Pre Application Technical Assistance Webinar

Transcript

Hello and Thank you for joining the Pre-Application Technical Assistance Webinar for the Precision Medicine Initiative® (PMI) Cohort Program.   My name is Joni Rutter and in addition to my role as Division Director of Neuroscience and Behavior at the National Institute on Drug Abuse, I am also working with the PMI Cohort Program team to launch these initiatives we will be discussion today.  We have a great PMI Cohort Program Team who are also in the room and will help answer the questions that are submitted.  Some of them will introduce themselves as the webinar proceeds. 

 

… we are hearing some noise on the phone, please mute your phones….

 

We are expected to reach our maximum capacity for people signing on to the webinar, and I just want to take a moment to ask that if there are any people on the webinar who might be able to disconnect and reconnect together with another colleague who is close by, that would be very helpful.  We hope to accommodate as many registrants as possible.  If we do reach capacity, we are recording this session and will be able to provide the materials to those who were unable to join.

On that note, The PMI Program Staff will respond to all questions that are submitted to the webinar, and we also have a dedicated email address for receiving questions related to the funding opportunities:  PMICPFOAInquiries@mail.nih.gov. We will follow-up with written responses to any questions that we are not able to answer within the timeframe of the webinar.  Answers to the Q&A portion of the Webinar, whether or not we were able to answer them during the webinar, will be uploaded to our Frequently Asked Questions page in a couple of weeks: http://www.nih.gov/precision-medicine-initiative-cohort-program/frequently-asked-questions-grants.  We will put the relevant links on the webinar soon so you can note the addresses.

Before I turn it over to Dr. Briggs, I want to let you know that the webinar is now open and ready for you to submit your questions at this time and we can get a head start in answering them now.

Now, I’d like to introduce Dr. Josie Briggs, who is the Interim Director of the PMI Cohort Program.  Many of you may know her in her other capacity as Director of the National Center on Complimentary and Integrative Health.  She will provide a brief background and overview of the PMI Cohort Program to provide some added context to the Q & A session.

Dr. Briggs….  (no transcript)

Thank you Dr. Briggs, we have received a few questions that have come in while Dr. Briggs was talking so we will dive in with answering those questions.

 

The first questions is:

Would creating a Participant Technologies Center research/advisory panel be duplicative with the PMI Steering Committee or Working Groups?

There will be an overarching advisory panel for the PMI Cohort Program. However, applicants may propose additional panels they think are necessary to achieve the goals of the RFA, and the larger PMI Cohort program. In order to minimize potential conflicts during the review process, applicants should outline the type of expertise they would put on such a panel, they should not name or ask specific people to serve on these panels prior to award. Once the awards are made, final decisions about needed advisors and stakeholder representation for the various components will be agreed upon through the steering committee within the governance structure.

The next question relates to an administrative and budget question.  I will ask Teresa Marquette to answer. 

 

I’m Teresa Marquette and I am a grants office at NHLBI helping with the PMI Cohort Program.  The question: Is profit allowed in the budget?

Under the Request for Applications (RFA) announcements, please refer to the NIH Grant Policy Statement (GPS) Section 7.9.1 Selected Items of Cost. Profit and fees are not considered to be "costs" and are not allowable under this program.

 

Does the Indirect cost (Overhead) of the subcontracts need to be included in the Direct Cost Cap?

Applications that include a limitation on direct costs are to exclude from that limit the facilities and administrative (F&A) costs requested by consortium participants.

In addition, applicants should propose their current negotiated F&A rate, as applicable.

The next question has to do with the health provider enrollment centers FOA. 

 

Can the NIH provide further clarification of the enrollment centers scope of work in year 5?

The enrollment centers should continue to enroll participants, and carry out other activities as described in the RFA in year 5. The bullet in Section IV.2.3.B “Plans to continue enrolling and consenting at least 35,000 study participants from diverse populations in each of years 2-4 (enrollment numbers proposed in the application should be well justified) and fostering study participant retention, data collection, and continued engagement in the PMI Cohort Program (see bullets under section “3).A. UG3 Phase” above)” should refer to years 2-5. 

 

The next question  is How extensive is the standard exam and what elements are required?

Once the awards are made, the final decision as to the content of the standard exam for the entire Cohort Program will be agreed upon through the steering committee within the governance structure.  What is the nature of the participant initial physical exam?

• Once the awards are made, the final decision as to the content of the standard exam for the entire Cohort Program will be agreed upon through the steering committee within the governance structure.
• For budgetary purposes, they should propose a health exam they feel is reasonable. 
• Consider reviewing the PMI Working Group report.

 

The next question is Are all surveys, brief exams and biospecimens to be collected at the point of recruitment? 

