Medicare Reimbursment for Orphan Drugs
In a major victory for patients, the House and Senate have agreed to several provisions designed to restore Medicare reimbursement to drugs and biologics provided in the hospital outpatient setting. Because access to orphan drugs for rare diseases were being threatened, the Mycosis Fungoides Foundation went to Washington to support this issue for patients.
Since January 1, the federal Centers for Medicare and Medicaid Services had reduced Medicare reimbursement for many outpatient drugs and therapies administered at hospitals, making it difficult for hospitals to continue to provide the medications to patients who need them.
The Medicare hospital Outpatient Prospective Payment System (OPPS), used by the Centers for Medicare and Medicaid Services to reimburse hospitals for services and supplies provided to outpatients, was at the heart of the problem. Treatments for rare disorders were bundled for payment with high-volume, non-orphan products that often cost considerably less. The result is that Medicare patients suffering from rare diseases like ours were being denied access to the newest -- and perhaps best -- treatments available.
Since January, many hospitals have reluctantly absorbed the cost of providing such treatments despite the fact that a number of facilities are operating under heavy budget deficits. However, hospitals cannot continue this practice without adequate funding. The new rule has cut reimbursement rates to hospitals by an average of 35 percent and is forcing hospitals to weigh the impact of new medicines on the lives of patients against the cost of the medications. None of us wants our doctors to be forced to make patient treatment decisions based on cost. But with this new rule, that is exactly what was happening.
Before the new payment system took effect, a few orphan drugs meeting new criteria were exempt from the standard reimbursement rate. This made sense, as demand for these treatments was low and variable, yet extremely costly to manufacture and stock. The new rule places far too stringent criteria around exempt orphan drugs and the Centers for Medicare and Medicaid Services isn't applying its own criteria uniformly. Only four therapies obtained this exemption with the new rule, leaving dozens of other products inadequately reimbursed.
The new rule also had the potential to limit patient access by reclassifying diagnostic and therapeutic radiopharmaceuticals. Although the Food and Drug Administration has classified these products as "drugs" or "biologicals," the Centers for Medicare and Medicaid Services has determined that it will not recognize them as such. This effectively limits reimbursement and thereby patient access to therapies that were previously available.
Although these provisions are now part of the final Medicare prescription drug coverage bill, Congress must pass the legislation for the provisions to become law. The Mycosis Fungoides Foundation will be following this closely, to make sure that seniors do not lose access to innovative drugs and cutting-edge.
by Judy Jones, MFF Executive Director
Rarely am I blown away by a comment, but it happened this week when trying to find an accounting firm to do an audit so we can apply for the Combined Federal Campaign Fund. I had sent our past newsletters to an accounting firm and they were impressed with what we had accomplished. When I told him what our budget was, there was complete silence. Then he said, "I just assumed your budget was over four times that amount…we don’t work with anything that small."
As I look back on this year coming to a close, I think we have done a pretty impressive job of fulfilling our mission to promote awareness and education, advance patient care, and facilitate research. We provided thousands of patients and physicians with information about the disease. We provided patient information and support though our CTCL-MF listserv. We presented a research symposium for physicians. We’ve introduced patients to others at support groups, educational forums and lunch!
The one thing I’ve fallen down on is asking for donations. We do run on a very limited budget. We are able to do this because we have a working, volunteer board of directors, executive director and medical advisory board. We do pay our webmaster/newsletter editor a part-time hourly wage, but have no paid staff. A higher percentage of donations to the MFF are going directly into patient services because of our low overhead.
If you have given to the MFF recently, thank you. If you are considering end-of-the year giving to a worthy cause, think of us. With your help, we can continue to expand our programs.
by Stuart Lessin, M.D., Fox Chase Cancer Center
A clinical trial is a carefully designed study to test the effectiveness of a new drug or disease treatment. Clinical trials are designed to answer three basic questions about a new treatment:
1) is it safe?
2) Does it work?
3) Does it work better than other treatments?
Usually, three separate clinical trials are designed to answer these basic questions.
A phase I clinical trial tests new agents that are administered to humans for the first time. The main objective of a phase I trial is to test for safety, that is, what types of side effects do patients experience and what is the safest dose. For new cancer treatments, phase I trials recruit patients with advanced stages that have not been successfully treated with other therpies.
