International MPN News, Science & Opinion

FDA and the MPN Patient Advocate Program

The MPN Patient Advocate and the FDA Opinion

Background

Earlier this year a group of MPN patients and caregivers through the Zebra Coalition and MPNforum Magazine started discussions of the potential value of a Patient Advocate (PA) at each MPN clinical trial site.

The idea was to field a PA to perform various communications and support functions, to act as liaison between the trial Principal Investigator and the enrolled patients. The MPN Patient Advocate would be available to all patients enrolled in the trial on a 24/7 basis and have access to the Principal Investigator and staff, the local Institutional Review Board and the Sponsor.

In this position, the Patient Advocate could help address patient concerns, monitor safety issues and patient compliance and contribute to the fulfillment of the clinical trial protocol.

The FDA and the MPN Patient Advocate

The FDA Commissioner was petitioned to convene a meeting to discuss the best way to proceed in exploring a Patient Advocate option and help determine how such an option might fit into the current clinical trial system

April, 2014 nine FDA executives participated in the meeting

Appearing for the FDA: Center for Drug Evaluation and Research:

Robert Temple, M.D., Deputy Director for Clinical Science

Richard Pazdur, M.D., Director, Office of Hematology and Oncology Drug Products (OHOP)

Ann Farrell, M.D., Division Director, Division of Hematology Products, OHOP

Stephanie Shapley, Health Science Policy Analyst, Office of Medical Policy Patrick Archdeacon, Senior Clinical Advisor, Office of Medical Policy Rose Cunningham, Regulatory Health Project Manager, Office of Executive Programs Office of Health and Constituent Affairs, Office of the Commissioner: Richard M. Klein, Director, Patient Liaison Program Office of Good Clinical Practice, Office of the Commissioner Sara Goldkind, M.D., M.A., Senior Bioethicist Joanne R. Less, Ph.D., Director, Office Good Clinical Practice.

The tone was exploratory and practical, defining the role of the Patient Advocate with FDA participants contributing concrete suggestions on means to implement the program.

.FDA Meeting Notes, prepared by Rose Cunningham, Office of the Commissioner

Purpose of the meeting:  MPNforum/ZebraCoalition wanted to explore the possibility of having a patient advocate involved with MPN trials to liaise between patients, investigators, sponsors, and IRBs, as necessary to improve communication (specifically information related to adverse events) between the four parties.

[Primary conclusion]

  • There is no FDA regulatory barrier to this sort of program, if a sponsor wished to designate a patient advocate as a contact. The consent regulations require that there be “an explanation of whom to contact for answers to pertinent questions about the research and research subjects’ rights, and whom to contact in the event of a research-related injury to the subject.” 50.25(a)(7). There doesn’t appear to be any reason that a patient advocate couldn’t be one of the designated contacts. The challenge for the coalition will be at the sponsor level, rather than at FDA. But the idea could be a selling point for studies, and sponsors might be quite open to the concept.[A copy of the full notes available on request]There are clinical trial mandated safeguards in place: patient examination by the Principal Investigator and medical staff; an Institutional Review Board at the site level, a Data Monitoring Committee at the higher full trial level. The lack of a PA has long been felt by MPN patients both those considering entering a clinical trial and those on clinical trial to help get their questions and concerns answered.   

 

  • The MPN Patient Advocate initiative is designed to fill an information and security gap and help make the clinical trial system, on which we all rely, more secure.
  • What is lacking is a patient advocate, a fellow MPN patient or caregiver, communicating on behalf of patients with clinical trial managers. A patient advocate, easily available to enrolled trial subjects, can enhance compliance and retention while serving as a conduit for patient concerns.

 

  • Conclusion:  There are clinical trial mandated safeguards in place: patient examination by the Principal Investigator and medical staff; an Institutional Review Board at the site level, a Data Monitoring Committee at the higher full trial level.What is lacking is a patient advocate, a fellow MPN patient or caregiver, communicating on behalf of patients with clinical trial managers.  A patient advocate, easily available to enrolled trial subjects, can enhance compliance and retention while serving as a conduit for patient concerns.

    The lack of a PA has long been felt by MPN patients both those considering entering a clinical trial and those on clinical trial to help get their questions and concerns answered.