The core data (survey, standard exam, health records, and biospecimens) for a given participant may be collected at deferred times and scheduled at the convenience of the participant.  However, per The Precision Medicine Initiative Cohort Program – Building a Research Foundation for 21st Century Medicine, p. 24 – “Each of these requirements must be met for each participant in order for them to be considered enrolled in the PMI cohort …”  

 

The next question is related to HPOs Does each enrollment site will have access to their own local dataset for additional ancillary studies?

As outlined on page 34-35 of The Precision Medicine Initiative Cohort Program – Building a Research Foundation for 21st Century Medicine, “A key factor to the success of building the PMI cohort will be effective partnership with HPOs.”  One of the recommendations from the ACD Working Group report is that: “The PMI –Cohort Program should share PMI cohort-generated research data with participating healthcare provider organizations that are providing ongoing data and biospecimens to the PMI cohort, according to participant preferences.”  Details of this framework will need to be evaluated and vetted through the PMI Cohort Program Steering Committee and Governance Structure.

 

The next question relates to multi-sites in the RFA.  For multi-site applications, are individual sites required to combine curated EHR data for transfer or do each site directly interact with the PMI Data Core?

As noted in the RFA, applicants should develop a pipeline to provide a set of structured clinical data from study participants to the Coordinating Center, and should include plans to curate EHR data locally- translate and reformat heterogeneous clinical data to a high quality research resource- before delivery to the Coordinating Center.  The applicant should propose an approach and budget for the approach that they consider most viable for meeting these goals for data transfer from multiple sites.

 

The next question is Will a pediatric population (0-17 years, or any age range within) be recruited as part of the PMI? 

As described in the research strategy, applicants may “Describe how overall enrollment data will be gathered, including for specific populations including families, children, difficult to reach and underserved populations.” For more information, please reference the PMI Working Group Report, and the RFA-PM-16-002 purpose and objectives section, where it states: “Individuals enrolled through HPOs should typically be invited to participate regardless of disease status and should represent all life stages."

 

The next question is related to the Participants Technologies center and I’d like my colleague, Bill Riley to introduce himself for this question.  Thanks Joni.  I’m Bill Riley I’m at the Office of Behavioral and Social Sciences Research and working on the PMI initiative.  The questions is:

Will the Coordinating Center be responsible for providing data access to the Technology Center or is the TC required to budget for data access?

Applicants to RFA-PM-16-003 are encouraged to propose systems architecture, data models and system interactions required to support the functional requirements outlined within the RFA.  The specifics will not be clear until the awards are in place and the steering committee is established.  We expect that these types of decisions will be vetted and finalized through the governance structure.  For the purposes of the cooperative agreement application, applicants may propose scenarios such that the PTC will store the data it collects long enough to provide feedback to the participants, but that if trend data are included/warranted, the CC might be the permanent place for long-term storage and retrieval. Alternatively, the CC may receive data directly with data systems designed to support dynamic interaction with PTC systems. The applicant should propose an approach and budget for the approach that they consider most viable for the coordination of data in collaboration with the CC.

Thank you, Bill.  The next question relates to the biosamples to be collected. Will the PMI cohort program be considering saliva collections in order to examine oral health?

• Final decisions on protocols for biospecimen collection, including the timing and specific specimens to collect will be determined by the PMI Cohort Program Steering Committee and Governance Structure. 
• Note that the NIH has a Request for Information: NIH Precision Medicine Initiative® Cohort Program – National Direct Volunteer Physical Evaluation and Biospecimen Collection (http://grants.nih.gov/grants/guide/notice-files/NOT-PM-16-002.html).
• Please note the deadline for response is January 15^th.  Recommendations coming in through that RFI will be made available for consideration by the PMI Cohort Program Steering Committee and Governance.

 

The next question relates to the biobanks and I will as Rebekah Rasooley to respond.  I’m Rebekah Rasooley, a program officer from the National Institute on Diabetes, Digestive and Kidney Diseases and I’m managing the biobanks project.

The biobank is expected to ship 60,000 kits annually in the implementation phase. If the implementation phase lasts 4 years we will ship 240,000 kits. This is well short of the 1 million participant mark. So we would like to confirm how many participants will be recruited during the implementation phase?

It is expected that the PMI Cohort Program will reach 1 Million volunteers in year 4; however, biospecimen collections may lag after enrollment.  The 60,000 kits may be used to help determine the budget, and is expected to be scalable.  The biobank will be expected to provide kits as needed based on expected recruitment numbers for any given year.  It is expected that the enrollment numbers will be lower in years 1 and 2, increase substantially in years 3-4, then begin to decline. 

The 60,000 number was included in the RFA was included for budgetary purposes.  It is recognized that the actual number of kits might be higher and that will be appropriately be dealt with at the NIH at that point in time.  We wanted to provide a number for budget estimation. 

