Behind the Counter: Tips from Your Specialty Pharmacist

By Marina Sampanes Peed

Traditionally, cancer treatments were delivered by infusion in clinics and hospitals. Today, many are patient-administered with 30% expected to become available in pill/capsule form in the near future. MPN patients routinely self-administer Hydroxyurea, Pegylated Interferon-a, or Ruxolitinib.

At our Atlanta MPN Patient Education program, Jennifer Powers, PharmD, of Walgreens Specialty Pharmacy explained what these changes mean for MPN patients today. Cancer drugs are classified as “specialty medications” and their coverage varies by prescription drug plan. They are typically not available through retail pharmacies. And the cost to patients varied widely.

Check Your Drug Plan

Above, Dr. Jennifer Powers, PharmD, presents at the Atlanta MPN Patient/Caregiver Program

Dr. Powers urges everyone to look at the new prescription drug plans before Open Enrollment season each year. Make sure the medications you expect to require are covered in your plan. Both public (Medicare/Medicaid) and private, commercial drug plans are subject to changes.  The name of the plan and monthly premium may look the same, but the drugs covered, dollar amount covered, and co-pays may change. Their pharmacy contracts may also change. Each plan has tiers of medications, with different levels of coverage. The tier level of a drug varies by pharmacy, as do the pricing and financial requirements. Some require multiple prior authorizations, continued follow-up, and appeals for off-label use.

More Hoops for Patients

Before you can receive the specialty medication prescribed by your doctor, the Prescription Benefits Manager (PBM) that administers the plan must issue a Prior Authorization. It is quite common for them to reject the initial treatment request, so an appeal must be filed. If the drug is not on their “formulary” (list of drugs covered), it often takes more than one appeal before treatment is approved. Once approved, the medication is ordered and shipped to the specialty pharmacy, then delivered to the patient.

Often refills must also go through the review, denial, and appeal process. With new treatments coming to market and “off-label” use of medications for other illnesses, the process adds to patient fatigue and stress.

Your pharmacist will work with your doctor and assist you through this process.

Financial Toxicity is a Known Side Effect

Financial toxicity comes from the direct costs associated with treatment. There is little room in most personal budgets to absorb ongoing, expensive treatment. Out-of-pocket medical costs (deductible, co-pay, co-insurance) as a percentage of income for managing chronic illness can lead to credit card debt, medical debt, trouble paying for other necessities (housing, food).

The expense of treatment is shown to impact several health related outcomes: treatment compliance; quality of life; and response to treatment.

Some Financial Assistance is Available

There are several Prescription Assistance Programs (PAP) available to patients, each with its own requirements and limitations. The main eligibility criteria include: diagnosis; funding availability; existing insurance coverage; patient’s finances; and FDA approval or off-label use.

Dr. Powers encourages patients to appeal if their application is denied. Some are determined by the prior year’s tax return information; if the current financial situation is worse, explain it in the appeal.   If you exhaust a grant, apply again.

(See the list of resources at the end of this article.)

Importance of Medication Adherence

While it may seem obvious, taking medicine as prescribed is important to manage disease. Dr. Powers highlighted four common challenges for patients:

  1. Affordability: some patients “stretch” the dosing by taking a lower dose than prescribed or skipping a dose regularly because of cost. Some patients buy pills as they have cash available.
  2. Unpleasant side effects: some patients stop taking a medication because of initial side effects or ongoing side effects that affect their quality of life.
  3. Incorporate with Lifestyle: it is important to find the right medication schedule for you. Some are best taken at night so side effects are less noticeable; some may be easier to manage when taken a certain day of the week.
  4. Access to Information: simply knowing what a drug is doing for you, what can interfere with its effectiveness, or how to alleviate side effects can help a patient comply with the treatment plan. Learning about financial and social resources can also help.

Travel Planning Tips

When planning to travel, don’t wait until departure day to think about your prescription medications.

  • Ask your pharmacist about “vacation overrides” to be sure you have enough medicine while away from home.
  • Check if you need a new prescription or a refill.
  • If you need medication sent to a temporary location, make arrangements before you leave home.
  • Check if you need a doctor’s note regarding your diagnosis and treatment to take with you.
  • Get additional packaging, supplies, and ancillary medications.
  • Remember a cooler and ice packs if needed.
  • Check TSA / airline rules about carry-on injectable medications.

