You can explore clinical trial information at clinicaltrials.gov. (This is a US government registry of all clinical trials held throughout the world.)
As WM is an orphan disease, most current therapies are hand-me-downs from other cancers such as leukemia, non-Hodgkin lymphoma and multiple myeloma. Clinical trials to evaluate new therapies for WM are rare, especially in Canada. Clinical trials offer the patient an extra treatment option in that the conventional treatments will always be available. If we are to accelerate progress towards a cure, more of us need to step forward and participate in clinical trials.
There are three phases of clinical trials:
Phase 1– establishes the safe dosage of a new drug.
Phase 2– determines the efficacy of new drug for a particular cancer.
Phase 3– compares the new drug against current therapies.
Clinical Trials for WM patients are available in the US. Please consult your physician and the US hospital and research clinics involved as to your compatibility for the trial. Further information can be obtained by contacting the WMFC.
WMFC maintains a current list of active or upcoming WM clinical trials in Canada:
Research Project: Dr. Chen, in a WMFC funded project, is researching confirmation of a WM diagnosis through blood samples rather than performing a bone marrow biopsy. Open for Participants. For additional information, contact the University Health Network, Toronto, at firstname.lastname@example.org.
“The Canadian Agency for Drugs & Technologies in Health (CADTH) is an independent, not-for-profit organization responsible for providing Canada’s health care decision-makers with objective evidence to help make informed decisions about the optimal use of drugs and medical devices in our health care system.” The review of cancer drugs by CADTH is carried out by the pan-Canadian Oncology Drug Review (pCODR) Expert Review Committee (pERC). Their role “is to assess the clinical evidence and cost-effectiveness of cancer drugs in order to make recommendations to the provinces and territories to help guide their drug funding decisions”.
BTK Inhibitor (BTKi) drugs such as Ibrutinib, Zanubrutinib, etc, are very good drugs for the treatment of WM, especially for relapsed and refractory WM. Unfortunately, unlike other drugs used in the treatment of WM, they are not currently funded by the Provinces other than Quebec (see below) for the treatment of WM, primarily because of their high cost. (This is despite the fact that most of these drugs are approved by Health Canada for the treatment of WM.) So, unlike other drugs used in the treatment of WM, paying for these drugs becomes an issue.
In Quebec, if your drug prescriptions are generally covered by RAMQ and you are able to obtain periodic authorization from your hematologist for Ibrutinib (other BTKi drugs are to be determined), then RAMQ covers your costs, subject to the same conditions as for any other prescriptions you may be getting.
In all other provinces, and in Quebec if your drugs are not covered by RAMQ, then the following sums up the situation.
The pharmaceutical companies making these drugs are currently providing significant assistance toward covering the cost of the treatment. They do, however, want to reduce their costs whenever possible. If you do not have medical drug insurance, they will typically provide the drugs through their respective Patient Assistance Programs.
But if do you have any sort of medical insurance, they will want to call on that insurance as a source of at least partial funding for the drug.
Thus, if you are prescribed one of these drugs, the pharmaceutical company making that drug will request that you find out from your insurer whether your insurance covers the drug (not all do). And if so, what percentage of the drug costs are covered and whether there are any annual or lifetime limits on the coverage.
Once you have all the information about your insurance situation, you will then need to discuss this with a representative of the pharmaceutical company. Their goal is to understand what financial assistance may be required after any insurance you have is taken into consideration, so that the total cost of the drug can be covered.
Coverage of your treatment is complex and can be overwhelming. But many hospital Oncology programs have resources that can help you navigate this process, take care of the paperwork, and obtain drug coverage.
It is important to remember that BTKi are “lifetime” drugs (you take them until there is disease progression or significant side effects or intolerance), so the annual and lifetime limits provided by your insurance are critical numbers to bear in mind during the discussion.
Once your Oncologist recommends a treatment, make sure you are comfortable with the decision. Conduct your own research, contact the Canadian Lifeline or discuss with your fellow support group members in case they have had to make a similar decision. In certain cases, it might involve getting a second opinion. With a disease as rare as WM, seeking a second opinion may benefit you as well as your Oncologist, as he/she, may have had very limited exposure to treating WM or may not be familiar with all the treatment options that are available to treat your symptoms or minimize side effects. Speaking with a consulting oncologist who may have experience with treating your specific symptoms or at least having treated numerous WM patients, could be very beneficial. This approach might be particularly useful in situations involving relapsed (cancer has returned) or refractory (treatment was not successful) treatment. It is very useful to maintain and forward copies of all your medical records and tests to minimize costs and time for the second opinion.