 

The next question  Since the Biobank is expected to both create aliquots as well as receive bulk frozen shipments from HPOs, we were wondering if the initial centrifugation of the serum and plasma separator tubes is going to be done in the field?

Final considerations and protocols will be developed and finalized through the PMI Cohort Program Steering Committee Governance structure. The plasma and serum separator tubes are intended to be spun at the collection site and then shipped to a laboratory that will be designated for processing. For budgetary purposes, applicants should provide additional plans for centrifugation they feel is reasonable.

                                                                                                                        

Thank you Rebekah.  The next couple of questions refers to the IRB.  Will the PMI central IRB be an existing IRB at one of the HPOs or an existing independent IRB? Or will the PMI central IRB be created specifically for PMI? If the latter, do the enrolling sites have the ability to be part of the central IRB?

The PMI Cohort Program consent and process will be established in the Steering Committee within the overarching Governance Structure.

Per the RFA, the HPO should “Describe how the HPO will plan to enroll at least 10,000 participants in year 1 from diverse populations (enrollment number proposed in the application should be well justified), obtain informed consent, obtain baseline data, arrange for an initial screening exam and acquisition of biospecimens as agreed upon by the Steering Committee.”

For further information on the NIH’s approach to the IRB, please review Section 1 of the Funding Opportunity Description of RFA-PM-16-001, which states “the PMI Cohort Program will have a single Institutional Review Board (IRB), to the extent permitted by law, constituted to ensure prompt and thoughtful consideration of the evolving protocols in the PMI Cohort Program and the central importance of participants as research partners. The PMI Cohort Program IRB will include significant representation by members of the public and representatives of the participant community.”  While recommendations for IRB membership may be solicited from the broad research community including organizations or institutions that are conducting PMI research, the NIH Institutional Official will appoint IRB members.

 

The next question is Does the proscribed Informed consent require a digital signature or a face to face informed consent process?

Under the “Resource Sharing Plan” in the Research Strategy section, it states: “A standardized and centralized electronic consent protocol should be used with all PMI Cohort Program participants to ensure consistency, minimize organizational burden, and maximize participant recruitment.”

 

The next question relates to Do we have a data model in mind for the data transfer from the HPOs to the CC?

The final decision on the data model will depend on who are the competitive applications for the HPOs and CCs.  And, the Participants should propose the model they think is most efficient and appropriate to meet the needs of the program, the final answers will be determined by the awardees in the context of the Steering Committee.

 

The next question relates to the coordinating center.  When do we anticipate the CC being ready to accept data transfers from the HPOs?

• The Coordinating Center is expected to be stood up and ready to accept data as quickly as possible after award. Dr. Briggs showed a timeline indicating around July in the Summer.
• Transfer of data from the HPOs to the Coordinating center will happen in the first year, as an expected milestone during the UG3 phase.

 

The question is The FOA mentions saliva from 10,000 participants in Phase 1, while Dr. Hudson’s slides from the PMI-CP Update she presented on December 10, 2015 (slide 25) describes Phase 1 as “Receive Saliva or Blood.”  Should both saliva and blood be collected in Phase 1? Is the question

While plans may evolve, the current plan for Phase 1 is to collect saliva only from Direct Volunteer participants.  Further information will be through the steering committee and through the governance structure.

 

The next question is For HPO awards with multiple sites, will it be viewed as efficient to send cohort data individually (from each site) or from one hub as a consortium?

• As noted in the RFA, applicants should develop a pipeline to provide a set of structured clinical data from study participants to the Coordinating Center, and should include plans to curate EHR data locally- translate and reformat heterogeneous clinical data to a high quality research resource- before delivery to the Coordinating Center. 
• The applicant should propose an approach and budget for the approach that they consider most viable for meeting these goals for data transfer from multiple sites.

 

The next question relates to procedures for CLIA.  Could you please describe the expectations around this statement in the RFA: “Procedures must meet the applicable requirements of the Clinical Laboratory Improvement Amendments (CLIA), using processes consistent with CLIA for all steps of biospecimen handling and laboratory tests.” 

 The answer to this question is that CLIA requirements apply to clinical laboratories themselves, but none of the CLIA requirements impose guidelines/requirements/processes for collection of biosamples.

 

The next question relates to the biobanks RFA.  Does the biobank RFA’s reference to “processes consistent with CLIA” refer primarily to the need to document chain-of-custody of all biosamples at all time?  Or does that reference refer to other requirements for how the PMI-CP Biobank collects, handles, and stores biosamples?

The RFA reference is primarily to the need to document chain-of-custody of all biosamples at all times.  Thank you for those questions.

 

The next question relates to the healthcare provider organization.  Could you share your views about the role of HPOs in the DV program….since not all of us will (be able to) have formal roles as Enrollment Centers.