Co-Pay & Insurance Assistance Resources

For Jakafi

Patient Access Network: Phone: 1-866-316-7263

PAN Foundation has co-pay assistance program for Myeloproliferative Neoplasms and many other diseases.

Good Days from CDF (formerly known as Chronic Disease Fund, Inc):     Phone:  1-972-608-7141

Good Days is a co-pay assistance organization and Myeloproliferative Neoplasms are covered.

HealthWell Foundation:

Phone:  1-800-675-841

NeedyMeds: Phone: 1-800-503-6897

Partnership for Prescription Assistance:

Phone:  1-888-477-266

Patient Advocate Foundation Co-Pay Relief Program:

Phone:  1-866-512-3861

CancerCare Co-Payment Assistance Foundation:

Phone:  1-866-552-6729

Rx Outreach:   Phone:  1-800-769-3880




Cancer drug fedratinib attracts $90 million for Impact Biomedicines

Less than two weeks after raising $22.5 million, San Diego cancer drug developer Impact Biomedicines has closed a deal for up to $90 million more.

The investment from Oberland Capital will support the launch of fedratinib, a blood cancer medication Impact Biomedicines was formed to bring to market. An application to sell the drug in the United States is being prepared for submission.

Impact is readying manufacturing and logistics so the drug can be available immediately after approval. With strong evidence the drug works against the bone marrow cancers polycythemia vera and myleofibrosis, approval is likely, said John Hood, the company’s CEO.

Drug information will be posted at

Oberland Capital will make two $20 million payments, contingent on Impact meeting milestones. In exchange, Oberland will get royalties on fedratinib sales. If fedratinib is approved, Oberland will lend up to $50 million. The deal was announced Oct. 26.

Fedratinib was first developed by San Diego-based TargeGen, which French drugmaker Sanofi purchased in 2010 for up to $635 million on the drug’s promise.

But in 2013, just as clinical trials appeared to be successfully concluding, a few patients developed a neurological disorder. The Food and Drug Administration put a hold on development, and Sanofi immediately dropped the drug.

Hood and Dr. Catriona Jamieson, a UC San Diego oncologist/researcher, have looked ever since for a way to bring back fedratinib. Efforts began almost immediately, but negotiating rights to the drug from Sanofi and getting the needed funding took time.

Jamieson said fedratinib produced durable remissions in some patients that no other drug could replicate. A number relapsed and died after the drug was no longer available, said Jamieson, the company’s interim chief medical officer.

Having been presented evidence the disorder wasn’t caused by the drug, the FDA has released its hold, and can now consider an application to approve it.

The second round of financing was prompted in part by recent results of a fedratinib trial. Published in The Lancet Haematology, the study found that the drug significantly benefited patients with ruxolitinib-resistant or intolerant myelofibrosis. Sold under the name Jakafi, ruxolitinib is the only US-approved drug for myelofibrosis.

The financing was structured to avoid diluting Impact’s equity, Hood said. The company did grant Sanofi an equity stake of about 10 percent to secure worldwide rights to fedratinib, he said.

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New Patient Guidelines From the NCCN Offer Much-Needed Clarity Around a Group of Rare Blood Cancers

New NCCN Guidelines for Patients® cover the basics and beyond for patients and caregivers coping with myeloproliferative neoplasms.

Newswise — [FORT WASHINGTON, PA  — October 19, 2017] – Patients with blood cancers known as myeloproliferative neoplasms (MPN) have a new resource to help guide them through diagnosis and treatment, in the form of the National Comprehensive Cancer Network® (NCCN®)’s latest addition to the NCCN Guidelines for Patients®. This NCCN Guideline for Patients focuses on the three most-prevalent types of MPN: polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF), which affect approximately 148,000, 134,000 and 13,000 patients in the United States, respectively. [1] Funding for these patient guidelines was provided through the NCCN Foundation® and the MPN Research Foundation.

“As a physician, I find it makes a difference when patients and caregivers have access to the information they need when making treatment decisions, to complement what they’re hearing from me,” explained Brady L. Stein, MD, MHS, Robert H. Lurie Comprehensive Cancer Center of Northwestern University. Dr. Stein is a member of the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Panel for MPN. “Sitting in the hematologists’ office can be an overwhelming experience. These patient guidelines provide the most comprehensive at-home resource available for people with these rare diseases. They cover everything from basic explanations to complicated decision-making around diagnostic confirmation, supportive care techniques, treatment sequencing, adverse effects, and more.”

