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Committee Detail

Hide Section - GENERAL INFORMATION

GENERAL INFORMATION

Committee NameOncologic Drugs Advisory CommitteeAgency NameDepartment of Health and Human Services
Fiscal Year2019Committee Number35
Original Establishment Date11/28/1990Committee StatusChartered
Actual Termination Date Committee URLhttp://www.fda.gov/AdvisoryCommittees/Committee...
New Committee This FYNoPresidential Appointments*No
Terminated This FYNoMax Number of Members*14
Current Charter Date9/1/2018Designated Fed Officer Position Title*Center for Drug Evaluation & Research, FDA
Date Of Renewal Charter9/1/2020Designated Federal Officer Prefix
Projected Termination Date Designated Federal Officer First Name*Lauren
Exempt From Renewal*NoDesignated Federal Officer Middle NameTesh
Specific Termination AuthorityDesignated Federal Officer Last Name*Hotaki
Establishment Authority*Authorized by LawDesignated Federal Officer SuffixPharm.D., BCPS, BCIDP
Specific Establishment Authority*21 U.S.C. 394Designated Federal Officer Phone*(301) 796-9001
Effective Date Of Authority*11/28/1990Designated Federal Officer Fax*301-847-8533
Committee Type*ContinuingDesignated Federal Officer Email*lauren.hotaki@fda.hhs.gov
Presidential*No
Committee Function*Scientific Technical Program Advisory Board
Hide Section - RECOMMENDATION/JUSTIFICATIONS

RECOMMENDATION/JUSTIFICATIONS

Agency Recommendation*Continue
Legislation to Terminate RequiredNot Applicable
Legislation StatusNot Applicable
How does cmte accomplish its purpose?*The committee reviews and evaluates data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of cancer and makes appropriate recommendations to the Commissioner of Food and Drugs. The Office of Oncology Drug Products also uses committee members as subject matter experts, on an as needed basis.
How is membership balanced?*Members are selected from academic and practice settings and include practitioners knowledgeable in the field of general oncology, pediatric oncology, hematological oncology, immunology oncology, biostatistics, and other related professions. The committee includes one technically qualified voting member who is identified with consumer interests. The Committee may also include one non-voting member who is identified with industry interests.
How frequent & relevant are cmte mtgs?*The committee met four times in FY-19.