Phase II clinical trials test how well a new treatment works, at doses determined from the phase I study. Depending on the nature of the new treatment and the cancer being studied, phase II trials may include one or more stages of cancer patients.
Phase III clinical trials compare a new treatment with other established treatments, to determine if the new treatment produces better responses rates. To gain Food and Drug Administration approval, a new cancer treatment must complete all three phases and clearly demonstrate that it is safe (with tolerable side effects) and effective in treating a particular cancer.
The design of a clinical trial, referred to as a protocol, is thoroughly reviewed by one or more panel of experts. An internal review board (IRB), a committee of health care providers, scientists and lay persons carefully review all human safety and ethical issues and must provide the final approval, before a clinical trial can begin. A clinical trial is continuously monitored and reviewed annually by the IRB to ensure that it is conducted properly and that the tested treatments are safe.
Where to find more information:
NCI's Cancer Information Service (CIS): The CIS provides toll-free access to trained cancer information specialists at 1-800-4-CANCER. Additional information is available at http://www.cancer.gov/cancerinfo/.
NCI's Clinical Trials Website: This website enables visitors to search for clinical trial results, developments, ongoing clinical trials, general clinical trial information, and more. http://www.cancer.gov/clinicaltrials
ClinicalTrials.gov: The U.S. National Institutes of Health, through its National Library of Medicine, has developed ClinicalTrials.gov to provide patients, family members and members of the public current information about clinical research studies. This website contains information on cancer and other conditions. www.clinicaltrials.gov
Thank you for your heartening response. We have been "in the dark" on how to support our mother with this issue that she has been dealing with for so very long. We assumed she was struggling with this by herself, and much to our relief she has found others like her. It has given her much joy to know that her children have taken interest in this with her--and now she has the support of "us."
I urge you to press other people in the MFF to speak openly with their friends and family about the MFF and what is has meant for them to be a part of this organization. It will most certainly light some light-bulbs for others who have been just as perplexed as we have been. We didn't know if we could talk about it with her--or if she felt comfortable because to mention it is a reminder to her, and to us, that our mother has cancer--and that is terrifying.
I'm sorry to ramble on, but I am deeply concerned for her well-being and her quality of life, and I am afraid to even utter the words, "the end of her life." I am the youngest (although 38) and I don't know what I am going to do without her. Cancer is a crossroad where logic and emotions collide. I wish it was just a rash; I am angry, but I don't know why; I cry when I think about the reality--but I have witnessed that happiness vastly improves the quality of life in whatever state anyone is in--so I am making it my task to encourage happiness for my mom. I thank you for doing the same for her--and for yourself.
I am dropping the check in the mail today, and thank you again for your help.
You can honor a friend or family member for any occasion with a gift to the Mycosis Fungoides Foundation. Please include the name and address of the individual being honored so that acknowledgement of your kind donation can be sent.
Thanks to those of you who have responded to our letter requesting donations. If you would like more return envelopes to pass out to friends, neighbors or relatives, let us know and I’ll send them to you. Your contributions make it possible to provide information and services to patients and physicians.
If you work for Johnson & Johnson, or live in the New Jersey, Southeastern Pennsylvania or Delaware area and give to the United Way, you can now designate your contributions to go to the MFF. Our United Way donor designation number is: 042977.
In Honor of (August-October 2003):
M. Scott Alexander Maitland, FL
Thomas Andrews Vail, AZ
Mrs. David Anderson King of Prussia, PA
Louis Batson Chapel Hill, NC
Edward Breen Atlantis, FL
Alexis Dizon Cardiff by the Sea, CA
Julia Durrance Ocala, FL
David Elefant Alameda, CA
Charolette Hamm Pinecrest, CA
Victor Matsey Mount Pleasant, PA
Joseph Lawrence Annandale, NJ
Diana Lewis Tucson, AZ
Nan Needham Coatesville, PA
Norach Rodriguez Park City, UT
Paul Zaydon, M.D. Pawtucket, RI
In Memory of (August-October 2003):
Adam, Jonah, Jordan & David Hewlett Harbor, NY
Paul Danforth Sebastian, FL
Mary and Armen DeCola Chads Ford PA
Doris Ann Delmarmo Tems River, NJ
Michael Drilling Palm Beach Gardens, FL
William B. Harned Bunceton, MO
Thomas E. Homulka Mt. Pleasant, PA
Barry Kupsch Barrhead, Alberta, Canada
Laurence Longcore Conway, MI
Phyliss McLemore Lake Success, NY
Joyce Pearson Chicago, IL
Ruth Robbins Sarasota, FL
Helen Shea Fredley, MN
Irving Silverberg Bronx, NY
The Reverend Andrew M. Weyermann Milwaukee, WI
Dorothea I. Zetler Dilon, Mt.
