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Webinar: Final Guidance on Patient Preference Information

Moderator: Irene Aihie

September 27, 2016

1:00 pm ET

Coordinator: Good afternoon and thank you for standing by, and welcome to today's 

webinar. All lines are in a listen only mode until the question and answer 

segment of today's conference. Today, we will be taking questions from the 

phone lines only. At that time, you may press star one to ask a question.

Today's conference is being recorded. If you have any objections you may 

disconnect at this time. I would now like to turn today's conference over to 

Ms. Irene Aihie. Thank you. You may begin.

Irene Aihie: Thank you. Hello, and welcome to today's FDA webinar. I am Irene Aihie of 

CDRH's Office of Communication and Education. On August 23, 2016, the 

U.S. Food and Drug Administration published the final guidance document, 

patient preference information, voluntary submission, review, and PMAs, 

HDE application, and de novo requests, and inclusion in decision summaries 

in device labeling, which outlines recommendations on patient preference 

studies that may result in valid scientific evidence and how stakeholders, 

including industry and patient advocacy organizations, can voluntarily collect 

and submit through FDA patient preference information.

The focus of today's webinar is to share the information and answer questions 

about the final guidance document. Today's presenters are Annie Saha, 
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Director of External Expertise and Partnerships in the Office of the Center 

Director and Martin Ho, Associate Director for Quantitative Innovation in the 

Office of Surveillance and Biometrics here in CDRH. Following the 

presentation, we will open the line for your questions related to topic in the 

final guidance only.

Additionally, there are other subject matter experts available to assist in the 

Q&A portion of our webinar. Now, I give you Annie.

Annie Saha: Thank you, Irene, and good afternoon everyone. For sake of brevity, I will 

refer to the guidance as just the patient preference guidance and not the full 

title for the rest of the presentation. And the agenda for this webinar is really 

to provide some context and overview of the patient preference information 

final guidance as well as describing some key updates to the benefit risk 

worksheet in the factors to consider when making benefit risk determinations 

in medical device premarket approval and de novo classifications guidance 

that was originally issued in 2012. And then as Irene noted, we will follow-up 

with question and answers.

So a bit of context and scope as to really how we get where we are in terms of 

the patient preference initiative and the guidance development. We're really 

seeing in the larger healthcare ecosystem the evolution of the role of the 

patient where in the traditional medicine sort of field where the provider has 

really led the treatment decision making, patients follow the instructions of 

their providers, into the late '80s when -- into the '90s when HIV/AIDS, cancer 

patient advocacy groups really banded together to ask for access and 

availability to new treatments.

And then with the internet, patients really became empowered through all the 

information that was then available to them and in the future, which is where 
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we are today, is that patients and providers are really in a partnership where 

they're really able to work together and determine what's the best treatment 

decision and make that decision for themselves. And patient preferences can 

really help us inform the regulatory decision making and that role has evolved 

internally as well.

So we issued -- this really started -- our first stake in the ground was in 2012 

with our updated guidance. Now, in 2016 where we really stake out what are 

the factors that should be considered when making a benefit risk assessment 

and that one factor that is key and of importance is really what patient's 

perspectives are on both the risks and their perspective on benefit, 

understanding that different patients are going to have different risk tolerances

and that's going to vary, and it may reveal that different participants are 

willing to tolerate a different level of risk to achieve a probable benefit, and 

that patients are the ones who are uniquely qualified to give us that 

information and help us balance our benefit risk assessments with that 

information, along with other clinical and non-clinical data.

This led to our strategic priority for 2016-2017 where we really recognized 

that patients -- we really need to partner with patients and we really must 

interact with them as partners to really advance development and evaluation 

of innovative medical devices. And this strategic priority really has two 

prongs. One is to promote a culture of meaningful patient engagement by 

facilitating interaction between our staff and patients, and then increasing the 

use of transparency of that patient input as evidence in our decision making 

processes. And that's really where the focus of the patient preference guidance 

and this discussion will be about.