On October 10, 2018 the committee discussed biologics license application 761088 for CT-P10, a proposed biosimilar to Genentech, Inc.’s RITUXAN (rituximab), submitted by Celltrion, Inc. The proposed indications (uses) for this product are for the treatment of adult patients with (1) relapsed or refractory, low-grade or follicular, CD20-positive, B-cell Non-Hodgkin’s Lymphoma (NHL) as a single agent; (2) previously untreated follicular, CD20-positive, B-cell NHL in combination with first-line chemotherapy and, in patients achieving a complete or partial response to CT-P10 in combination with chemotherapy, as single-agent maintenance therapy; and (3) non-progressing (including stable disease), low-grade, CD20-positive, B-cell NHL as a single agent after first-line cyclophosphamide, vincristine, and prednisone. The committee uanimously agreed that the totality of analytical, pharmacologic, immunogenicity, and clinical evidence with orthogonal results filling information gaps, demonstrates robust evidence of high similarity between US-licensed Rituxan and CT-P10 and supports the licensure of CT-P10 for all three proposed indications. One member indicated the safety signal as a concern but considered the lower number of patients treated as a possible reason for the imbalance. One member stated that the molecular discrepancies identified in analysis were overwhelmed by the clinical results. Please see the transcript for details of the committee discussion. Agency Action: The Agency is currently evaluating recommendations made during the advisory committee meeting.
On February 26, 2019 the committee discussed new drug application (NDA) 212306 for selinexor tablets, application submitted by Karyopharm Therapeutics Inc. The proposed indication for this product is in combination with dexamethasone, for the treatment of patients with relapsed refractory multiple myeloma who have received at least three prior therapies and whose disease is refractory to at least one proteasome inhibitor, at least one immunomodulatory agent, and an anti-CD38 monoclonal antibody. The majority of the committee (8 to 5) agreed that the approval of selinexor should be delayed until the results of the randomized phase 3 BOSTON trial are available. These members agreed that the data from the STORM trial are inconclusive to allow for an adequate assessment of the safety and efficacy in the proposed patient populations, does not meet the FDA regulatory standards needed to prove the safety and effectiveness of selinexor. They concluded that it was undeterminable whether selinexor provides a benefit that outweighs the risks. The five committee members who voted “No” noted that patients need options for this indication and selinexor should be granted accelerated approval now. Agency Action: On July 3, 2019, the Food and Drug Administration granted accelerated approval to selinexor (XPOVIO, Karyopharm Therapeutics) in combination with dexamethasone for adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody.
On May 14, 2019, during the morning session, the committee discussed new drug application (NDA) 211810 for pexidartinib capsule, submitted by Daiichi Sankyo, Inc. The proposed indication (use) for this product is for the treatment of adult patients with symptomatic tenosynovial giant cell tumor also referred to as giant cell tumor of the tendon sheath or pigmented villonodular synovitis, which is associated with severe morbidity or functional limitations, and which is not amenable to improvement with surgery. During the afternoon session, the committee discussed new drug application (NDA) 212166 for quizartinib tablets, submitted by Daiichi Sankyo, Inc. The proposed indication (use) for this product is for the treatment of adults with relapsed or refractory acute myeloid leukemia which is FLT3-ITD positive, as detected by an FDA-approved test. Many committee members stated that pexidartinib demonstrated benefit as measured by response rate; however, many felt that although the patient-reported outcomes also represented benefit, there is less certainty in the magnitude of effect due to missing data. The committee members discussed that there is a clear benefit to some individual patients, but that the evidence is less clear regarding benefit at a population level. The committee members discussed that there is concern about hepatotoxicity and that a Risk Evaluation and Mitigation Strategy (REMS) is needed to educate prescribers and further eavluate this risk. The committee members also discussed the number of open questions regarding this drug, given the relatively small sample size due to the rarity of the disease. These questions included whether the optimal dose and duration of therapy had been selected given the risk of hepatotoxicity. The majority of the committee agreed that the demonstrated benefit of pexidartinib outweighs the risks of the drug in the proposed indication. Several committee members who voted “Yes” emphasized the lack of treatment options for patients with TGCT as a factor for their vote. Those voting “No” stated concerns over liver toxicity, proper use in clinical practice, and the possibility that pexidartinib may be used in patients for whom surgerical resection is appropriate. Please see the transcript for details of the Committee discussion. The committee members discussed the uncertainty that remains around the overall survival (OS) analysis and the question of benefit and magnitude of benefit. Several committee members stated that the high drop-out rates and the lack of follow up in those patients are very concerning. The committee also discussed concerns about the uncertainty of clinical benefit of other non-standard endpoints such as increased transplant rates and CRi (complete remission with incomplete hematologic recovery). Committee members voiced the opinion that it would be acceptable to advise prescribers of the risk of using QT prolonging drugs with quizartinib, but a contraindication for use with QT prolonging drugs is not warranted, since in the current era of targeted therapies, oncologists are expereienced with managing patients on drugs with QT prolonging effects. Additionally, the Committee indicated a relatively low concern over the risk of QT prolongation with the administration of quizartinib for patients with relapsed or refractory AML who would have no other treatment options, The committee also discussed advising the administration of beta blockers to prevent arrhythmias was not something that should be blanketly recommended. The majority of the committee members voted “No” that the results of Study AC220-007 did not demonstrate that treatment with quizartinib provides for a benefit that outweighs the safety risk for patients with relapsed or refractory FLT3-ITDpositive AML. Two committee members stated that the type of information from the trial was similar to that typically provided by a Phase II trial and that additional study is needed to demonstrate clinical benefit. Many committee members stated that the difference in patients randomized but not treated in the control arm vs. the quizartinib arm and the potential bias that this presented was very concerning. One member who voted “Yes” stated that there is potential for risk with QT prolongation, but that it wasn’t clearly seen. Please see the transcript for details of the Committee discussion. On August 2, 2019, the Food and Drug Administration approved pexidartinib (TURALIO™, Daiichi Sankyo) capsules for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Pexidartinib is the first systemic therapy approved for patients with TGCT. Agency Action: The Agency is currently evaluating recommendations made during the afternoon session of the advisory committee meeting.
