Tonight's picture was taken in December of 2002. Mattie was 8 months old, and that was his first Christmas. I can't think of a cuter elf, and as you can see from Peter's expression, it seemed like a very happy and exciting time. As Christmas is approaching eight years later, a whole different feeling lingers in our home. A feeling I would never have thought could exist or happen to us.
As we acknowledge the fact that Mattie has been gone from our lives for 64 weeks, I felt the following poem was appropriate for tonight.
We Remember Them ~ Gates of Prayer
At the rising of the sun and its going down, we remember them.
At the blowing of the wind and in the chill of winter, we remember them.
At the opening of the buds and in the rebirth of spring, we remember them.
At the rustling of the leaves and in the beauty of autumn, we remember them.
At the beginning of the year and when it ends, we remember them.
As long as we live, they too will live.
They are part of us, we remember them.
I did not sleep well last night, though that isn't anything new since I haven't slept well in the past couple of weeks. My sleep goes in cycles! However this morning as Peter awoke for work at 5:45am (the hour he gets up each day!), I got up with him. I wanted to get myself ready so that I could get to the Food and Drug Administration meeting on time. Kristen, Mattie's oncologist and my friend, invited me to attend the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee meeting.
Just so we are all on the same page, the FDA is responsible for the following (pulled right from their website):
(1) The FDA is responsible for protecting the public health by assuring the safety, efficacy and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation.
(2) The FDA is also responsible for advancing the public health by helping to speed innovations that make medicines more effective, safer, and more affordable and by helping the public get the accurate, science-based information they need to use medicines and foods to maintain and improve their health. FDA also has responsibility for regulating the manufacturing, marketing and distribution of tobacco products to protect the public health and to reduce tobacco use by minors.
(3) Finally, the FDA plays a significant role in the Nation’s counterterrorism capability. FDA fulfills this responsibility by ensuring the security of the food supply and by fostering development of medical products to respond to deliberate and naturally emerging public health threats.
At the meeting four drugs [(1) Crizotinib, manufactured by Pfizer, Inc.; (2) Pralatrexate, manufactured by Allos Therapeutics, Inc.; (3) Denosumab, manufactured by Amgen, Inc.; and (4) Eribulin, manufactured by Eisai Inc.] were reviewed and discussed, however, I must admit prior to attending this meeting, I really did not understand the process of how a pediatric cancer drug gets developed, brought to a clinical trial, and thereby allowing children access to new and potentially effective treatments. Keep in mind that the FDA has NOT approved a new pediatric cancer drug in over a decade, and in the words of one parent who testified this morning...... THIS IS UNACCEPTABLE!
The meeting began at 8am and ended probably around 5pm. By 3pm, I had as much as I could absorb and left. However, it was my understanding that this was the very first meeting of this subcommittee which was opened to the public. The conference room I was in today, on the FDA campus, was built specifically for these public forums. So in a way, I observed a very ground breaking meeting!
The format of the meeting consisted of the following: 1) an introduction to the drug company, 2) a presentation by the company of the drug in question, 3) clarifying questions posed by the FDA subcommittee to the drug company, 4) an open public hearing, where parents of children who may have used the drug in question, through a pediatric trial, could voice their support or concerns for the drug, and 5) questions and discussion among the FDA subcommittee regarding the drug.
This subcommittee was comprised of over 20 professionals. Many of whom are pediatric oncologists from either well known national hospitals or they are affiliated with the National Cancer Institute (part of NIH). It took me about an hour to understand the format, to get on board with the lingo, and to understand the dynamics of the members of the subcommittee. It was clear that some of the members are veterans to the world of pediatric oncology and at first, I found the scientific banter upsetting. Upsetting because pediatric cancer was discussed in a rather cold and calculated way. However, as I began to absorb what was being said, I realized that many of the oncologists are very compassionate about their patients and their families, and when a drug was being considered, doctors were truly thinking about its feasibility, effectiveness, toxicity, and the ethical nature of putting a family through certain drug regimens.
There were several breaks during the day, including time for lunch. During those times, I got to connect with Kristen. It is ironic that Kristen was Mattie's doctor, we fought an amazing battle together to save Mattie's life, and yet despite those memories we have become friends. However, what I suspect all along, is that Kristen and I have a lot in common, and now that we do not have this patient-doctor relationship, we are free to get to know each other. Kristen is used to my questions, and one of the things I appreciated about her as Mattie's doctor was that she was open to all kinds of medical questions and when she did not know something she admitted it and researched it. That was a very refreshing change compared to the first oncologist we worked with. I fired many questions at Kristen today as I was desperately trying to figure out the procedure for how a pediatric drug gets tested on patients.
Keep in mind that the drugs given to children to combat cancer, are the result of extensive testing on adults. However, also keep in mind that ADULT cancers and CHILDHOOD cancers are DIFFERENT! Typically adult cancers arise from behavioral and environmental factors, whereas childhood cancers are the result of genetics. Therefore the constant theme presented at the meeting today was CAUTION. These pediatric oncologists cautioned the drug companies, because drugs designed to treat adult cancers, most likely won't work in the same fashion or use the same pathways in children.
You have to ask, what was the purpose of today's meeting? After all, the majority of pediatric oncology trials are regulated through the Children's Oncology Group (a worldwide clinical trial cooperative group supported by the National Cancer Institute (NCI) and fashioned with the mission of studying childhood cancers). So what role does the FDA play in all of this? Well I am learning! In fact, I chatted with a fellow mom today who lost her son to brain cancer. I have met her at other pediatric cancer events, and she told me she has been engaged in the cancer world for 10 years, and she is still trying to figure out the complexity of pediatric drug development. That made me feel better, because there doesn't seem to be a layman's playbook to follow.
