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COVID Vaccine and Our Patient Communities

Across the country we are starting to see the light at the end of the tunnel as the rollout of COVID-19 vaccines has begun. For many of our patients with primary immunodeficiency diseases or autoimmune conditions, however, getting vaccinated is not a straightforward decision. They have lots of questions and must weigh carefully a number of considerations.

Immunologist and rheumatologist Dr. Terry Harville has some concerns, too. “There are questions in my mind that haven’t been fully answered,” he says about the new vaccines that have recently been granted emergency use authorization by the Food and Drug Administration (FDA). “And I’m worried that there will be unintended consequences.”

The fact that we have only two months’ worth of safety data from the clinical trials testing these new vaccines from Pfizer/BioNTech and Moderna leaves many medical professionals worried about longer-term effects. Others are unsure about how the vaccines will react in patients with rare autoimmune and primary immunodeficiency diseases, because testing did not include people who live with these conditions.

Despite these concerns, Dr. Harville—along with most other healthcare practitioners—stresses that most people should still get the vaccine. Because when hundreds of thousands of people get COVID and one or two percent of them die, the statistics overwhelmingly support COVID-19 vaccination.

Dr. Harville, who is a professor of medicine at the University of Arkansas for Medical Sciences and an expert in primary immunodeficiency disorders, offers some guidelines for those who are trying to make a decision about the vaccine and might be at greater risk for complications from it.

If you have a primary immunodeficiency (PI) disease, you probably already know that you need to be careful about taking immunizations, especially if they are made with live virus. The COVID-19 vaccines that are currently being administered, however, are not made with live virus. They are created from a man-made part of the virus’s genetic code called mRNA. This means they are safer for those who are unable to take live virus vaccines.

With a few exceptions, Dr. Harville says, “It is the general consensus that we don’t see any reason to not vaccinate those with primary immune deficiency diseases.”

One exception is interferonopathies, IDs associated with dysfunction in components of the immune system called interferons. Another exception is CTLA4 deficiency. These are conditions in which patients tend to develop autoimmune disorders. Dr. Harville recommends that individuals with these types of ID should probably not get the currently available COVID vaccines. While there is no data to support this at this time, there is concern among immunologists that vaccination in this group of patients may trigger an overly aggressive immune response that would be harmful. As more data becomes available, better recommendations will be forthcoming.

If you use immune globulin therapy, there appears to be no reason you shouldn’t get the vaccine. In fact, even if you get the disease, you may actually be better off than others.

“What’s interesting is,” Dr. Harville says, “if you look at SARS-CoV-2 infection in patients with antibody deficiencies—whether that’s X-linked agammaglobulinemia (XLA) or common variable immunodeficiency (CVID)—when these patients are receiving appropriate immunoglobulin (IG) replacement therapy, they tend to have milder disease.”

Intravenous immunoglobulin (IVIG) has even been used, in some cases, to treat COVID-19 infection. While clinical trials to prove effectiveness are still ongoing, some physicians believe IVIG does decrease the risk of severe complications. IG manufactured from the plasma of people who have recovered from COVID-19 infection is also being tested as a treatment.

If you have had a severe reaction to immunizations in the past, this may be a reason to opt out of getting the COVID vaccine. This includes those who have a primary immunodeficiency disorder and developed the infection after receiving a vaccine. It also includes autoimmune disease patients who have experienced a severe flare of their symptoms after a vaccination.

In the 1976, there was a rise in cases of the autoimmune condition known as Guillain-Barré syndrome (GBS) that was thought to be triggered by the swine flu vaccine given that year. This was an inactivated virus vaccine, not the type of mRNA vaccine we now have with COVID. Nevertheless, if you’ve had GBS in the past, be sure to let your healthcare provider know this, as it may be a consideration for whether or not you should get the COVID vaccine.

During the COVID vaccine clinical trials, a very small number of participants who received the Pfizer vaccine developed a severe, life-threatening allergic reaction. Since then, others have also had what is called an anaphylactic response soon after getting vaccinated. If you have ever experienced shortness of breath, tightening in the throat, swollen lips or face, or other severe allergic symptoms, you should probably not get this vaccine in particular. (Other vaccines using whole virus are currently in development and will likely be safer.) Anyone who does get a COVID vaccine should be observed for at least 15 minutes and be sure the healthcare personnel administering it have emergency equipment needed to respond to anaphylaxis.

