
    <ul>
      <li class="mb-1">
    Jingjing Ye (FDA) - Ahead of FDARA Sec. 504 Implementation: Years in
    Review and Years Ahead in Pediatric Cancer Clinical Trials
      </li>
      <li class="mb-1">
    Ziliang Li (Vertex), Chenkun Wang (Vertex), Sammi Tang (Servier) - Designing a Clinical Trial for Rare Disease with Historical
    Control - A Practical Roadmap with Case Studies
      </li>
      <li>
    Rima Izem (Children’s National) - Comparative Effectiveness Using Natural History Data, A Case Study
      </li>
    </ul>       
  </li>
</ul>   
  </td>
</tr>
<tr>
  <td>12:00 PM - 1:00 PM</td>
  <td>Lunch</td>
</tr>
<!-- Session 3 -->
<tr>
  <td>1:00 PM - 2:10 PM</td>
  <td>
<ul class="agenda">
  <li class="mb-2">
    Session III: Statistical Methods for Rare Disease Clinical Trials (II)
  </li>
  <li class="mb-2">Chair: Sammi Tang (Servier)</li>
  <li class="mb-2">Speakers:
    <ul>
      <li class="mb-1">
    Ming-Hui Chen (University of Connecticut) - Leveraging Historical Information: Methods and Applications
      </li>
      <li>
    Qing Liu (Quantitative and Regulatory Medical Science, LLC) - On Analysis of Single Arm Trial with Natural History Controls: A Virtual
    1-to-Many Matched Control Method
      </li>
    </ul>
  </li>
  <li>Discussant: Mike Hale (Takeda)</li>
</ul>   
  </td>
</tr>
<tr>
  <td>2:10 PM - 2:30 PM</td>
  <td>Coffee Break</td>
</tr>
<!-- Session 4 -->
<tr>
  <td>2:30 PM - 4:15 PM</td>
  <td>
<ul class="agenda">
  <li class="mb-2">
    Session IV: Case Studies in Rare Disease Drug Development
  </li>
  <li class="mb-2">Chair: Jeffery Palmer (Pfizer)</li>
  <li>Speakers:
    <ul>
      <li class="mb-1">
    Balram Gundapaneni (Pfizer) - Results of the Pivotal Phase III ATTR- ACT Study Using a Novel Primary Endpoint / Rank-Based Method
      </li>
      <li class="mb-1">
    Feng Tai (Agios), Yanwei Zhang (Agios), Yanqiong Zhang (Agios) - Bayesian Dynamic Borrowing through Hierarchical Model to Assess
    Treatment Effect - A Phase I Case Study
      </li>
      <li>
    Peng Sun (Biogen) - Adaptive Historical Data Borrowing in a Rare Disease Setting
      </li>
    </ul>
  </li>
</ul>   
  </td>
</tr>
<tr>
  <td>4:15 PM - 4:20 PM</td>
  <td>Closing Remarks: Yang Song & Sammi Tang (2019 NERDS workshop co-chairs)</td>
</tr>