On June 20, 2019 the Pediatric Subcommittee of the Oncologic Drugs Advisory Committee met. During the morning session, the particular matter for this meeting was reviewed and discussion of the FDA Reauthorization Act of 2017 (FDARA) mandated Relevant Pediatric Molecular Target List posted on the FDA website. FDA is required by statute to review and update the previously approved and published lists. The focus of the discussion was limited to two target “classes” included in the Relevant Pediatric Molecular Target List: (1) targets linked to cell lineage and (2) targets on normal immune cells and cells in the tumor microenvironment. Planned introductory presentations were on: (1) cell-based therapy approaches to childhood cancer and (2) novel membrane antigen determinants in pediatric tumors. During the afternoon session, information was presented to gauge investigator interest in exploring potential pediatric development plans for a product in development for adult cancer indications. The subcommittee considered and discussed issues concerning diseases to be studied, patient populations to be included, and possible study designs in the development of this product for pediatric use. The discussion also provided information to the Agency pertinent to the formulation of written requests for pediatric studies, if appropriate. The product under consideration was ONC201, presentation by Oncoceutics Inc. Members of the subcommittee suggested adding several targets to the current list including: Cluster of differentiation (CD) 99, Pregnacy-associated plasma protein-A (PAPP-A), Insulin-like growth factor binding protein-like 1 (IGFBPL1), and Paired-like homeobox 2B (PHOX2B). The subcommittee agreed that sufficient evidence was present for the addition of the four molecular targets mentioned. The subcommittee members made no comments on the removal of any relevant target currently on the List of Molecular Targets Associated with Specific Cell Lineage Determinants. The subcommittee members suggested moving Transforming growth factor beta (TGFβ) receptors to this list from the “Others” list and adding Interleukin 15 (IL-15), Hypoxia-inducible factor (HIF), V-domain Ig suppressor of T cell activation (VISTA), Colony-stimulating factor 1 receptor (CSF1R), and CD105 to the target list. One committee member commented on the current usage of several oncologic agents for pediatrics that target IL15 receptors. Both VISTA and CSF1R were mentioned to have active, ongoing trials that provided justification for addition to the list. In addition, a subcommittee member referenced Vascular endothelial growth factor (VEGF) targets being relevant in the adult oncology space and questioned their relevance in the pediatric world. Another member noted data that highlighted CD105 and its compensatory pathway for VEGF inhibition. The subcommittee members made no comments on the removal of any relevant target currently on the List of Relevant Targets on Normal Immune Cells and Cells in the Tumor Microenvironment. The subcommittee discussed the need for strong preclinical data or substanital data from adult combination models that provide promising signals before starting combinatorial approaches in the pediatric oncology space. A subcommittee member stated the need for caution when considering combinatorial approaches, given the fact that we do not have a full understanding of the potential toxicities when combining these agents. Another subcommittee member commented that current chimeric antigen receptor T (CAR-T) cell therapy is a combination therapy with lymphocytes. In the discussion of CAR-T therapy, another member mentioned the associated antigen loss observed with CAR-T therapy and the importance of rational decision making on multiantigen targeting strategies to ensure durable remissions. Furthermore, suggested combinations included: (1) checkpoint inhibitors and immune-based therapies, (2) T cell activation, and (3) epigenetic modifiers to enhance T cell function. Another subcommittee member commented on strategies needed to upregulate major histocompatibility complex (MHC) loss were vital which also included comments on using nanotherapy targeted approaches or some small molecule delivery systems. In addition, one member mentioned that adding other therapies, such as chemotherapy, could represent a regulatory challenge on how to determine how effective immune-based therapy was. Please see the transcript for details of the subcommittee’s discussion. The subcommittee agreed that the Pediatric Preclinical Testing Consortium (PPTC) is the right mechanism for evaluating this agent and should be considered for ONC201 in pediatric pre-clinical tumor models and possible pediatric development of ONC201 beyond high grade gliomas. The subcommittee further commented that given the uncertainty in determining when, where, and whether the Dopamine receptor D2 (DRD2) or Caseinolytic mitochondrial matrix peptidase proteolytic subunit (ClpP) mechanism is effective for anticancer activity, further evaluation is needed to clarify and understand the relevance of its true mechanism of action. The subcommittee stated their interest in trying to identify other pediatric cancers and to identify which are the pediatric cancers that are relevant to those two targets (DRD2 and ClpP). The subcommittee agreed that designated strata should be in place to study central nervious system (CNS) metastases. The subcommittee also agreed that broad phase 2 studies should clearly state that CNS metastates are included to be able to understand the CNS penetration properties of ONC201 and its potential role in addressing brain metastases in children. One member mentioned that CNS metastases is often an exclusion criteria when conducting phase 2 studies. The subcommittee agreed that robust historical data for diffuse intrinstic pontine gliomas (DIPG) would be sufficient to conduct a prospective trial that compares clinical outcomes such as overall survival. Moreover, a subcommittee member commented that a comparator arm would not be needed and the historical controls could be used to isolate the effect of ONC201. One member mentioned the additive effect with radiation therapy as a consideration. The subcommittee agreed that there is no current evidence for toxicities in younger patients and that patients younger than 2 years of age should not be excluded. One member noted that patients greater than 3 years of age have a different prognosis and more historical control data than patients less than 3 years of age and that needs to be considered in trial design. One member addressed a potential contributing factor to exclusion is formulation, given that pediatric patients, specifically in the DIPG context, could have swallowing dysfunction. This member encouraged development of a non-pill formulation. Additionally, a member stated the need for a defined dose for younger patients in clinical studies even if older patients were included in the study population. The subcommittee agreed that for DIPG, a robust historical control could be utilized as a comparator and that a potential endpoint could be overall survival. Another member commented that single agent activity could be studied, such as in neuroblastomas and other tumors. The member further stated that preclinical signals could then be identified and help serve as a guide for future trial development. Another member discussed the potential for a trial design that allowed patients to receive radiation therapy after receiving the test drug, given the current data on combination therapies. Please see the transcript for details of the subcommittee’s discussion. Agency Action: The Agency is currently evaluating recommendations made during the afternoon session of the advisory committee meeting.
It is expected that this committee will meet four to six times in FY-20.
Why advice can't be obtained elsewhere?*The committee provides an outside source of scientific expertise in evaluation of clinical trials for oncology drugs. The alternate means of obtaining this advice would be to hire large numbers of scientists on a full time basis at great expense to the government.
Why close or partially close meetings?There were no closed meetings during FY-19.
Recommendation RemarksThis committee was not required any reporting for FY-19.
Hide Section - PERFORMANCE MEASURES