• We plan to award up to seven HPOs as formal enrollment centers.
• Recruitment of direct volunteers will be initiated through the pilot program, and then transferred to the Coordinating Center. Anyone in the U.S. will be welcome to volunteer. DV recruitment is not envisioned as a role for the original partner HPOs.

•  As the cohort program continues to evolve, the governance structure may further define the potential role of HPOs that are not among the  original Cohort Program awardees.
•  We also hope that interested researchers become involved in  future research opportunities that use the PMI Cohort Program resources, even if they are not selected as one of the original awardees.

 

Ok.

The next question relates to Who will bear the costs of physical evaluation - the CC or HPOs?

• The HPOs will cover the costs of the physical exam for participants enrolled through the HPOs.  The CC will cover the costs for the direct volunteers.

 

The next question relates to access--What are requirements for Participant access to data and privacy control systems?

• Participant’s access to data, information, and results is a core principle of the Precision Medicine Initiative.
• One of the primary goals of the Participant Technologies Center is to “Develop, test, maintain, and upgrade PMI Cohort Program mobile applications and associated server systems that will provide enrollment, consent, data collection, and communication and feedback functions in a secure environment.
• It is expected that the Participant Technologies Center will propose approaches that securely deliver participant-configurable feedback, both on personal data provided by the participant, and on cohort-wide data as requested.

 

The next question relates to other –omics measurements.  The question is What is the estimated timeline for RFAs for -omics measurement centers? How will these relate to the coordinating center?

• As the PMI cohort program matures, we expect to expand to collect additional measures, this will likely include genomic and other –omic measures.  The exact timeline for starting such activities and relationships within the consortium will be determined as the program evolves. 

 

Ok.  the next question relates to some … application submissions.  Are there any page limits to the research strategy portion of the application?

Yes. The research strategy section of the application is limited to 12 pages. See also: Table of Page Limits outlined within the FOA. The Coordinating Center is a limit of 12 pages within each of the sections.  There is an overall section and each of the core sections.

 

The next question is about diseases in population. Would a project focused on a specific disease population be seen as competitive?

An application that focuses on a specific disease/population will not be responsive to the funding opportunities.  However, applicants may build upon and expand an infrastructure developed for a specific disease/population as long as it is clear in the application that this will not inhibit its generalizability and applicability to the NIH PMI Cohort Program.

 

The next question is about budget for outyears and relationship to the biobanks.  How detailed do budgets for years 2-5 have to be, considering that much of the project work has not been fully developed for these years?

While details of the protocol have not yet been fully developed, the overall goals of the program are described in the RFA and applicants should propose a budget that achieves those goals.

 

Another question relates to the statement to that states “the PMI cohort program biobanks is responsible for receiving, etc.  we have questions about the identification of the biospecimen kits. Will these be unique identifies or will they be assigned by the coordinating center to match the RUIDs ?  and will biospecimens require additional id to be issued.  

The answer is the application should propose a robust tracking system that is linkable to the identifiers preassigned by the CC.  The decision as to whether or not to use an additional identifier will be made  after award by the steering committee.

 

There was a follow up question about protecting research participant confidentiality by means of biospecimen collection kits. Or something else  What meta data is expected by biobanks?

The logistics of shipment remain to be determined after award, so it’s possible  that the biobank will be responsible for sending and receiving kits from individuals. Proposals should specifically address how individual identifiers will be handled in case the biobank is responsible for sending and receiving kits from individual participants.  The biobank is expected to receive data about the collection including time and date, but little other meta data and no clinical or demographic data about the participant.

 

The next question is even though pilot FQHC recruitment will be undertaken, can an UG3/UH3 application include a subcontract to and FQHC network.

The simple answer to that question is yes.

 

A follow up question in application for HPO enrollment sites, can we include FQHCs as part of our network. 

Again, the answer is yes.

 

There is a question related to the cryovials and aliquot tubes.  What sizes will be used?  Is there a brand and catalog number that is specified or will the awarded biobank partner be able to dictate that? 

As noted in the FOA: “The selected vial and storage format will be chosen after award in order to facilitate use of automated biobanking equipment in Phase 2(b)”.  Applicants should propose the specific sizes and brand to be used, but the final decisions on the protocol will be made by the awardee in the context of the overall governance structure of this cooperative agreement. However, vials must conform to the ANSI SLAS 1-2004 (R2012) standard SBS dimension, as stated in the RFA.

Please consult the RFA for those specific details.

 

The next question relates to the coordinating center.  How many volunteers shall we plan for coordinating center being directly responsible for recruiting and managing? 