Overall, we see that patient information could really come to us in a variety of 

ways and patient input is really that broad umbrella of the types of 
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information that FDA receives in terms of the perspectives of patients, 

anything from anecdotal comments and correspondences to FDA, testimonies 

at advisory committee meetings, opinions that are expressed via social media, 

as well as surveys and even -- and then the more quantitative measurements of 

patient reported outcomes, patient preferences, et cetera. And then a subset of 

that information is really patient perspectives, which is really a type of patient 

input and that's really information that's really relating to the patient's 

experience with a disease or condition and how they manage that condition.

And this is really useful for both understanding the disease and having a better 

understanding of that impact on patients, identifying what outcomes are most 

important to patients, and then really understanding what are those benefit risk 

tradeoffs for treatment that are important to patients. So patient input is kind 

of the house if you will and then the two-car garage, patient perspective, is 

both your patient preference information and then your patient reported 

outcomes.

So what is patient preference information? So for the purposes of this 

guidance, we refer to patient preference information as the qualitative or 

quantitative assessments of the relative desirability or accessibility to patients 

as specified alternatives or choices among outcomes or other attributes that 

differ among alternative health interventions. And it's also important to note 

that when we use the term patients, we are -- would also include ideas like --

include care partners, such as parents who are in the pediatric population or 

potentially the children of elderly patients maybe who have dementia, et 

cetera, and healthcare professional preferences could also be taken into 

account.

So what can patient preference information provide and how can it be used?

So the patient preferences can really provide us with valuable information
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about what are the benefits and the risks that are most important to the 

effective patient population, what are the benefit risk tradeoffs that are 

acceptable from a patient perspective. So what benefits are patients willing to 

take in exchange for certain risks and how do patients really think about those 

tradeoffs? And then are there relevant subgroups of patients that would really 

accept a particular benefit risk profile or choose a potential treatment option 

over other alternatives that exist.

Various potential uses for patient preference information could include 

informing both end points for (effect) size, for clinical studies, as well as 

informing subgroup considerations. Patient preference information could 

ultimately also impact the labelling changes or expanded indications. Areas 

where patient preference information could be used outside a regulatory 

context could certainly include areas such as shared medical decision making.

What's the difference between patient preference information and patient 

reported outcomes? And this is a question we get quite a bit so we want to try 

to explain what -- the differences there are. So patient reported outcomes is 

really a report of the status of a patient's health condition that comes directly 

from the patient without any interpretation from -- of the patient's response by 

a clinician or anyone else. And these instruments are really designed to 

measure a patient's perceptions of health status before, during, or after 

therapy.

So for example, a patient reported outcome might be related to pain and so 

clinicians will ask patients for how they're feeling both before treatment, 

during the treatment, then after a therapy whereas patient preference 

information, patient preference studies really study what specified type of 

therapy or attributes of a given product a patient might prefer. So if we 

continue with the pain example, it might be what is the level of pain you're 
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willing -- that you would like to be reduced for you to accept a certain risk. So 

it's about that trade off and about what those different attributes are versus the 

health state.

Now, we'll go into an overview of the guidance itself and kind of highlight the 

objectives, the scope, and the various sections of the guidance. So the overall 

objectives of the guidance are to encourage submission of patient preference 

information, if available, whether by sponsors or other stakeholders to submit 

that information to FDA to aid in our regulatory decision making. We then go 

into outlining the recommended qualities of those patient preference studies, 

which then may be result in valid scientific evidence, and then to provide 

recommendations for collecting and submitting that information, as well as 

discussing how patient preference information will be included in our decision 

summaries and provide recommendations for the inclusion of that information 

in labeling.

The scope, really, is explaining the concepts of the guidance that sponsors or 

other stakeholders should consider when choosing to collect patient 

information, which may effect and inform our benefit risk determinations for 

pre-market review of PMA devices, HDEs, as well as de novo requests, as 

well as discussing the inclusion in terms of how we'd be transparent about the 

use of that information.