Regardless of your disease, everyone can reduce their own risk. Dr. Harville stresses that, even if you get vaccinated, it’s still important to wear a mask, wash your hands, and maintain physical distance.

“Ultimately whether you take or do not take the vaccine is an individual patient decision,” Dr. Harville says. “This should be made after weighing all the risks and benefits and based on a discussion with your physician.”

We highly recommend that you learn more about the risks and benefits of COVID-19 vaccination as it relates to your particular situation. The Centers for Disease Control and Prevention (CDC) offers these guidelines for COVID-19 vaccination for those with underlying medical conditions. In addition, a number of disease organizations offer educational programming about the impact of the virus and the vaccine on specific disease conditions.

Please also see disease-specific statements about COVID-19 vaccines from these patient organizations:

Guillain-Barré syndrome – GBS|CIDP Foundation

Immune Deficiency Foundation

Myasthenia Gravis Foundation of America

The Myositis Association

Platelet Disorder Support Association

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Patient communities

Stumbling onto Rare Disease

Susan Foley used to brag that if she told you she forgot, she was lying. For most of her life, she remembered everything. In the last ten years, however, her mind is more like someone with a traumatic brain injury. She loses her train of thought, and sometimes she can’t get the right words to come out when she talks. 

Susan has Hashimoto’s encephalopathy (HE), a type of autoimmune encephalitis (AE) that causes swelling in the brain. When this happens, patients experience an odd array of symptoms. They can say strange things, act like they have no feelings, and stumble, as Susan says, like a drunken sailor. Too often, family members and even doctors think these people are just behaving badly. So getting an accurate diagnosis for this rare disease can be a challenge.

About a year before she was diagnosed, Susan started having trouble walking up steps, noticed tremors in her hands, suffered debilitating headaches, and couldn’t make her bed in the morning without needing to take a nap. She blamed these symptoms on her stressful job. When she began having absence seizures (a type of epilepsy in which the person blanks out for a few seconds, staring off into space) it scared her. She knew she needed to get help.

“I had so many different symptoms that the doctors had no idea what was going on,” Susan says. “My primary doctor kept telling me it was stress related, but I knew there was something more going on. Finally, I asked her to send me to a neurologist.”

Still, it took almost a year of every imaginable diagnostic test and a trip to see a specialist at a large teaching hospital for the neurologist to put all the pieces together. Among the tests the specialist did was a blood test for thyroid antibodies. The results were off the charts. This was the clue the neurologist needed.

“HE has nothing to do with your thyroid,” Susan says. But doctors have noticed that this form of encephalitis is associated with elevated thyroid antibodies. As in Susan’s case, this is often how physicians identify the disease.

Having a diagnosis is one thing but figuring out what this means for your life is a whole different process. When Susan went to the internet in search of accurate information about HE, all she found was a single article. It took a lot more digging through online forums and support groups to find even one other person who had the disease.

That one other person turned out to be Nicola Nelson, a Chicago attorney who had also been recently diagnosed with HE. The two became good friends and, when Susan told Nicola about her idea to write a book, she wholeheartedly agreed.

“I had big dreams, you know?” Susan says. “I thought, people who are diagnosed with HE have no information. They need to know from other patients what to expect, what that patient has gone through.”

Hashimoto’s Encephalitis: A Guide for Patients, Families, and Caregivers, now in its second edition, is still the only book about HE available. It includes stories of patient experiences along with interviews with prominent physicians who specialize in the disease.

In addition to the book, Susan and Nicola started a nonprofit patient organization to provide ongoing support and information for those affected by HE and other autoimmune encephalopathies. HESA (Hashimoto’s Encephalopathy/SREAT Alliance), provides both scientific and experiential information on living with HE (also known as steroid-responsive encephalopathy associated with thyroiditis [SREAT]; encephalopathy associated with autoimmune thyroid disease [EAATD]; and nonvasculitic autoimmune inflammatory meningoencephalitis [NAIM]).

HESA raises awareness and advocates for the HE community and raises funds to support research that patients hope will one day shed more light on the underlying cause of HE/AE and create new, more effective therapies. Last year, HESA joined together with other AE organizations to host a symposium to educate physicians and other healthcare professionals. (Recordings of these and other presentations are available on the HESA website.)

HESA’s support groups include two private Facebook pages, one specifically for teens. Susan and her medical advisory board also consult with individuals privately on everything from physician referrals to how to get insurance to approve IVIG therapy to sensitive issues such as incontinence.