PERFORMANCE MEASURES

Outcome Improvement To Health Or Safety*YesAction Reorganize Priorities*Yes
Outcome Trust In GovernmentYesAction Reallocate ResourcesNo
Outcome Major Policy ChangesYesAction Issued New RegulationsYes
Outcome Advance In Scientific ResearchYesAction Proposed LegislationNo
Outcome Effective Grant MakingNoAction Approved Grants Or Other PaymentsNo
Outcome Improved Service DeliveryNoAction OtherYes
Outcome Increased Customer SatisfactionYesAction CommentFDA approves or chooses not to approve an investigational new medical product.
Outcome Implement Laws/Reg RequirementsYesGrants Review*No
Outcome OtherNoNumber Of Grants Reviewed0
Outcome CommentN/ANumber Of Grants Recommended0
Cost Savings*Unable to DetermineDollar Value Of Grants Recommended$0.00
Cost Savings CommentThe utilization of the Oncologic Drugs Advisory Committee enabled the Agency to obtain required and frequently scarce professional services from medical and scientific experts not otherwise available to the Agency; and to obtain the services of these experts only on an as needed basis rather than on a full time basis. The service of the Committee resulted in advice for the improvement of public health, for which it is difficult to assign a financial value.Grants Review CommentN/A
Number Of Recommendations*160Access Contact Designated Fed. Officer*Yes
Number Of Recommendations CommentThe Committee made 160 recommendations from FY-03 through FY-19. See question 20a of the annual report for specific accomplishments.Access Agency WebsiteYes
% of Recs Fully Implemented*80.00%Access Committee WebsiteYes
% of Recs Fully Implemented CommentThe function of an advisory committee is purely advisory in nature. Although the FDA most often accepts the recommendations from its committees, the advice is purely advisory in nature, and therefore, the Agency has the option of not implementing the advice.Access GSA FACA WebsiteYes
% of Recs Partially Implemented*10.00%Access PublicationsYes
% of Recs Partially Implemented CommentThe function of an advisory committee is purely advisory in nature. Although the FDA most often accepts the recommendations from its committees, the advice is purely advisory in nature, the Agency has the option of not implementing the advice.Access OtherNo
Agency Feedback*YesAccess CommentN/A
Agency Feedback CommentIt usually does. Product approval issues are first released to the sponsor. When appropriate, information is made available to the public. Actions related to guidance documents or other general matters issues are available publicly when implemented.Narrative Description*FDA’s strategic priorities in responding to the public health challenges of the 21st century are to advance regulatory science and innovation; strengthen the safety and integrity of the global supply chain; strengthen compliance and enforcement activities to support public health; expand efforts to meet the needs of special populations; advance medical countermeasures and emergency preparedness; advance food safety and nutrition; promote public health by advancing the safety and effectiveness of medical products; establish an effective tobacco regulation, prevention, and control program; and manage for organizational excellence and accountability. The Oncologic Drugs Advisory Committee supports FDA's strategic priorities by reviewing and evaluating available data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of cancer and makes appropriate recommendations to the Commissioner of Food and Drugs. This supports the development of safe and effective new medical technologies, and advances the status of the Agency as a science-based and science-led regulatory agency, providing global leadership in the protection of public health.
Hide Section - COSTS