I want to be clear, though, patient preference information submission to FDA 

is voluntary and there are certainly cases in device areas where patient 

preference information may not be relevant or appropriate. Certainly, we see 

that it's useful for sponsors to essentially collect or submit this information 

when the usage decisions by patients and the healthcare professionals are 

preference sensitive. So the idea there being that there might not be a gold 

standard of treatment or there's variable evidence about the different benefits 
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and risks of different types of treatment that exist. Or there's different --

there's heterogeneity within the patient population and different patients are 

going to have different decisions about which type of device, or drug, or, you 

know, whether to have surgery for treatment.

So really the devices that could benefit from patient preference information 

are those with characteristics like a direct patient interface, devices that could 

yield significant health or appearance benefits that are directly related to 

quality of life, certain lifesaving but high risk devices, devices that really are 

developed to fulfil an unmet medical need or treat a rare disease or condition 

offer some kind of alternative benefits to something that's already on the 

market are areas where there's a novel technology and the benefits and risks 

aren't necessarily well known or understood.

I will now turn it over to Martin Ho to discuss about how patient preference 

information uses valid scientific evidence.

Martin Ho: Thank you very much, Annie. FDA respect and appreciate input from patients.

Therefore, we evaluate submitted patient preference information as valid 

scientific evidence along with other evidence from clinical and non-clinical 

testing when making benefit risk determinations. The guidance will not 

change any review standards for safety or effectiveness. The patient 

preference information evidence provides additional insights from patient for 

benefit risk assessment. The guidance provides recommendation regarding the 

voluntary collection of patient preference information that may be submitted 

for considerations as valid scientific evidence as part of the FDA's benefits 

assessment.

Well designed and conducted patient preference study can provide valid 

scientific evidence regarding patient's risk tolerance and perspective on 
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benefit. This may inform our evaluation of a device benefit risk profile during 

the PMA, HDE application and de novo request review processes. The 

guidance has listed 11 recommended qualities of preference studies. While 

there are different ways to group these qualities, for the purpose of this 

presentation we organize them in three different domains.

They are: all about patients, good study design, and good study conduct and 

analysis. Next, we will discuss individual qualities. First and foremost, patient 

preference studies should be patient focused. If feasible, studies should 

primarily measure the preferences and perspective of well-informed patients, 

not other people or their physicians. This study should use a representative 

sample of adequate size so that the study result can be reasonably generalized 

to the population of interest. The patient's benefit risk tradeoff preferences 

may be heterogeneous even among those with the same disease or condition.

So therefore, studies should reflect the preferences of patients from the full 

spectrum of disease for which the device is intended to be used.

Finally, study participants should understand the harm, risk, benefit, 

uncertainty, and other medical information being communicated to them. The 

second domain of recommended qualities is good study design. A study 

design determines the qualities of a patient preference study because it 

specifies what the study measures and how to measure them. In general, 

quality of a study may be established if it follows guidelines for good research 

practices established by a recognized professional organization. Effective 

communication of benefit harm, risk, and uncertainty can reduce uncertainty 

caused by misunderstanding. For example, we will opt for multiple formats of 

presenting of probabilities because different format may work for different 

patients. We also highly recommend conducting pretests of the 

communication format or survey instrument to make sure the respondents

understand of the questions.
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It is very important for us to minimize cognitive biases such as framing, 

anchoring, simplifying heuristics, or order effects. And in order to collect 

relevant evidence to inform regulatory decision making, it is very important 

that the sponsors come talk to the FDA at early design stage. At that time, we 

can discuss relevant harm, risk, benefit, and uncertainties included in the 

studies. We may also want to maintain some level of consistency among the 

benefits, harm, risk, and other attributes between the patient preference studies 

and the clinical evidence.

Finally, it's also very important that the relevance of a specific endpoint to 

potential clinical outcomes be clearly communicated to patients who convey

their preferences.

Implementation and good study design are both equally important for a valid 

scientific study. So the third domain of recommended qualities is good study 

design and good study conduct and analysis. Just like a valid clinical study, 

compliance of research staff and study participants with the study protocol 

determines the scientific integrity of the study. If feasible, study analysis plans 

should include some types of consistent checks, consistency checks such as 

internal validity test of logic and consistency. Finally, robustness of analysis 

results can be evaluated by identifying the potential sources of uncertainty in 

conducting sensitivity analysis as specified in the studies analysis plan.