Despite the fact that Susan’s career in business ended when she became sick, she’s busier now than she’s ever been. HESA is her labor of love.

“I will tell you, when one door closes, another door opens,” she says. “HESA has been a life saver to me. I still feel like I’ve got something to give, and I tell people when they’re feeling sorry for themselves, they need to find a purpose. Do not feel sorry for yourself. Do something. It will give you a reason to get up in the morning.”

One of the most effective therapies for autoimmune encephalopathy is IVIG. CSI Pharmacy is excited to welcome HESA and those with AEs into our family of care.

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Advocacy CSI Pharmacy stories

Do you have a COVID-19 Preparedness Plan?

(BPT) – If you’re concerned about getting COVID-19, you have good cause.

According to the latest COVID-19 Data Tracker from the Centers for Disease Control and Prevention (CDC), the U.S. alone has already logged over 16.3 million cases and the overall weekly hospitalization rate is at its highest since the pandemic began.

While it’s critical to stay informed about the spread of the virus and know how to protect yourself and others, many people may not realize how important it is to be prepared with an action plan should an outbreak occur in your community or household.

The better informed you are, the more empowered you can feel about coping with the pandemic.

COVID-19 Preparedness Tips from a Professional Patient Advocate Who was Hospitalized

Professionally, Michelle Vogel works in patient advocacy and provider relations, helping other patients navigate the healthcare system. That experience has taught her that sometimes, patients must be proactive and push to find their best options. Preparing as much as possible in advance is paramount.

Have a testing plan. Know the symptoms and if you have been exposed or experience symptoms, know where to get tested in your area, and know how you’ll isolate or self-quarantine if necessary. Relevant information is listed and regularly updated on the CDC website.

Get to know the hospitals in your area. Look up local and regional hospitals in your area and make a plan for where you would seek treatment if you needed to be hospitalized. If you want access to investigational treatment candidates, websites like www.StopStorm.com can help you find research hospitals in your region as they may be enrolling people in clinical trials.

Ask about treatments and clinical trials. Everyone reacts differently to the coronavirus. As such, there’s no one-size-fits-all approach when it comes to treatment. If you’re hospitalized with COVID-19, ask about treatment options and if you may be eligible to enroll in a clinical trial. Since eligibility criteria can change, it’s important to keep asking your hospital and doctor about clinical trials daily.

Throughout this crisis, the biotech and pharmaceutical industries have worked tirelessly to develop vaccines and therapeutics to help overcome this pandemic. However, with over 4,000 COVID-19 clinical trials listed on www.clinicaltrials.gov and the healthcare system almost overwhelmed, the burden to get access to clinical trials is now often falling to the patient or their families who may need to proactively plan, ask and engage.

Clinical trials are crucial in the development of innovative medicines and participating in a clinical trial helps advance potential treatment options for people in need.

“Typically, hospital researchers will discuss clinical trial options proactively with patients who may be eligible to enroll,” said Vogel. “I think that there is so much research going on right now, patients and their families have to be more vocal and engaged in asking about potential clinical trials than ever before.”

What Happens When the Professional Patient Advocate Becomes the Patient?

The expression, ‘when it rains, it pours,’ doesn’t even begin to capture how torrential Michelle Vogel’s experience was with COVID-19. It was the midst of Florida’s hurricane season, the virus was surging in the state and her mom was just diagnosed with COVID-19 in Boynton Beach, Florida, days prior.

While Michelle, a 53-year-old Jacksonville, Florida, resident, waited to get her COVID-19 test, delayed due to heavy storms, her symptoms were getting worse. After her test came back as positive, she went to the research hospital in her area and was admitted to the Mayo Clinic in Jacksonville. She was diagnosed with pneumonia and was treated with convalescent plasma, remdesivir and steroids. Her condition deteriorated and she developed a hyper-response of the immune system that causes harmful inflammation, known as cytokine storm. The condition affects almost 90% of patients hospitalized with COVID-19 and is a leading cause of COVID-19 progression, use of a ventilator and death.

Vogel asked about clinical trials and learned that the Mayo Clinic was one of the sites enrolling patients in a Phase 3 study evaluating lenzilumab, an investigational treatment candidate designed to combat cytokine storm. She pushed to be enrolled in the clinical trial.