    The MPN Patient Advocate initiative is designed to fill an information and security gap and help make the clinical trial system, on which we all rely, more secure.

    line

Final Draft Notes — FDA

FDA and Zebra Coalition/MPNforum

April 3, 2014

Attendees:

MPNforum/Zebra Coalition:

Zhenya Senyak, Editor and Publisher, MPNforum Magazine

Jeremy Smith, Board Member, MPNforum Magazine, coordinator for governmental relations

Center for Drug Evaluation and Research:

Robert Temple, M.D., Deputy Director for Clinical Science

Richard Pazdur, M.D., Director, Office of Hematology and Oncology Drug Products (OHOP)

Ann Farrell, M.D., Division Director, Division of Hematology Products, OHOP

Stephanie Shapley, Health Science Policy Analyst, Office of Medical Policy

Patrick Archdeacon, M.D., Senior Clinical Advisor, Office of Medical Policy

Rose Cunningham, Regulatory Health Project Manager, Office of Executive Programs

 

Office of Health and Constituent Affairs, Office of the Commissioner:

Richard M. Klein, Director, Patient Liaison Program

 

Office of Good Clinical Practice, Office of the Commissioner

Sara Goldkind, M.D., M.A., Senior Bioethicist

Joanne R. Less, Ph.D., Director, Office Good Clinical Practice

Agenda:          To discuss with FDA the best way to proceed in exploring a Patient Advocate option, how such an option might fit into the current clinical trial system and what steps MPNforum/ZebraCoalition might consider taking   

  • Introductions were made and the meeting was turned over to Zhenya Senyak to explain his concerns about patient advocacy and representation in clinical trials.
  • MPNforum/ZebraCoalition wanted to explore the possibility of having a patient advocate involved with MPN trials to liaise between patients, investigators, sponsors, and IRBs, as necessary to improve communication (specifically information related to adverse events) between the four parties.

 

  • MPNforum/ZebraCoalition described their reason for pursuing the addition of a patient advocate to the clinical trial model is to prevent a situation Mr. Senyak described where patients felt that adverse event information was not conveyed to individuals enrolled in a clinical trial in a timely manner.

 

  • FDA asked about the role, responsibilities, and qualifications of a subject advocate[1]. Asked about who would be responsible for supporting the advocate role, the coalition responded that the sponsor should support the position because the subject advocate function could aid in study recruitment and help with compliance.

 

  • The subject advocates could be the voice of the patient, but would not give medical advice. Their role would be to improve communication.

 

  • The coalition believes that if there were someone – perhaps a fellow patient in the trial trained as a subject advocate – that was accessible to all patients, communications regarding adverse events could be improved. The advocate would have a process for following up with the investigator, sponsor, and/or IRB as necessary regarding any adverse event related issues patients feel they are unable to resolve with the investigator.

 

  • One potential downside of the proposal mentioned is that patients may contact the patient advocate about adverse events rather than the sponsor/investigator, which could potentially delay access to treatment.

 

  • Questions were raised about what qualifications a designated subject advocate might require to fulfill this role. Training in FDA regulatory requirements, training in confidentiality concerns and protections and being a member of the patient community were suggested.

 

  • There was discussion of current protections for patients in clinical trials including:
    • Informed consent regulations- 21 CFR part 50 (e.g., 50.25(a)(7) requires consent forms to include contacts for questions about the research and subjects’ rights and for research-related injury)
    • IRB regulations- 21 CFR part 56
    • Regulations for investigators- 21 CFR 312.64 requires investigators to immediately report serious adverse events to sponsors
    • Regulations for sponsors- 21 CFR 312.32 requires sponsors to notify FDA and investigators of important safety information in an expedited fashion

 

  • Although the coalition was discussing just their disease (blood cancers) this model could be used for other diseases.

 

  • MPNforum/ZebraCoalition asked if FDA was aware of any regulations or guidance that would prevent such a model and asked FDA if there are any reasons that the coalition should not contact various drug companies developing clinical trials to see if sponsors would support this. FDA provided some background on existing protections for patients in trials and did not have concerns with MPNforum/ZebraCoalition reaching out to drug companies to discuss this model.

 

  • There is no FDA regulatory barrier to this sort of program, if a sponsor wished to designate a patient advocate as a contact. The consent regulations require that there be “an explanation of whom to contact for answers to pertinent questions about the research and research subjects’ rights, and whom to contact in the event of a research-related injury to the subject.”       50.25(a)(7). There doesn’t appear to be any reason that a patient advocate couldn’t be one of the designated contacts. The challenge for the coalition will be at the sponsor level, rather than at FDA.       But the idea could be a selling point for studies, and sponsors might be quite open to the concept.

 

  • They will be contacting sponsors to work toward establishing patient advocates at clinical trial sites.

 

  • The coalition asked if there was any further advice FDA could give.

 

  • FDA’s Office of Health and Constituent Affairs would be willing to work with the coalition. Please see the following links on the FDA webpage for further information:

 

Patient Representation:

http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/default.htm

 

IRB Reg:

http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/cfrsearch.cfm?cfrpart=56&showfr=1

 

 

ACTION ITEMS:

 

  • Send list of attendees and their titles to the participants (Completed 4/4/14.)

 

[1] Although MPNforum/Zebra Coalition representatives used the term “patient” advocate, the term “subject” advocate would be more accurate.

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