The coordinating center should provide an enrollment plan with a timeline and plan for the direct volunteers.  It is expected that the CC will enroll about 30-40% for the total enrollment for the cohort program, whereas the HPOs are expected to enroll in the range of 60-70% of the cohort.  This is an aggressive timeline of 1 million or more volunteers enrolled by 2019.  Applicants may want to refer to the recent presentation about the PMI Cohort Program given to the Advisory Council to the Director on December 10^th, and you can view this on the Precision Medicine Website page.

 

The next question relates to the CC for the PMI Cohort Program Center.  Will the NIH allow a commercial entity be a subcontractor to the CC? 

A commercial entity as a subcontractor is allowable.

 

The next question relates to biosamples within the HPOs.  Will the biosamples be sent directly to the central lab, the biobank, or is preprocessing at the HPO site required? 

The collection kits will be provided by the PMI biobank and the HPO will process the biospecimens to create aliquots of plasma, serum, and other biosamples and send them to the PMI designated central biobank.  The biobank will then be responsible for all other sample processing.  Again, as the PMI cohort program evolves, the steering committee may make some changes, but this is how it’s envisioned so far.

Inaudible.

 

The next question relates to biospecimens. During enrollment, will a “new” biospecimen be required?

As stated in the ACD working group report and the RFAs, new biospecimens are required for full enrollment into the PMI Cohort Program.  A follow on question to that is will blood samples be required for everyone?  The answer to that is that while biospecimens are required, and as stated in the objectives of the PMI cohort program, all volunteers will be asked at entry for consent to join the Cohort Program and to be contacted for future studies. They will be asked to complete a brief survey and to undertake a standard exam, to share their healthcare records, and to be willing to submit biospecimens, including blood, urine, and saliva. So approaches to those initial biospecimens are required. 

 

The next question relates to incentives.  Will any incentive be allowed in order to increase enrollment?

The answer to this is that Incentives may be proposed, and will need to be part of the protocol that will require IRB approval and through the governance structure moving forward.

 

The next question relates to storage of medical data.  Are the awardees expected to store the medical data from their patients in a central data warehouse environment?

Per the RFA, in the UG3 phase the HPOs should work collaboratively “with the CC, other HPOs, the participant technologies center, and the PMI Cohort Program Biobank to develop and pilot procedures, including recruitment and enrollment of study participants.” Mobile technologies will be the responsibility of RFA-PM-16-003 and their use within the PMI Cohort Program will be determined by the Steering Committee.

The HPOs should curate data locally. They should also “provide a set of structured clinical data from study participants to the coordinating center at least quarterly.” In the UH3 phase they should streamline “the pipeline that sends data to the Coordinating Center data management system to enable data from the supported HPOs to be jointly used for data analysis.”

 

We are noticing that a number of these questions are asking about how specific responsibilities will be handled.  We want to note that all four of these FOAs are cooperative agreements or “U awards” e.g., that as a cooperative agreement, the network awardees are expected to work closely with each other and with the NIH to achieve the goals of PMI-Cohort Program.  All aspects of the program protocols will require input of the steering committee and overall governance.  Applicants should propose approaches and protocols they feel are most appropriate, recognizing that specific details will be negotiated as the start of the project.   

Dr. Briggs, is there anything you would like to add?  No, the questions have been excellent.  Everyone understands that this is a process that will evolve very cooperatively with the awardees and the NIH leadership and with active involvement of the participants. We are aiming for a pretty rapid start, a pretty simple protocol, and over time, the addition of more complex analyses and data collection tools.  So the initial set of information from the HPO will probably not include a great detail of legacy of health information, but start with a core data set for example.  The questions have been terrific. This is complex.  We are building this as you are thinking of these issues as you are working on these applications.  WE expect to learn a great deal together.

Thank you Dr. Briggs.

One of the question is relates to the enrollees.  Question: Are enrollees expected to be “new”, and not any existing enrolled participants from the applicant’s organization?

To reiterate, enrollees may come from participants who are already enrolled in research studies conducted by the HPO – but each participant must be specifically enrolled into the PMI Cohort Program (they cannot be brought in other under consents)

Specimens required for the PMI Cohort Program will be newly collected – the Program will not accept specimens collected for other purposes.

 

Another question relates to the HPO enrollment centers.  Is the HPO Enrollment Center comprised of a single healthcare provider or can the structure be that of a consortium of healthcare provider organizations?

A consortium of HPOs may be proposed, and there are no limits to the number of HPOs within the proposed consortium to come in as an application. 

 

The next question relates to multiple PIs.  Can we have co-PIs from different institutions, and if allowed, should we submit a Leadership Plan?

The answer to that is that multiple PI/PDs are allowed, and a leadership plan must be submitted.  For institutions/organizations proposing multiple PDs/PIs, visit the Multiple Program Director/Principal Investigator Policy and submission details in the Senior/Key Person Profile (Expanded) Component of the SF424 (R&R) Application Guide.  There are clear instructions there.