There are some regulatory considerations regarding submissions of patient 

preferences information for studies submitted with other pre-market review 

data, applicable regulations including IDE regulations in (21 CFR Part 812) 

must be valued. For studies done independent from a device clinical study, 

FDA generally considers the study to be non-significant risk. Conditions of 

approval may be applied in some cases. FDA may impose conditions of 

approval in certain PMA approvals including where the agency take the 
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patient preference information into account to mitigate risk and facilitate use 

in patients for whom benefits are expected to outweigh risk.

FDA may also require collections of post-market evidence through a postmarket approval surveillance studies for the PMAs. As I said, FDA 

encourages sponsors and other stakeholders to have early interactions with the 

relevant FDA review divisions if they're considering collecting and submitting 

patient preference information. You can request an information 

(unintelligible) submission meeting with us to discuss plans for designing and 

submitting patient preference studies. Annie and I provided our email 

addresses here so that you can request our participation in your interaction.

Next, I'm turning to Annie.

Annie Saha: So we've also in the guidance outlined how the inclusion of patient preference 

information may be included in both our decision summaries as well as 

potentially in device labelling. So when we do consider a patient preference 

study in our premarket submissions, we generally will include that in the 

decision summary. And as part of our strategy priority efforts for 2016-2017, 

we have already started this and will be continuing to do so. The inclusion of 

this information we feel is going to be helpful for both healthcare 

professionals and patients in terms of making their decisions, especially in 

areas where there are difficult benefit-risk tradeoffs are novel treatments.

And additionally, patient preference information that's reviewed by FDA and 

supports an approval or marketing authorization should also be described in 

the device labeling, and that that labeling should contain sufficient 

information about the benefits and risks of the treatment for diagnostic options 

under consideration. And certainly, please see our final guidance about device 

labeling for additional information. We also provide here some examples and 
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some resources for you to use if you're considering doing a patient preference 

study.

CDRH embarked on our sort of proof of concept study on how we could use 

patient preferences in obesity and the citation for the paper, which outlines 

how that information was then incorporated as evidence in our regulatory 

decision making, is included here for your -- for you to take a look at and was 

published in Surgical Endoscopy. We also have the benefit risk guidance --

the factors to consider for benefit risk determinations and pre-market 

approvals of de novo classifications as well as guidance on medical device 

patient labeling and through collaboration on the science side -- regulatory 

science side -- we worked with the Medical Device Innovation Consortium on 

their patient centered benefit risk project, where they have a framework for 

how patient preference information could really be incorporated across the 

device lifecycle, as well as a catalog of methodologies. And they've recently 

also just published a resources for sponsors with some frequently asked 

questions and their website for MDIC and the resources there are listed as 

well.

And finally, we'd like to give you an update on the benefit risk -- the summary 

changes to the benefit risk pre-market guidance and some updates we made to 

the benefit risk worksheet. Overall, we updated the guidance to ensure 

consistency with the terminology and the concepts in the patient preference 

guidance. And with that, in section four of the guidance, we talk about the 

factors FDA considers when making benefit risk determinations and really 

added some additional information and clarity about those additional factors 

and the assessment of the probable benefits and risks of devices.

We also included the addition of patient preference information and fleshed 

that out more in the benefit risk worksheet that FDA staff uses to guide benefit 
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risk determinations of PMA and de novo requests, including the section on 

patient perspective on risks and benefit. And to note both the patient 

preference guidance and the benefit risk guidance have a 60-day 

implementation period from the date of publication.

So in summary, patient preference information is voluntary. It can be 

informative during the benefit risk assessment. As Martin noted, this doesn't 

change any of our standards for safety or effectives and that also, patient 

preference information can really be informative earlier at device assessment 

and appendix A of the guidance has sort of a flow diagram of where patient 

preference information can be incorporated and how it could be used, all the 

way from discovery, device design into clinical study parameters such as 

endpoint selection, or effect size. And again, we really would like to 

encourage early interactions with the FDA staff if you are interested in 

planning, or designing, or submitting a patient preference study. And Martin 

and I are available to -- via email or to discuss both a potential study or be part 

of those review meetings.