Cutaneous T-cell lymphoma (CTCL) received special attention at the National Institute of Arthritis, Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health (NIH) two-day conference entitled “Immunomodularity Drugs in the Treatment of Skin Diseases: What Can We Learn About Pathophysiology?” Because CTCL is a rare disease, it is infrequent that it enjoys a moment in the spotlight. The more CTCL is addressed in these formats, the greater the opportunity for additional drug testing and research.
Members of academia and the pharmaceutical industry attended the conference. The goal of the conference was to develop a better understanding of skin diseases through the evaluation of their responses to immunomodulatory drugs. Immunomodulatory drugs refer to new biologic agents that have been developed to target the immune system in beneficial ways.
Skin diseases that respond to immunomodulatory drugs include those that have an immune abnormality associated with them. Diseases that were discussed include atopic dermatitis, psoriasis, CTCL, lupus, vitiligo, alopecia areata, pemphigus, pyoderma gangrenosum, graft vs. host disease and non-melanoma skin cancer.
The use of multimodality treatment in CTCL, i.e., use of biologic response modifiers such as photopheresis, Interferon-alpha, retinoids, etc., was discussed by Alain Rook, M.D. of the University of Pennsylvania. Madeline Duvic, M.D. of the MD Anderson Cancer Center reviewed the experience with bexarotene (Targretin®) and other retinoids, and their favorable response rates. Lars French, M.D. of the University of Geneva reviewed preliminary data of a new treatment approach with use of a novel synthetic molecule that targets the CD-40 receptor, and appears to have beneficial effects in CTCL.
The conference highlighted many other new findings, and provided stimulus to investigators in academia and industry for future collaborations to continue to develop and test immunomodulatory drugs for the treatment of skin diseases. The inclusion of CTCL on the agenda increased the disease’s visibility. Such visibility is very beneficial and can increase opportunities for new immunomodulatory drugs to be tested in CTCL.
“Ask the Doctor” is a new regular feature of the MFF Forum. If you would like a question answered, please send it to: MFF Forum, PO Box 374, Birmingham, MI 48012-0374. Or, send us an email.
The following questions were answered by Stuart R. Lessin, M.D. Director of Dermatology, Fox Chase Cancer Center, Philadelphia, PA
How do I benefit from participating in a clinical trial?
Participation in a clinical trial may provide you with access to a beneficial treatment that would otherwise be unavailable to you.
Does my doctor have to approve my participation in a clinical trial? If not, should I consult with my doctor anyway before volunteering?
Participation in all clinical trials is strictly voluntary. It is your decision to participate. Participation eligibility does not require a doctor's approval. In order for you to make an informed decision about your health, however, it is important to gather as much information as necessary, so you can understand the benefits and the risks of the treatment options that are available to you. Your doctor is a valuable source of information and advice, and you will benefit from consulting your doctor concerning participation in a clinical trial.
How do I find out about clinical trials?
The first source is your doctor. Then, a medical center that specializes in your disease will usually have information about clinical trials. National organizations, such as the American Cancer Society and the National Cancer Institute have information about cancer clinical trials. Patient advocacy groups and disease foundations (www.mffoudation.org) provide information and help in learning more about clinical trials. The Internet has become a great tool in helping patients learn more about clinical trials (www.clinicaltrials.gov, www.centerwatch.org, www.leukemia-lymphoma.org).
Can I participate in a clinical trial that is outside my local area? Who pays for my travel expenses?
Yes, you can participate in a clinical trial outside your local area. Some clinical trials will reimburse participants for their travel expenses, but usually for local travel. You need to ask the organizers of the clinical trial about travel expenses, if you are considering participating in a clinical trial outside of your local area.
Don't you have to have "no other choices" before you participate in a clinical trial?
No, you can participate in a clinical trial if you meet the study's eligibility criteria. The type of clinical trial, the treatment being tested, the stage of your cancer and your medical history all contribute to determining whether you are eligibile to participate in a particular clinical trial. Often times, clinical trials become attractive treatment options to cancer patients who have not responded to standard therapies. A clinical trial may provide them with an opportunity to choose a new treatment.