According to Dr. Stein, it’s not uncommon for patients diagnosed with MPN not to understand at first that their condition technically represents a chronic form of blood cancer, or that it can progress. In fact, most patients and caregivers have never even heard of MPN prior to diagnosis.

“When I was first diagnosed with ET, I actually left the office feeling relieved,” said Christy, a patient living with MPN. “It wasn’t until my pharmacist explained I had a prescription for a chemotherapy drug, that I really had any idea that I had cancer. I just assumed I’d only be taking a blood thinner. I’m grateful that these patient guidelines are helping me to understand what’s going on in my body, and why this chemotherapy is the right treatment path for me.”

“Having this free information available online and on their smartphones is particularly important for patients who can’t just reach out to a friend or relative who’s been through the same experience,” said Robert W. Carlson, MD, NCCN Chief Executive Officer. “The goal is not just to make them feel more informed, but also less isolated.”

NCCN Guidelines for Patients and NCCN Quick Guide™ sheets—one-page summaries of key points in the patient guidelines—are written in plain language and include patient-friendly tools, such as suggested questions for doctors, a glossary of terms, and medical illustrations of anatomy, tests, and treatment. They are based on the same clinical practice guidelines used by health care professionals around the world to determine the best way to treat a person with cancer. Each resource features unbiased expert guidance from the nation’s leading cancer centers designed to help people living with cancer understand and discuss their treatment options with their providers.

The NCCN Guidelines for Patients and NCCN Quick Guide™ sheet for MPN are available to read and download for free online at and via the NCCN Patient Guides for Cancer mobile app. Printed editions can also be ordered from for a small fee.

NCCN currently offers NCCN Guidelines for Patients for the following: Brain, Breast, Colon, Esophageal, Kidney, Non-Small Cell Lung*, Ovarian, Pancreatic, Prostate, Rectal, Stomach and Thyroid Cancers; Acute Lymphoblastic Leukemia; Adolescents and Young Adults with Cancer; Chronic Lymphocytic Leukemia; Chronic Myelogenous Leukemia*; Distress/Supportive Care; Hodgkin Lymphoma; Lung Cancer Screening; Malignant Pleural Mesothelioma; Melanoma*; Multiple Myeloma*; Myelodysplastic Syndromes*; Myeloproliferative Neoplasms; Nausea and Vomiting/Supportive Care; Non-Hodgkin’s Lymphomas; Soft Tissue Sarcoma; and Waldenström’s Macroglobulinemia. * Indicates NCCN Guidelines with new updates coming soon.

NCCN Guidelines for Patients and NCCN Quick Guide™ sheets DO NOT replace the expertise and clinical judgment of the clinician.


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3rd Annual Women & MPN Conference Videos

Women & MPN Logo_Official_RGBsm
We are pleased to have completed the 3rd annual Women & MPN conference on Friday, September 29th, in Los Angeles.  We had wonderful speakers and a lively panel discussion on issues impacting women and all those living with MPNs. One recurring theme was revealed by the newly diagnosed attendees; their physicians didn’t seem to know a lot about their ET, PV or MF and gave little support or direction to resources. MPN Advocacy & Education International is committed to a more vigorous outreach program to physicians and will continue to provide direction, education and resources to all patients and caregivers lacking the support they need.  Our special guest, Emmy-award winning actress, Finola Hughes, will be a great spokesperson for the MPN community because of her ability to reach much greater numbers through the media.


Panel Discussion

Living in Paradox Blog: October 2017

Do Some Things Never Change? It’s On Us!

By Marina Sampanes Peed

While participating in the Women & MPN™ conference in Los Angeles at the end of September, I met several women who were newly-diagnosed with a myeloproliferative neoplasm. They are bright, can-do women who recently learned what is causing their crazy symptoms and cost some of them their employment. As I listened to their stories, a rush of memories ran through me:

Ten years ago, after severe abdominal thromboses, sepsis, and organ failure, a hematologist told me, “At least you don’t have cancer. You should see some of my really sick patients. When you recover from the surgery, you should start working out and lose some weight. You’ll feel better.” Eight years ago, when I almost stroked with a hematocrit of 69.1, another hematologist started treatment (phlebotomies and hydrea) and encouraged me to consult with a MPN expert.   That expert noted some CBC results from several years earlier (separate health care organization) that showed platelet count over 900,000. He explained that I likely started with ET; it wasn’t diagnosed back then because they didn’t know as much and the internist didn’t connect my symptoms to a blood disorder.