With that, I'd like to thank you for your time and we'll open it up for 

questions.

Coordinator: Thank you. At this time, if you do any have any questions or comments, you 

may press star followed by the number 1. Again, that is star 1 to ask a 

question. Please unmute your phones and state your first and last name when 

prompted. One moment please.

(Mike McGurty) joined - has a question.
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(Mike McGurty) Hi, thanks for taking the question. Regarding the 60 day date of applicability, 

is that for entirely new applications or would it apply to applications that are 

already in process?

Annie Saha: So this would be for new applications and so the effective date is October 23.

(Mike McGurty) All right, thanks very much.

Annie Saha: And again, it's voluntary so it's not a requirement of sponsors to submit a 

patient preference study as part of their submission.

(Mike McGurty) Okay. Just to follow onto that, is this something where trying to incorporate 

PPI into an application that's already in process, are the reviewers at ODE 

going to think that maybe the whole package needs to be rewritten or can you 

attach this sort of thing in a modular fashion to a PMA, for instance, that's 

already in progress.

Annie Saha: It could be attached as a modular to a PMA that's already in progress.

(Mike McGurty) Okay. Thank you very much.

Coordinator: Thank you. Once again, if you do have any questions or comments, you may 

press star one. Karen Jaffey, you may ask your questions.

Karen Jaffey: Yes, hi. I had a question with regards to extending this preference type of 

assessment to a user, so if it's a prescription device, the actual -- there's the 

patient preference information but then there's also the physician as a user of 

the device itself. Is that something that you see in the future? Is that something 

that's going to be extended by this guidance? What are your thoughts there?
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Annie Saha: In our definition of patient preferences, we do include if there is a healthcare 

professional sort of preference that could be considered as part of the package 

or as part of a patient preference study.

Karen Jaffey: And so that would be something that you could collect in the same fashion 

that you described during the seminar here as far as gathering that data and the 

information and so forth?

Martin Ho: Yes, I think the same criteria applies.

Karen Jaffey: Okay. But it could not -- it should not be an endpoint, as an example though, 

in a study?

Annie Saha: I don't foresee a scenario where that would be the case, but certainly 

something to come in and talk to us about.

Karen Jaffey: Perfect. Thank you.

Katie O'Callaghan: Katie O’Callaghan - I'm the CDRH assistance director for strategic 

programs and I have the honor and privilege of overseeing this partner 

(unintelligible) this guidance. I just wanted to expand on that. The patient 

preference work could potentially be used to support justification for selection 

of a given endpoint out of a list of potential endpoints. And in addition to that, 

could also be used as part of the justification to support a given effect size that 

would be considered clinically meaningful.

Karen Jaffey: That's helpful. Thank you.

Coordinator: Thank you. (Yasmin Sakhet), you may go ahead.
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(Yasmin Sakhet): Hi, good afternoon. Thank you for this presentation. We're from (Accivent) 

and we have a quick question. We understand about the patient reported 

outcomes guidance, but we were interested in learning whether or not the 

patient preference guidance can be applied to small molecules of biologics.

Annie Saha: So the Center for Biologics did sign onto this guidance specifically if the 

submission is related to PMA and de novo requests or HDE applications. So 

it's specific to those -- to that application type. Certainly, feel free to reach out 

to the Center for Biologics if you have additional questions in other areas 

where they might be interested in looking at preferences.

(Yasmin Sakhet): How about some small molecules? Any other similar guidance that we can 

refer to?

Annie Saha: I would, again, follow-up with the Center for Biologic Evaluation, Research, 

and they can discuss with you how to incorporate the patient voice in small 

molecules, molecular,.

(Yasmin Sakhet): Okay. Thank you.

Coordinator: Thank you. Once again, if you do have any questions or comments you may 

press star one. At this time, I am showing no further questions. One moment 

please. Julie Lesley, you may go ahead.