COSTS

Payments to Non-Federal Members* Est Payments to Non-Fed Members Next FY* 
Payments to Federal Members* Est. Payments to Fed Members Next FY* 
Payments to Federal Staff* Estimated Payments to Federal Staff* 
Payments to Consultants* Est. Payments to Consultants Next FY* 
Travel Reimb. For Non-Federal Members* Est Travel Reimb Non-Fed Members nextFY* 
Travel Reimb. For Federal Members* Est Travel Reimb For Fed Members* 
Travel Reimb. For Federal Staff* Est. Travel Reimb to Fed Staff Next FY* 
Travel Reimb. For Consultants* Est Travel Reimb to Consultants Next FY* 
Other Costs Est. Other Costs Next FY* 
Total Costs$0.00Est. Total Next FY*$0.00
Federal Staff Support (FTE)* Est. Fed Staff Support Next FY* 
Hide Section - Custom Links

Custom Links

     Committee Level Reports               
Hide Section - MEMBERS,MEETINGS AND ADVISORY REPORTS

MEMBERS,MEETINGS AND ADVISORY REPORTS

To View all the members, meetings and advisory reports for this committee please click here
Hide Section - CHARTERS AND RELATED DOCS

CHARTERS AND RELATED DOCS

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Hide Section - DATA FROM PREVIOUS YEARS

DATA FROM PREVIOUS YEARS

Committee

Data from Previous Years

 
ActionCommittee System IDCommittee NameFiscal Year
 COM-001647Oncologic Drugs Advisory Committee2017
 COM-002242Oncologic Drugs Advisory Committee2016
 COM-004021Oncologic Drugs Advisory Committee2015
 COM-004395Oncologic Drugs Advisory Committee2014
 COM-005893Oncologic Drugs Advisory Committee2013
 COM-006521Oncologic Drugs Advisory Committee2012
 COM-008158Oncologic Drugs Advisory Committee2011
 COM-008680Oncologic Drugs Advisory Committee2010
 COM-010270Oncologic Drugs Advisory Committee2009
 COM-010763Oncologic Drugs Advisory Committee2008
 COM-011887Oncologic Drugs Advisory Committee2007
 COM-012838Oncologic Drugs Advisory Committee2006
 COM-014006Oncologic Drugs Advisory Committee2005
 COM-014528Oncologic Drugs Advisory Committee2004
 COM-016016Oncologic Drugs Advisory Committee2003
 COM-016499Oncologic Drugs Advisory Committee2002
 COM-017946Oncologic Drugs Advisory Committee2001
 COM-018477Oncologic Drugs Advisory Committee2000
 COM-019848Oncologic Drugs Advisory Committee1999
 COM-020439Oncologic Drugs Advisory Committee1998
 COM-021659Oncologic Drugs Advisory Committee1997
 COM-034637Oncologic Drugs Advisory Committee2018