Incredibly, in 2017, some hematologists still don’t understand the very diseases they diagnose. I get it; there’s a lot to know. Given the complexities of blood cancers and diseases, they have a responsibility to acknowledge their limitations and refer patients to specialists. Unfortunately, many newly-diagnosed MPN patients receive partial information, treatment plans that look more like Swiss cheese, and faulty expectations.

I naively assumed a decade after my first experience, MPNs would not be foreign to local practice physicians. In the last ten years, significant progress was made with new mutations identified, patient studies, new treatments, and diagnostic and treatment protocols. MPNs receive attention at American Society of Hematology (ASH) and other related conferences. I thought greater awareness and understanding would filter through the hematology network beyond university and research centers. Based on recent conversations with several new patients, this is not happening. And people in smaller communities are at greatest risk.

Fortunately, these women took the information from their physicians and started researching on their own. One way or another, they found MPN Advocacy & Education International’s conference and decided to attend. The physicians who spoke at the conference shared timely information in relatable ways. “I can’t tell you how much relief I feel! Meeting other patients and talking to doctors who know what they are talking about gives me hope,” remarked an attendee.

Another shared, “My doctor told me that PV is no big deal. Then I looked it up and read it is cancer and could kill me. After listening to all the speakers, I’m starting to believe that we can get a handle on this after all. I don’t understand half of what they said, but at least I know it’s complicated and treatable. Now I need to find a good MPN doctor!”

Don’t fight a fact. Deal with it!

So as the MPN progress train lurches forward, we must remember that everyone isn’t on board. Let this be a reminder to take charge of our care. Ask questions. Seek second opinions. Connect with others. Be mindful. Don’t give up.   This caring MPN community is here for you.

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My Child is Being Evaluated for an MPN

How to Approach the Conversation with Your Child’s Hematologist

Dr. Nicole Kucin, MD, MS Assistant Professor of Pediatrics, Pediatric Hematology/Oncology, New York Presbyterian Hospital/Weill Cornell Medicine.

By Nicole Kucine, MD, MS
It can be difficult to know what to expect when your child is being evaluated for an MPN. There is limited guidance on the internet, which can make specialist visits overwhelming to families. As parents, you can expect that you and your child will be asked many questions about symptoms he or she might be having, including headache, abdominal symptoms, rashes, and itching. Your child will undergo a number of blood tests, including some genetic tests, as well as a bone marrow examination. Children who are having symptoms specific to a certain body area may need radiologic tests (such as an ultrasound or MRI.) You should feel free to ask anything you want, and make sure if you are searching the internet you look at reliable sources (such as the MPN Research Foundation, MPN Advocacy Education International, or Some of the questions I am often asked include the following:

Does my child have cancer?
This is a tricky question, and others may disagree, but I do not think of children with MPN as having cancer. In adults, the World Health Organization criteria considers MPN to be chronic forms of leukemia. We are still evaluating MPN in children, and at this point it is not clear that they are all the same disorders in kids as they are in adults. I view the classical MPN in children as chronic bone marrow disorders, and while they have the potential to transform to acute leukemia, this is not something that has been reported in the literature. By envisioning these as chronic illnesses, I think it helps to set the expectations for long-term follow-up and aiming for keeping day to day activities as normal for your child as possible.

Does my child need the bone marrow evaluation?
The answer to this is definitely yes. The bone marrow exam is an extremely important part of the diagnostic process. It can provide a lot of information about what is going on with your child’s blood cells at the source. Things like storage iron, fibrosis, and the appearance of the precursor blood cells are studied. The procedure itself is performed with anesthesia to make sure your child is asleep during the bone marrow test and doesn’t remember it. The pain following a bone marrow test is generally very mild, and children may not require any pain medicine or might require a dose of Tylenol.

