FOOD AND DRUG ADMINISTRATION CENTER FOR DRUG EVALUATION AND RESEARCH

PanCAN Chairman Paula Kim's Remarks at the Meeting of the Quality of Life Subcommittee of the Oncologic Drugs Advisory Committee

My name is Paula Kim and I am here to represent what is a newly formed advocacy group for pancreatic cancer. I'm sure you're all aware about pancreatic cancer being one of those cancers that's a deadly one. Just to share a little bit as far as who and what, it's a cancer that affects 29,000 people a year, approximately. It's the fourth leading cause of cancer death for both men and women in this country. It's a disease that usually strikes very silently and has the highest mortality rate of all cancers at 99 percent. Most of the patients do not live beyond a year, and their quality of life is very poor. It's poor because the disease itself is very aggressive, and so it's the effects of the cancer itself, but their quality of life is also poor because, unfortunately, there are very, very few treatments available for pancreatic cancer. So, it's a little bit of insult to injury in terms of having anything available to them.

There is no cure at this point. There is no early detection. So, they're left with a very terrible set of circumstances to deal with. Therefore, quality of life becomes extremely important for this particular community because, as many of the doctors have said to us, their patients say to them, well, I realize I have this prognosis, I realize that it's terminal, but can we try and make it as nice as possible while we go through this journey where we know where the end target is. One of the biggest struggles that the doctors are dealing with is how to help these people maintain their dignity in what is a really difficult situation and dealing with the end stages of their disease to try and maintain some sense of quality of life. In many instances, these people are given a diagnosis and then three to four months later, that's it. So, that time that they have shrinks and shrinks and they need good quality time for that.

Why do we need to be doing this? Because we have a population that is increasingly aging and we're going to be dealing with a lot of people that are going to demand, and we demand good quality of life.

Quality of life, if we can include this as an endpoint, will allow a more comprehensive approach. It includes being able to have a physical and a mental component to all of the care, and not just looking necessarily always at this clinical benefit and outcome.

There are studies that have shown where certain aspects of improving things such as pain management can affect survival outcome. There was a study done by Dr. Keith Lillemoe, one of the surgeons at Johns Hopkins, talking about pain management subsequent to whipple surgeries. His study clearly demonstrated increased survival. So, I think that if we look at the big picture, there can be benefits derived from that.

"We have an obligation and a responsibility to provide
good quality of life the best that we can."

The questionnaires and the measurement assessments that have been developed over the past several years are improving. They need to get better, but they are improving.

So, what we need to do at this point is we need to look at what the barriers are or what the obstacles are to achieve better quality of life assessment and how to include that in the overall picture. We need to find the weak spots such as the areas that tend to skew the data like missing data and things like that. We need to figure out how to fix that so that you can fix those problems.

People talk about the increased burden of the investigators having to have additional costs and monitoring and all these things that go with that. That may be so, but I think what needs to happen is there needs to be training and there needs to be a system set up in place that these people are given the support, the clinicians and the data managers. They need the support to be able to provide the data. I think they all work very hard at what they're trying to do, but sometimes there is a lack of support, tools, and resources that allow them to do their jobs correctly.

Then what we need to do is the data needs to be reliable. It needs to be valid. The way that that can be accomplished is in the beginning a really good system has to be set up where you establish good criteria, solid criteria, meaningful criteria, and then you take that and then you develop that into the analysis methods that you need.

So, how we do this is taking advantage of technology available today. Much of the data that's collected and much of the methods that are used are really very old-fashioned. They need to be brought forward and they need to take advantage of the technology. We have a lot of technology at our fingertips. Yes, sometimes systems that are this large are slow to respond, but we need to use the technology available to us because then, in turn, the results will generate themselves down the road.

We have to innovate. We have to strategize better on how to analyze the data and then how to use the data. When a cancer diagnosis is given, in many of the instances, fortunately due to good science and due to clinical trials and due to these things, cancer is not always a death sentence. It is oftentimes an acute disease that can become a chronic disease, which is good progress.

In the case of pancreatic cancer, though, we don't have that progress at this point. We have a diagnosis and then we have death. So, either one, whether it's going from acute to chronic or diagnosis to death, it really doesn't matter because all types deserve quality of life because just because you have a diagnosis doesn't mean that you should lose hope and it doesn't mean that you have to suffer from poor quality of life.

So, on behalf of our community, we would greatly encourage you to strongly consider including this because I guess the way that we look at it is, yes, there is a thing as the primary endpoint and all the clinical definitions of everything, but there's really more than one way to skin a cat. So, what we need to look at is how offer the best. Particularly to those with this type of disease, it's a tremendous burden. Thank you very much.