MEDCAC Meeting 11/17/2010 - On Label and Off-Label Use of Autologous Cellular Immunotherapy Treatment of Metastatic Prostate Cancer

August 31, 2010	Announced meeting.
Those interested can register at: http://www.cms.gov/apps/events/event.asp?id=610&Kw=&Mh=NoMonth&cboOrder=date&Yr=NoYear&type=2
September 24, 2010	Posted FR Notice and questions to panel.
October 26, 2010	We will be broadcasting the meeting via Webinar. You must register for the Webinar portion of the meeting at https://webinar.cms.hhs.gov/txmetaprostatemedcac1117/event/registration.html
November 10, 2010	Posted technology assessment.
November 15, 2010	Posted agenda, roster and speaker list.
November 18, 2010	Posted scoresheet [PDF, 35KB] from meeting.
December 27, 2010	Posted minutes [PDF, 129KB] and transcript [PDF, 339KB] from meeting.

Federal Register Notice
Clifford Goodman, PhD, Chair
Saty Satya-Murti, MD, Vice Chair
James Rollins, MD, Division Director, Division of Items and Devices, Coverage and Analysis Group
Maria Ellis, Executive Secretary

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7:30 – 8:00 AM	Registration
8:00 – 8:15 AM	Opening Remarks—Maria Ellis/James Rollins, MD/Clifford Goodman, PhD
8:15 - 8:25 AM	CMS Presentation of Voting Questions – Lori Paserchia, MD
8:25 – 8:50 AM	James L. Gulley, MD, PhD, FACP, Director, Clinical Trials Group, Laboratory of Tumor, Immunology and Biology, Principal Investigator, Medical Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health
8:50 – 9:50 AM	TA Presentation:  David Mark, MD, MPH, Senior Scientist, Blue Cross Blue Shield Association, Technology Evaluation Center
9:50 – 10:05 AM	BREAK
10:05 – 10:50 AM	Scheduled Public Comments (Refer to Speaker List)
Public attendees, who have contacted the executive secretary prior to the meeting, will address the panel and present information relevant to the agenda. Speakers are asked to state whether or not they have any financial involvement with manufacturers of any products being discussed or with their competitors and who funded their travel to this meeting.
10:50 – 11:05 AM	Open Public Comments
Public Attendees who wish to address the panel will be given that opportunity
11:05 – 12:00 PM	Questions to Presenters
12:00 – 1:00 PM	LUNCH (on your own)
1:00 – 2:00 PM	Initial Open Panel Discussion: Dr. Goodman
2:00 – 3:00 PM	Formal Remarks and Voting Questions
The Chairperson will ask each panel member to state his or her position on the voting questions
3:00 – 4:00 PM	Final Open Panel Discussion: Dr. Goodman
4:00 – 4:30 PM	Closing Remarks/Adjournment: Dr. Rollins & Dr. Goodman

Download meeting minutes [PDF, 113KB].

FDA Label -
PROVENGE is an autologous cellular immunotherapy indicated for the treatment of asymptomatic or minimally symptomatic metastatic castrate resistant (hormone refractory) prostate cancer.

Voting Questions

For all voting questions, the health outcomes of interest are: overall survival, control of disease-related symptoms, and the avoidance or minimization of the burdens to patients associated with anticancer therapy.  The comparator is the management that the patient would otherwise have received.  

For the voting questions, use the following scale identifying level of confidence - with 1 being the lowest or no confidence and 5 representing a high level of confidence.

1. How confident are you that there is adequate evidence to determine whether or not the use of autologous cellular immunotherapy treatment of asymptomatic or minimally symptomatic metastatic castrate resistant prostate cancer significantly improves:

   1. Overall survival?

      1

      2

      3

      4

      5

      
      

   2. Control of disease-related symptoms?

      1

      2

      3

      4

      5

      
      

   3. Avoidance or minimization of the burdens associated with anticancer therapy while maintaining overall survival and control of disease-related symptoms?

      1

      2

      3

      4

      5

      Note: Questions 2 -6 should be addressed only for those outcomes under question 1 where the panel is confident that there is at least intermediate confidence (mean vote of 2.5) that there is adequate evidence to make the determination of improvement.

2. How confident are you that there is adequate evidence to conclude that autologous cellular immunotherapy treatment significantly improves overall survival in patients with asymptomatic or minimally symptomatic metastatic castrate resistant prostate cancer?

   1

   2

   3

   4

   5

   
   

3. How confident are you that there is adequate evidence to conclude that autologous cellular immunotherapy treatment significantly improves control of disease-related symptoms in patients with asymptomatic or minimally symptomatic metastatic castrate resistant prostate cancer?

   1

   2

   3

   4

   5

   
   

4. How confident are you that there is adequate evidence to conclude that autologous cellular immunotherapy treatment significantly improves the avoidance of the treatment burdens (e.g., access, delivery, or side-effects) associated with anticancer therapy in patients with asymptomatic or minimally symptomatic metastatic castrate resistant prostate cancer?

   1

   2

   3

   4

   5

   
   

5. How confident are you that these conclusions are generalizable to unlabeled use in:

   1. Patients whose prostate cancer has not metastasized?

      1

      2

      3

      4

      5

      
      

   2. Patients who have metastatic, castrate resistant disease and symptoms more severe than minimally symptomatic?

      1

      2

      3

      4

      5

      
      

   3. Patients who have metastatic prostate cancer but who have not failed hormonal therapy?

      1

      2

      3

      4

      5

   
   

6. How confident are you that these conclusions are generalizable to:

   1. Community based settings?

      1

      2

      3

      4

      5

   2. Patients belonging to demographic groups that may have been under-represented in the enrolled clinical trial populations?
      

      1

      2

      3

      4

      5

   Discussion Questions

7. Do you believe that there is adequate evidence to identify patients who are more likely or less likely to respond favorably to autologous cellular immunotherapy treatment based on pretreatment evaluation of any of the following factors?

   1. Site(s) or number of metastasis(es) as detected by imaging studies
   2. Gleason score
   3. Alkaline phosphatase (U/L)
   4. Hemoglobin (g/dL)
   5. Serum LDH (U/L)
   6. Serum PSA (ng/mL)
   7. Pain associated with metastatic, castrate-resistant prostate cancer
   8. Other
   
   
8. What significant evidence gaps exist regarding the health outcomes attributable to autologous cellular immunotherapy treatment?
   1. For the FDA-labeled indication?
   2. For off-label uses?
   
   
9. What clinical study designs would adequately address any evidence gaps?

Download scoreshet [PDF, 35KB]

Transcript 1
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