Teresa, anything to add?

No.

Again, no restriction of sites for healthcare provider organizations.

 

Next question relates to foreign components.  Question:  Are we allowed to propose/include activities that would be carried out by off-shore/overseas components of organizations whom we hire as consultants? Or must all of the work we do as part of the PMI-CP team occur here in the USA and be carried out by persons who are in the USA at the time the work is performed?  

Answer: As stated in the RFAs, foreign components are not allowed.  Per the Foreign component definition in the NIH Grants Policy Statement, “use of facilities or instrumentation at a foreign site” is within the grant-related activities that may be significant.  At the time of implementation of award, the functional activity described in your question would raise programmatic concerns.

 

The next question relates to HPOs, Question: Should we assume that patients who leave the HPO’s system may be followed through some kind of PMI Cohort Program remote or mobile application to submit their data?

Answer is that those who leave the HPO system through which they enroll will be encouraged to remain as participants in PMI-Cohort Program, by converting to Direct Volunteers.

 

The next question is Will direct volunteers be able to use any local healthcare facility to submit biosamples, assuming that the DV is not living near an HPO? If so, how will the facility be paid for obtaining the sample?

We are currently evaluating options on how to collect biosamples from direct volunteers, not included in biobank. This is the subject of the current RFI that is due tomorrow and which may be accessed through our Website.  We hope to have a plan that will work for interested DVs, no matter where they live.

Currently the CC will be responsible for providing funding for direct volunteer exams and biospecimen collection.

 

Another question relates to the number of PI sites. 

Again, no restrictions on number of sites, and multiple PIs are permitted.

Right now we are caught up, inaudible. 

Pause

 

For the next question, we are going to show a slide.  What is estimated total award value, direct plus indirect.  So each of the FOAs specifies a maximum of the direct costs, per year in the award budget section under section II award information in the RFA.  Applicants must use their negotiated rates for indirect costs, and you can also refer to the slide that we are now putting on the webinar.

The next question relates to Question: What is the PTC expected to be developing from year 1 to year 5?

We expect that core Participant Technologies Center software development efforts will occur in the first year or two of the award.  Ongoing system maintenance and additional development activities are likely throughout the award as new technologies are developed or integrated into the PMI Cohort Program.

Dr. Riley, do you have anything to add to that? 

That’s exactly right.  We expect that most of this work will happen in the early stages.  There will be a fair amount of maintenance and upgrades that will have to happen along the way.  Applicants will need to be aware that new technologies will become available and placed online in PMI over time so there will be additional development efforts as those come along. 

Thank you, Dr. Riley.

 

Will the PTC be housing the data or is it the responsibility of the CC?

Applicants to RFA-PM-16-003 are encouraged to propose systems architecture, data models and system interactions required to support the functional requirements outlined within the RFA.  The specifics will not be clear until the awards are in place and the steering committee is established.  We expect that these types of decisions will be vetted and finalized through the governance structure.  For the purposes of the cooperative agreement application, applicants may propose scenarios such that the PTC will store the data it collects long enough to provide feedback to the participants, but that if trend data are included/warranted, the CC might be the permanent place for long-term storage and retrieval. Alternatively, the CC may receive data directly with data systems designed to support dynamic interaction with PTC systems. The applicant should propose an approach and budget for the approach that they consider most viable for the coordination of data in collaboration with the CC.

Dr. Riley or Dr. McClain, anything to add?  OK.  Thanks

 

The next question is Question:  What is the Role of pilot data of the direct volunteer program for the PTC functioning?

Answer:  We expect that the data collected from the direct volunteers pilot studies (DV) pilot will be transferred to the Coordinating Center.  We also expect that the findings from the DV pilot will be utilized by the PTC in developing the web and mobile technologies.  We anticipate that the DV pilot awardee will also share with the PTC the relevant software code that could be of use to the PTC, but we cannot require the DV pilot awardee to transfer any proprietary code or platforms to the PTC.  

The next question is where can we get a transcript of the statements made during the Webinar?  So this webinar will be archived and captioned on our website. Key questions  will be added to our FAQs website.  Any questions that we do not cover, we will be covering on the main PMI cohort program webpage.

 

Another question about the budget, we discussed this question earlier; we discussed this earlier but another question came in, so to reiterate:

The question is: Do the HPO enrollment sites need to budget for the collection of biospecimens? It appears that the biobanking core will provide kits, so presumably we do not budget for this cost in the HPO enrollment sites.

The answer is: The PMI Cohort Program Biobank will supply the vials and pay for shipping.  Please review RFA-PM-16-004 for further details.

 

Although we’ve answered this question before as well, it’s asked a little differently, so I want to make sure it’s clear.  The question is does study enrollment include agreeing to join, completing the baseline exam, providing a biospecimen, or doing some combination?