In March, 2023, we had a National Zoom on the subject of second opinions, presented by a patient advocate with personal experience in obtaining second opinions during her CLL diagnosis and treatments. The video can be found here, on our YouTube channel. The slide deck can be found here, for your convenience.
In Canada: Ask your GP or Oncologist to refer you to a second Oncologist for a second opinion. No one knows all the answers for this disease and requesting a second opinion is valuable to both the patient and the referring Oncologist. You need to be comfortable with your Oncologist’s recommendations especially given the rarity and the few patients with WM your Oncologist may have treated.
In the United States: There are a few hospitals in the United States that conduct research in WM and also specialize in treating WM patients. Just by the sheer numbers, these Oncologists typically see a significantly larger number of WM patients than Oncologists in Canada and may have seen other patients with similar concerns. Medical work in the United States can be expensive, so we recommend considering the following suggestions:
Request a Doctor with Doctor Consultation: Discuss with your Oncologist as to whom he should contact for a second opinion. A few WM experienced US Oncologists are prepared to offer this type of consult as a courtesy to your Oncologist at no cost or nominal cost to you. The WMFC can make some recommendations as to whom to contact should you have some concerns. This is definitely the quickest and the least expensive route, although you may be left out of the discussion.
Send your medical records and tests directly to the consulting Oncologist: This will take more time and there will be a cost involved. Quite often, the consulting Oncologist will be familiar with the Canadian treatment protocols and drug approvals and can make recommendations for treatment in Canada. Also, the consulting Oncologist may request further testing that, with the approval of your Oncologist, can be conducted in Canada. This can take a few days or weeks to evaluate your records and make recommendations for treatment and any further testing that is required and will be moderately costly.
Travel to the United States and see an Oncologist: This can be expensive; however, it might be worth it especially for those patients who want to be directly involved in the second opinion process. Again, the consulting Oncologist may work with your Oncologist and arrange to have as much testing and treatment done in Canada to minimize the costs. There is also the added advantage of possibly being exposed to the opportunity of participating in Clinical Trials in the US, that may be compatible for treatment in Canada, to offer you a different treatment option. Typically, many of the costs associated with Clinical Trials are assumed by the hospital or drug companies involved, in an attempt to encourage your participation.
The WMFC has, through a research funding relationship with Bing Centre for Waldenstrom’s Macroglobulinemia, at the Dana Farber Cancer Institute in Boston, become familiar with their second opinion protocols, which we summarize below. It is our hope that in time the WMFC will be able to expand the listing of US institutions offering second opinions to Canadian WM patients.
Request a Doctor with Doctor Consultation:
Dr. Castillo, at the Bing Center, who has seen literally hundreds of WM patients, is willing to have a phone discussion with a Canadian patient’s oncologist to answer questions about the patient’s unique case. This is a courtesy that Dr. Castillo offers to Canadian oncologists and there is no fee for this service. Please note, this is a doctor to doctor phone call and the patient does not participate in the call. This is definitely the quickest and the cheapest route. Your oncologist can contact Dr. Castillo at JorgeJ_Castillo@dfci.harvard.edu
Sending your medical records to the consulting oncologist:
Travel to the United States and see an Oncologist:
To arrange an appointment, the patient can contact Christopher Patterson, Administrative Director at the Bing Center. Mr. Patterson connects the Canadian patient with The DFCI New Consult Coordinator and he/she can assist the patient in scheduling the next available time slot to see the WM specialist and all the required logistics that are needed for the appointment. The costs are explained in the breakdown during the set up of the appointment by the consult coordinator. It’s all a matter of what is needed prior to the appointment; CT scans, bone marrow biopsy, blood work, just the visit or any combination thereof. Insurance plays a part in factoring the costs, too, which is explained by the new consult coordinator. All in all, the costs vary depending on the person. Christopher Patterson can be contacted at email@example.com or directly by phone at 1-617-632-6285.
A Second Option for US Second Opinions:
As an alternative to travelling to Boston, the Mayo Clinic in Rochester Minnesota is also an excellent choice with a strong focus on both the treatment and research in WM. They are very receptive to discussing treatment options with Canadians and will definitely provide second opinions. Should you be interested in pursuing discussions with the Mayo clinic, please refer to the contact information below.
Mayo Clinic Hematology
200 First St SW
Rochester, MN 55905
Should you encounter any difficulties in arranging an appointment, please contact Dr. Stephen Ansell at firstname.lastname@example.org directly.