In the last three months, we’ve had lots of activity in getting patients face-to-face with other patients. As much fun as it is to meet new people, it is even more fun to see people that have come to previous meetings and catch up on how they’re doing.
LA Support Group
In September, The Leukemia & Lymphoma Society, The UCLA Oncology Center and The Mycosis Fungoides Foundation co-sponsored a "Meet the Doctor" evening. Dr. Lauren Pinter-Brown, Clinical Professor of Medicine at UCLA, presented an overview of cutaneous lymphomas, including mycosis fungoides, lymphomatoid Papulosis and Anaplastic large cell lymphoma.
Thanks to Cheryl Abe, LCSW and Frini Li-Chiu, LCSW from the UCLA Oncology Center as well as Robin Shlien, Community Outreach Manager from the Leukemia-Lymphoma Society for pulling together all the details.
When Judy travels to meetings, she puts a message out on the CTCL-MF listserv asking if anyone wants to meet for lunch. This is a great way to get to meet and talk to patients in an informal setting. Here’s what she had to say about this "special group".
This is the first time we’ve ever had an all female group for lunch. Let me tell you, it was also the longest "lunch"! What a dynamic group of women.
LRF Educational Forum
Kudos to Sue Bliss, Executive Director of The Lymphoma Research Foundation Educational Forum, and her staff. They once again put on a great educational forum. Melanie Smith, Director of Public Policy, introduced an advocacy training program which was very informative. Jennifer Mills in her new position, Director of Patient Education, and Carolyn Bell, Director of Patient Services, coordinated the dedicated people of LRF in their efforts to bring quality information to lymphoma patients.
Dr. Richard Edelson, Director of the Yale University Comprehensive Cancer Center, presented an outstanding talk on cutaneous T-cell lymphoma. The sessions were audio taped, and if you are interested in getting a copy, they are $7.00 + $1.50 shipping and handling. They can be ordered from Network Communications over the internet at: www.swiftsite.com/stloa2003. Phone number: 800-747-1426. The number of the tape is: LF-N07.
Pittsburgh Support Group
Sue McCann, who is the Photopheresis Coordinator at the University of Pittsburgh Medical Center, managed to get 50 patients, friends and family members together for the Brian V. Jegasothy Support Group for CTCL in October, which turned into an educational forum. She gave a presentation on the Nursing Perspective on CTCL.
Dr. Larisa Geskin, Director of Cutaneous Oncology, presented an overview followed by Dr. Frank Strobl from Therakos Pharmaceuticals, who described photopheresis (ECP). Khanh Bui, from Ligand Pharmaceuticals, gave a product overview and Judy Jones gave a patient prospective and brought everyone up-to-date on the Mycosis Fungoides Foundation activities.
Thanks to Therakos and Ligand for providing us with lunch and a chance to get acquainted with other patients. Linda Sharp and Joyce Grater facilitated the caregiver breakout and Judy Jones facilitated the patient support group.
These meetings provide an excellent opportunity for people to learn more about this disease and the treatments that are available.
The Brian V. Jegasothy Support Group for CTCL is a yearly event. Please join us next year!
The Mycosis Fungoides Foundation, in collaboration with Therakos, Inc. hosted a live webcast on mycosis fungoides, Sézary syndrome and other forms of CTCL. Drs. Madeleine Duvic of the M.D. Anderson Cancer Center and Marie-France Demierre of the Boston University Medical Center discussed treatments including photopheresis, upcoming clinical trials, and other important issues concerning this disease.
Joe Lawrence, a photopheresis patient, shared his perspective in his short presentation. His clear understanding of treatment options, combined with his personal experience, made Joe’s contributions to the presentation valuable.
Participants e-mailed questions that were answered in a question and answer period afterwards. We received more questions than we could answer in our allotted time, so we are going to record a supplemental question and answer period. Both the webcast and supplemental questions will be assembled on a CD, so that it can be watched off-line. We will have more information available on how to obtain this on our website and in our next newsletter.
To watch the webcast, go to our website at: www.mffoundation.org and follow the directions. We are fortunate to be able to bring speakers of this caliber into your homes to give information and answer questions. Thanks to Therakos, Inc. for the tremendous time and effort that went on behind the scenes to put this together. Without their generous grant, this would not have been possible.