So I wanted to reiterate, in order to be a part of the PMI Cohort Prgoram, all volunteers must consent to join the PMI cohort program and be recontacted for future studies. They must provide a biospecimen, complete a baseline exam, and complete an enrollment survery.  If they have an electronic health record data, they must agree to share it, however electronic health record data is not required at this time to be a direct volunteer.  HPOs however, must share core data elements from electronic health record to the PMI cohort for each enrolled participant.  This was outlined clearly in the ACD PMI working group report The Precision Medicine Initiative Cohort Program – Building a Research Foundation for 21st Century Medicine, p. 24 – “Each of these requirements must be met for each participant in order for them to be considered enrolled in the PMI cohort …”  

 

We have about 20 minutes left for the webinar.  Please continue to send in your questions if you have them.

The next question is What is the anticipated frequency of follow-up visits for cohort participants?

The answer is that each HPO applicant should propose its own plans for follow-up, including the how and the frequency of the tracking of its participants’ health and clinical outcomes

A final decision on whether follow up clinical examinations will be implemented under the current funding period will be determined by the governance structure.

A bit of a lull .. anything that the group would like to add?  Keep your questions coming, and we will hang out until we see the next question come in.

Pause

Pause

Ok thanks for bearing with us, we do have a few questions that came in.  one is are the enrollment centers responsible for pushing the electronic health record data that reside at the CC.  The answer is yes, the enrollment centers will be responsible for pushing the electronic health record data.  The specifics of the these requirements for standards and content are within the FOA, so please consult the FOA specifically.

 

The next question:  Do you envision the up to 7 recruitment centers to be chosen on a regional basis?...that is, will geographic and demographic distribution be considered as part of review and award?

The answer to this is No, the HPOs will not be chosen on a regional basis, but the objective of the Cohort Program is to broadly represent the diversity of the US, including geographic diversity.  Please see the RFAs for review criteria.

And, the HPO should explain what demographic diversity they believe they can achieve in their area.

 

The next question is on consents. Are the HPOs responsible for developing their own consent forms?  Or will there be a common form for year 1?

The answer to this is No, the HPOs will not be responsible for developing their own consent forms. There will be a common set.  The PMI Cohort Program consent and process will be established in the Steering Committee within the overarching Governance Structure.

Per the RFA, the HPO should “Describe how the HPO will plan to enroll at least 10,000 participants in year 1 from diverse populations (enrollment number proposed in the application should be well justified), obtain informed consent, obtain baseline data, arrange for an initial screening exam and acquisition of biospecimens as agreed upon by the Steering Committee.”

Again, this will be established after the award.

 

The next question relates to partnerships with other Territories.  With respect to the response to the research conducted overseas.  Puerto Rico is a Territory of the US, but is not a state and not part of the mainland.  Can the project propose to recruit participants from Puerto Rico?

The answer to this is Yes.  Puerto  Rico and US Territories are allowed.

Thank you for that question.

 

The next question relates to the biobank.  Will the biobank address the saliva kits to be sent to individual participants?  Will the Biobank have this PHI?

The logistics of shipment remain to be determined. Please keep that in mind.

Will this PHI data be retained?

Proposals should specifically address how individual identifiers will be handled in case the Biobank is responsible for distributing and receiving kits from individual participants. 

 

The next question is does the Does the PMI have  a specific timeframe when Phase 2(b) should begin and when the Biobank should have the automated storage/retrieval systems completed?

The answer to this is that It is anticipated that automated storage/retrieval systems will be operational by the end of year 2 of the Biobank.  Applicants should provide milestones as needed for the transition to Phase 2(b)

Bio banking kits will be sent to enrollment centers for all ascertainments. Yet, the bio bank RFA mentions only 60,000 kits per year total for ALL centers.

For the purpose of budgeting, please use the numbers provided in the RFA.  The exact numbers and procedures will be determined after award by the PMI Steering Committee and appropriate budgetary adjustments will be made, if necessary.

 

If a biobank is currently performing all aspects of the consortium on a routine basis, would we have to submit separate grants for each aspect of the corsortium or could we roll it all into the biobank FOA?

Biobank applicants should submit a single proposal addressing all aspects of the work outlined in the Biobank RFA.

Do you have something to add, Rebekah?  No.  Thanks.

We have a bit of a lull, as before, I’ll give it a few minutes to see of anything surfaces to top.

Pause

Pause

OK thank you for waiting for just a moment.  We had another question come in.  To what extent or scientific rationale is needed for targeting types … sorry… To what extent or scientific rationale is needed for targeted types or distributions of patients?  For this question, applicants should provide a scientific rationale for any targeted recruitment based on disease and patient recruitment.