To me the FDA serves two roles. First it was very clear that this subcommittee is comprised of a diverse and very knowledgeable group of pediatric oncologists, who literally listened to research proposals from the drug companies, and then for the next 90 minutes gave them feedback, brainstormed research design, its feasibility, and specifically addressed whether it was even worthwhile to pursue pediatric studies with these adult drugs. At the end of the day, they want to assure that children enrolled in a study will have access to effective treatment with low toxicity and side effects. This may appear to be an academic exercise (making a presentation, answering questions, and getting feedback), but it is actually much more than that, because the committee is shedding clinical insights on the proposed drug. The other role the FDA is serving, is it is providing an incentive to drug companies to test and design cancer drugs targeted to children. The FDA implemented the Pediatric Exclusivity Provision (which if you are interested you can find out some information about this below), which in essence provides marketing incentives to manufacturers who conduct studies of drugs in children. This law provides six months exclusivity in return for conducting pediatric studies. Meaning that a drug company is given the exclusive rights to market and sell the drug for its particular purpose, without the competition of generic versions for example. This clearly is a way to generate income for these drug companies, which is naturally an incentive.
It is unfortunate that such incentives are needed in the world of pediatric cancer. But in comparison to the adult population, there appears to be fewer numbers of children developing cancer, and even so, there are so many different forms of pediatric cancer, which all require different treatments. So the task is daunting, especially when the sample size to test drugs on is so small. Though I think it depends on your outlook. Since to me, 46 children diagnosed in this Country with cancer each day is A LOT!
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Implementation of the Pediatric Exclusivity Provisions
http://www.fda.gov/NewsEvents/Testimony/ucm115220.htm
Children suffer from both child specific diseases and diseases that also occur in adult populations. In both cases, children, by necessity, are often treated with the same drugs as those used to treat adults. Even today, only a small fraction of all drugs marketed and used as therapies for children in the United States (U.S.) has been studied in pediatric patients, and a majority of marketed drugs are not labeled, or are insufficiently labeled, for use in pediatric patients. Safety and effectiveness information for the youngest pediatric age groups is particularly difficult to find in product labeling. This is a particular concern as it is this youngest population that is undergoing marked physiologic and developmental changes which are affected by drug therapies. The absence of pediatric testing and labeling poses significant risks for children. Without appropriate testing and labeling, inadequate dosing is likely to occur. Inadequate dosing information exposes pediatric patients to the risk of adverse reactions that could be avoided if such information were provided in product labeling and may reduce the benefits that could be gained if the therapy were properly dosed. In addition, pediatric patients may be denied the ability to benefit from therapeutic advances because physicians choose to prescribe existing, less effective medications in the face of insufficient information about the use of a new medication in the pediatric population. The failure to produce drugs in dosage forms that can be used by young children (e.g., liquids or chewable tablets) also can deny them access to important medications.
The Pediatric Rule, proposed in 1997, finalized in 1998, and effective on April 1, 1999, requires that manufacturers of certain new and marketed drugs and biological products conduct studies to provide adequate labeling for the use of these products in children. Under this regulation, FDA can require pediatric studies of a new drug or biological product if the product is likely to be used in a "substantial number of pediatric patients" or would provide a "meaningful therapeutic benefit" to pediatric patients over existing treatments. FDA also can require pediatric studies of marketed drugs if either of these conditions applies and inadequate labeling could pose significant risks. The term "meaningful therapeutic benefit" is defined as a significant improvement in the treatment, diagnosis, or prevention of a disease, compared to marketed products adequately labeled for that use in the relevant pediatric population (Title 21, Code of Federal Regulations "314.55(c)(5)). FDA considers the term "substantial number of patients" to mean 50,000 pediatric patients in the U.S. with the disease or condition for which the drug or biological product is indicated (63 Federal Register 66636).
FDA IMPLEMENTATION OF THE PEDIATRIC EXCLUSIVITY PROVISION
The Agency has implemented the pediatric exclusivity provision according to the requirements of FDAMA by:
- publishing a list of drugs for which pediatric information may be beneficial;
- working with sponsors to develop and issue Written Requests for pediatric studies;
- reviewing submitted studies;
- making exclusivity determinations; and,
- submitting a Status Report to Congress in January 2001.
During the meeting a discussion arose about Osteosarcoma. Since one of the drugs was suggested to be of value in treating this disease, though there was NO data to support this thinking. Nonetheless, one of the doctors on the FDA panel discussed multifocal osteosarcoma. He basically said that this is a terminal disease and was questioning what kind of role the drug in question would play. As I was listening to him, I felt that he understood my plight, and the challenges Peter and I had to face. What do you do as a parent, do you fight the terminal disease or refuse aggressive treatments and focus upon end of life care for your child? I do not think there is a right answer to this question, other than in our case, fighting was the stance we took. At the end of the seven hours I spent at the meeting, I felt inspired by what I heard. Not that I am under a delusion that pediatric cancer will be cured any time soon or ever, but I was impressed by the caliber people thinking through these issues and struggling with the desire to bring new drugs to children and yet not allowing this to happen without substantial data to back up drug effectiveness.
Peter and I went out to dinner tonight with Jerry and Nancy (Mattie's favorite musical duo at the Hospital). As always we had a lot to chat about, to catch up on, and to share. However, after a full day, I am exhausted, and will leave you with this message we received from Kristen tonight. Kristen wrote, "Although I was fortunate to spend some time with Vicki today I certainly missed seeing you Peter! I want you both to know I am thinking of you on this Tuesday and everyday. Much love."
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