Julie Lesley: Yes, what are your thoughts on including something like a question on net 

promoter value where after a patient or a physician goes through a clinical 

trial, you ask them if they -- what would be their chances of recommending 

this device to others? Would you consider that information?
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Martin Ho: Yes. I think it's a very meaningful question to collect a (unintelligible) and 

(unintelligible) the clinical trials. But it's -- in terms of the nature of the data, 

it's different from the patient preferences information that we have been 

presenting.

Julie Lesley: Thank you.

Coordinator: Thank you. (Richard Counts) you may go ahead.

(Richard Counts): Yes, I was just wondering if it's a -- if it's a good idea to talk to FDA before 

conducting this patient preference and whether it's required given that many 

times these patient preference surveys are given to patients who are not yet 

being treated since there's no -- I can't see any risk to the patient. So I guess 

my question is it necessary -- is it good to talk to FDA beforehand and is it 

required?

Annie Saha: We certainly recommend if you're interested in conducting or performing a 

patient preference study to come talk to us early and we would be happy to do 

so. In terms of requirement, it is not a requirement. The submission of patient 

preference information is voluntary.

(Richard Counts): Thank you.

Coordinator: Thank you.

Martin Ho: Yes, I just wanted to expand on that in these sense that when coming to talk to 

us, you not only be -- help us and understand your design better and so that 

you end up with a better study. But also it's more important that the result of 

the study can yield data that can support and inform our regulatory decision 
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making. But this is a critical point so as Annie said, come early please.

Thanks.

Coordinator: And would you like to go to the next question?

Irene Aihie: Yes, please.

Coordinator: Thank you. CDRX, you may ask your question.

(Dean Brundang): Yes, this is (Dean Brundang). A question regarding the recognized 

professional organizations from the webinar today. Does FDA have a list of 

those that would be considered FDA recognized to do this type of work?

Annie Saha: We do not endorse anyone to do patient preference studies. There are other 

groups that certainly are doing work in terms of methods and potentially 

standards development for patient preference studies but that is not something 

the FDA will do or be involved in.

(Dean Brundang): Thank you. Heather Nagel, you may go ahead.

Heather Nagel: Thank you. I'm just curious approximately how many patient preference 

studies have contributed to either a clearance or approval to date. I'm 

wondering if this is an increasing trend in the industry or if this is a relatively 

new concept still?

Katie O'Callaghan: This is Katie O'Callaghan. Thanks for your question. The use of patient 

perspectives is certainly an increasing trend across the healthcare space and 

certainly within the device and other medical product industries. The use of 

quantitative patient preference studies are relatively more rare. We are seeing 

an increase in that. We expect to see a continued increase in that with the 
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finalization of this guidance in this policy, but certainly the inclusion of 

patient perspectives and other forms of patient input is quite a growing trend 

across the medical product space.

And so if folks have any interest in speaking with the FDA about other forms 

of patient input outside the scope of this guidance, we would be happy to 

follow-up after this webinar and you can direct those questions to the email 

that's on the screen.

We'll take our next question.

Coordinator: Thank you. Jose Cabrera, you may go ahead.

Jose Cabrera: Hi, thanks for the talk. So far very interesting. The concept of usability studies 

has been around for a while and essentially been standardized in pre-market 

applications to the FDA. It seems like if the user is the patient it would be 

plausible to be able to integrate some of these patient preference concepts into 

currently standardized usability studies. Would you guys agree with that more 

or less?

Martin Ho: I would say that the focus of the studies for user feasibility study in a patient 

preferences are different. Feasibility studies may need to (unintelligible) to 

determine whether some features that are user friendly or whether it's 

something that could be improved upon the interface so that the machines or 

devices can be correctly used. Versus patient preference studies, they are 

studying the preferences among different attributes or benefit risk profiles 

between different (unintelligible) options. So it's about the tradeoff between 

benefits, risks, and other (unintelligible) treatments.

Jose Cabrera: Okay. Thanks.
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Coordinator: Thank. (Ron Schoengold) you may ask your question.

(Ron Schoengold): Yes, is it possible to apply the principles of PPI to a traditional 510K?

Annie Saha: So the scope of this guidance is, of course, pre-market PMAS, de novos, and 

HDE requests. But, you know, contact the review division or contact Martin 

and I if you have questions about usability or generalized ability to a 510K.