What type of MPN does my child have?
The diagnostic criteria for the various types of MPN are based on years of study and data on adult patients. They include features like appearance of bone marrow cells, genetic findings, and lab criteria. Making the appropriate classification for adult patients is important for discussions of prognosis and treatment. We do not yet know if we can directly apply these criteria to children, and knowing the exact type of MPN each child has may not be possible. While it is important to gather all of this information in children, it may not be as important to specifically name the exact type of MPN, and “MPN, unclassifiable” is an appropriate diagnosis for a number of children. The decisions about how to counsel families, what treatments may be recommended, and what follow-up is needed, will be made based on a variety of findings.

Does my child require treatment for his or her MPN?
The answer to this question varies, as I do not believe treatment is required for all children with MPN. I usually determine the need for treatment based on an individual child’s symptoms and lab findings. I generally do not recommend treatment for children who are asymptomatic and have reassuring labs. Children with mild symptoms in the setting of a high platelet count can often benefit from low-dose aspirin, as long as they are not showing evidence of bleeding or acquired vonWillebrand disease. Children with high red blood cell counts can also benefit from low-dose aspirin or phlebotomy. When a child has a severe clinical event such as a blood clot, or does not have improvement of symptoms with initial therapy, then cytoreductive therapy is appropriate. Which medication is used should be decided based on a conversation with the family and the treating doctor. I have been asked about what is my “cutoff” for high platelet or red cell counts for treatment, and there isn’t a standard cutoff. For example, I don’t think an asymptomatic child with a platelet count of 1.3 million necessitates treatment if they are feeling well and otherwise healthy. If you asked 10 different hematologists, you would probably get 10 different opinions on when to treat, so I think it’s important to have a conversation with your hematologist about the risks and benefits of different treatments.

While it can be frustrating to be facing a rare disease, there is ongoing research to help us better understand these conditions in children and adolescents. Keeping an open mind and making sure you have good communication with your child’s hematologist is the most important thing you can do.

Secondary Myelofibrosis Recruiting Phase 1 Trials for DB00493 (Cefotaxime)

This pilot clinical trial studies the side effects of combination chemotherapy, total body irradiation, and donor blood stem cell transplant in treating patients with secondary myelofibrosis. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving combination chemotherapy and total body irradiation before a donor blood stem cell transplant helps to stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient’s bone marrow make stem cells, red blood cells, white blood cells, and platelets.

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MPN Advocacy and Education International Attends a Workshop Hosted by the FDA

By Ann Brazeau, CEO MPN Advocacy & Education International

MPN Advocacy & Education International was invited to attend a very important and timely meeting with the FDA last month. The invitation was extended to us and a few other MPN focused organizations. This meeting launched a unique beginning for those of us representing the MPN Community.

The FDA’s newly formed Oncology Center of Excellence and the Office of Patient Outcomes are committed to engaging MPN patients and advocates to better respond to the need for quality treatment options.

Among the many topics presented by their hematologists, researchers and cancer patient liaison, was an in depth explanation on drug approval processes and how valuable and critical the patient’s voice is to their mission.

After a drug is approved, companies are required to send frequent updates on any and all reported adverse side effects from their drug. Physicians also report on these events and patients can do so by contacting the FDA directly through Med Watch Consumer Voluntary Reporting. Go to Patient Reported Outcomes are extremely important and valuable to the Drug Approval Process and after a drug is approved.

We also learned the complexities surrounding drug “holds” and what happens and why when that hold begins and when it is released. They stressed the importance of ongoing communication between BioPharma and the FDA and encourage regular conversations and frequent updates with comprehensive data.

As the MPN Community pursues the use of Interferon and hopes for accessibility to Interferon as a viable option for treatment, we learned that company(ies) producing Interferon would have to apply for approval specifically for MPNs. It could be a daunting process but there may be avenues we can utilize through patient advocacy efforts.

Although we did not share all of our concerns at this meeting, we look forward to subsequent meetings where we hope to tackle the issues of endpoints, pricing, fast-tracking, and other challenges surrounding MPN drug approval. We were very pleased to have had this opportunity.





Contemporary Use of Interferon Therapy in the Myeloproliferative Neoplasms

The purpose of this article is to review the current evidence behind interferon therapy in patients with myeloproliferative neoplasms.

Recent Findings

Preliminary analysis suggests that interferon may be non-inferior to hydroxyurea in patients with polycythemia vera and essential thrombocytosis. Responses have been observed regardless of JAK2 mutational status, but the presence of non-JAK2 somatic mutations may negatively influence response rates.

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