Standing by…

Ok.  thank you for holding.  We have a little less than 10 minutes to go and we have a few more questions that have come in and we’d like to address.

I’m going to turn it over to Teresa Marquette.  HI we received a question of will capability in existing *inaudible*  to allow multi users in one application.  The answer is no, it will not.

Thank you Teresa.

 

The next question is Do the HPOs obtain local IRB approval for enrollment in Year 1 until the PMI Cohort Program Steering Committee and Central IRB approves the program-wide consent process? Or do we need to wait for the central consent process to be implemented before we can begin enrolling?

The answer to this is that The PMI Cohort Program is forming a central IRB and will establish a consortia-wide consent process.

 

The next question is are the HPOs allowed to propose a budget higher than the budget guidelines?

As is typical, the answer is no, there is a cap on the RFA and that cap must be honored.

We are slowing down a little, so I will give it a little more time here

OK there was a question about enrolling families, and I’d like to turn this over to Dr. Briggs. 

We encourage in the application for you to tell us how you would propose to include families.  We believe family enrollment is going to be an important strategy to meet the overall goal of age diversity in the program.  This is a very valid issue for you to address in your application.

Thank you.

Give it a few more minutes for additional questions to roll in.

Pause

 

We have a question of whether applicants should be prepared to enroll from day 1.  It is our intent for applicants to begin to enroll as rapidly as possible after award. Realistically, we are anticipating a month or two for finalizing the protocol issues that have come up during our call and during the discussion of the PMI Program, but applicants should be prepared to begin enrollment within a couple of months after the award date.

 

We have about 3 minutes remaining.  A few questions to go.  One question is Why is saliva proposed for phase 1 of the U24 biobank, but not phase 2?

Saliva is being proposed for phase 1 to simplify the initial phase of PMI. The final decision about which samples will be collected during phase 2 will be made by the PMI CP Steering Committee.  It is possible that saliva will be included in the collection from phase 2.

For the purposes of the proposal, Biobank applicants should address the current plan to collect only blood and urine.

 

we are getting a few questions from individual asking "How to volunteer for the PMI Cohort Program?"

This is great!

The Cohort Program will begin taking volunteers later in 2016. As soon as we’re ready for people to volunteer, we’ll have a communication campaign to let people all over the country know that the time has come to join and how they can join. In the meantime, if you sign up to receive e-mail updates on the National Institutes of Health (NIH) PMI Web site, you’ll receive an announcement when we are ready for volunteers to join. To sign up for PMI e-mail updates, look for the box at the upper right hand side of the Web page at http://www.nih.gov/precisionmedicine.

Thank you for that question.

 

Another questions is that Since the PMI Cohort Program Biobank is a separate effort from the volunteer recruitment effort, we assume that the “planned enrollment report table” and “inclusion of women and children” sections of the grant application for the PMI Cohort Program Biobank should be marked as “not applicable”. Can you please confirm that this approach is acceptable?

Yes, we confirm that the approach is acceptable.  You are Correct.

 

The next question is Should we assume that blood and saliva samples will be mailed daily to the central bio-bank and thus not require local storage or should we budget costs to store blood locally at the HPO (for example if we are expected to store blood and mail samples monthly and not daily).

The final protocol will be determined by the PMI Cohort Program Steering Committee.  It is possible that HPOs will process and store samples locally for a period of time and send bulk shipments.  Applicants should address this possibility in the application.

 

And Just to clarify...will the HPOs cover the costs of the baseline physical exams and then any proposed follow-up exams?

Again, the answer to this is yes.  Thanks for that clarification.  HPOs will cover the costs of the baseline physical exams and then any proposed follow-ups.

 

We are nearing the end of our time for this webinar.  I think we have one more question. It’s a three-fer.  The first part of this question is the PMI CC center responsible for identifying the enrollment and the blood processing sites for the direct volunteers.  And the answer to this question is that there is an RFI due tomorrow—an RFI—request for information, is due tomorrow to provide input.  The CC will be responsible for coordinating recruitment and biospecimen collection.

The next question is what is the extent of the assessment required in person?  For example the physical exam?  Physical exam will be determined by the steering committee.

What about the electronic health data for direct volunteer?  Direct Volunteer will provide electornic health record data to the extent possible.  We hope to use a process like “blue button” and other technologies to help facilitate transfer of electronic health data from direct volunteers.

 

We will end on that note. We’d like to thank the registrants.  Your question s have bene fantastic and we are excited about the prospects for these applications. We’d also like to thank the people in this room, especially Dr. Briggs for leading this effort.  And the staff have been working very diligenetly to help answer these question to help you make the applications the best that they possibly can be.  Please continue to send in your question to the PMI inquities inbox, and we will continue to answer your question.  Thank you again, and Good luck.