(Ron Schoengold): Right, this is an incidence where a direct patient interface is required with 

a medical professional. So it's a little bit -- not just a straight device 

submission.

Annie Saha: Okay. Well, feel free to reach out to us or also to DICE, dice@fda.hhs.gov for 

additional questions or follow-up.

(Ron Schoengold): Thank you.

Coordinator: Thank you. Once again, if you do have any questions you may press star 

followed by the number one. (Mac McKean), you may go ahead.

(Mac McKean): Thank you. Thank you for this call. Very informative. Regarding the PPO, 

industry, particularly in the implantables cardiac space have in the past used 

quality of life surveys and on occasion there are validated instruments such as 

hall walks. Do you see a PPO in this type of a design being similar to a quality 

of life endpoint?

Martin Ho: I think by PPO you mean PRO, correct?

(Mac McKean): Right, Patient Reported Outcome, correct.
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Martin Ho: Yes, can you repeat the question? I'm sorry?

(Mac McKean): Well, just aligning the PLL with a quality of life survey that (unintelligible) 

industries conducted on certain implantables such as a hall walk. And in some 

cases there was quality of life surveys become validated instruments to be 

used in clinical trials. How would you compare that to a PRO?

Annie Saha: So quality of life measures you discuss like this six minute walk test, it would 

be a patient reported outcome and we do have guidance about patient reported 

outcomes and how they could be validated. I just pulled up the slide where 

discuss kind of what the difference is between a patient preference study and a 

patient reported outcome. So what you mentioned was really a patient 

reported outcome in terms of the health status from the patient directly about 

how they're doing versus a patient preference study, which is really about the 

types of attributes or in terms of the benefit risks where that patients would 

prefer. And primarily, they wouldn't have even had the treatment at that point.

(Mac McKean): Thank you.

Katie O'Callaghan: Other interesting potential intersection point between patient preference 

and patient reported outcomes is that it's the one we're interested in using 

patient preference methods to obtain a better understanding of what's most 

important to the patient out of a list of potential outcomes and one or more of 

those potential outcomes is a PRO. That would be potentially highly 

informative, particularly in areas for which there are either novel technologies 

being developed and so the regulatory framework is still evolving along with 

that technology development, or in areas where we're seeing novel features or 

other changes being made to an existing technology, which may change 
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patients' views on what is important to them and how they would value those 

various attributes alongside the benefit and risk that's offered.

Martin Ho: Another interesting (unintelligible) between PRO and PPI is that for a subtype 

of PRO that's called preference based patient reported outcomes, they evaluate 

general health states into various different health status. And for each status, 

they link with something called (unintelligible). So that is -- perhaps I digress 

a little bit, but this is a possibility that we can link both together.

Coordinator: Thank you. And once again, if you would like to ask a question or have any 

comments you may press star one. Again, that is star one to ask a question.

One moment please. (Unintelligible) you may ask your question.

(Linda): Hi, this is (Linda) from (Yansen). I had a question related to geography of 

conducted PPI studies. Is the assumption that the patient population is strictly 

U.S.? 

Annie Saha: Primarily (unintelligible) information -- patient preference information could 

be elicited, whether in the U.S. or abroad -- but if you were to use any OUS 

data or any OUS patient preference studies that would have to conform to 

other guidances that we have about the use of outside the U.S. studies. So 

same policy as any other outside the U.S. data.

(Linda): Thank you.

Coordinator: Thank you. At this time, we are showing no further questions. I'd now like to 

turn the call back over to Ms. Irene Aihie for any closing comments.

Irene Aihie: Thank you. This is Irene Aihie. We appreciate your participation and 

thoughtful questions. Today's presentation and transcript will be made 
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available on the CDRH Learn webpage at www.fda.gov/training/cdrhlearn by 

Wednesday, October 5. If you have additional questions about the final 

guidance, please use the contact information provided at the end of the slide 

presentation. And as always, we do appreciate your feedback.

Again, thank you for participating and this concludes today's webinar.

Coordinator: Thank you. You may go ahead and disconnect at this time. This concludes 

today's conference.

END