The Unsung Heroes

“Patients participating in clinical trials are too often the unsung heroes in the development of innovative treatments and the pandemic has heightened the importance of patient advocacy in the clinical trial process,” said Cameron Durrant, MD, MBA, chief executive officer of Humanigen, the biopharmaceutical company developing lenzilumab as a potential COVID-19 treatment. “The global biopharmaceutical industry has made tremendous strides to develop viable COVID-19 vaccines, but we need treatment options for all patients while we wait for widespread safe and effective vaccine use and availability.”

For Michelle Vogel, her condition improved rapidly after participating in the clinical trial. She had read that lenzilumab could help normalize her C-reactive protein and that is what she experienced. Just days later, she was weaned off supplemental oxygen and discharged from the hospital.

“If I hadn’t pushed to see what other options were out there, I don’t know if the lenzilumab trial would’ve been offered,” she said. “Because of my professional experience as a patient advocate, I knew to keep on pushing to see what my options were. But there are many patients who don’t even know that they can ask what their treatment options are.”

Sadly, however, Michelle’s mom passed away from COVID-19 while Michelle was in her own COVID-19 battle. “I tried hard to advocate for the best treatments for my mom but there were no clinical trial options at her hospital and it’s difficult to even think about whether she would have survived if she had access to a clinical trial.”

To learn more and to find a lenzilumab clinical trial site near you, visit www.StopStorm.com.

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Advocacy Patient stories

IG Shortage is Coming

Walter “Kip” Caro started noticing tingling, numbness, and pain in his feet back in 2007. Gradually these symptoms progressed up his legs and into his body to the point where he had to be hospitalized because his torso was affected and he couldn’t breathe. It took three years for doctors to diagnose him with chronic inflammatory demyelinating polyneuropathy (CIDP). In that time, Kip suffered permanent nerve damage in his feet.

For the last 11 years, however, Kip has been treated with intravenous immune globulin (IVIG), which has worked like a charm. He never has to worry about his symptoms, unless he can’t get his treatments every three weeks. If he doesn’t, as happened last June when there was a shortage and his infusion center couldn’t get immune globulin (IG) products from their distributor, his disease starts to relapse.

“It was scary,” he says of this experience.

“Over the years, we’ve met people said they could make a change to their treatment schedule, and their symptoms would not be affected,” says Kip’s wife Bonnie Joslin. “But we’ve learned, for Kip, his body just can’t do that.”

Kip feels lucky that Bonnie is such an advocate for him. By the time his infusion center called and told him they weren’t able to get his medication, Bonnie had already been reading about other patients’ experiences online. She immediately contacted Michelle Vogel, who administers a Facebook page for people who depend on IG therapy. Michelle, who also serves as vice president for patient advocacy at CSI Pharmacy, was able to get Kip hooked up with home infusions within a week.

“He was already starting to relapse,” Bonnie says. “But Michelle pushed it through, and Natalie Edwards in the insurance department worked to get approval. I know it wasn’t easy, but it was like a miracle happened.”

By early 2021, however, nearly all IG manufacturers expect to see shortages again. This time it’s because pandemic shutdowns earlier in the spring have decreased plasma donations. Plasma is the raw material from which IG and other protein therapies are made. The manufacturing process takes seven to nine months to create IG from donated plasma, so unlike last year’s shortage, this time we know it’s coming and can be prepared.

When IG products are in short supply, rationing will happen, Michelle says. “When the market tightens and we have shortages, what tends to happen is that certain sites of care like hospitals may stop treating outpatients. If they do treat outpatients, they will limit treatment to primary immune deficiency, Kawasaki disease, and transplant patients.”

That’s because supplies of IG are not universally distributed. Hospitals and infusion centers have contracts with certain manufacturers or distributors, so they can only get specific brands. Last year, patients like Kip who were served by these sites of care sometimes found their treatments delayed or dropped completely.

Specialty pharmacies like CSI Pharmacy that offer home infusion services, however, have access to more brands of IVIG as well as subcutaneous immune globulin (SCIG). In 2019, CSI Pharmacy was able to work with patients, their healthcare providers, and their insurance plans to allow them to continue IG treatment in the home.

At times this meant switching to a different brand of IVIG when certain brands were not available. Some patients even switched to subcutaneous (meaning under the skin) infusion after being on the intravenous (IV) form.

While changing to a different product can be scary for patients who have confidence in a brand of IG that is working well for them, please be assured that  a switch can be relatively comfortable if you work closely with your doctor and pharmacist. The pharmacist can find a brand that is close in formulation to your current brand, for example. If side effects become a problem, the infusion rate can be reduced and pre-medications prescribed. 

If you depend on IG therapy, now is the time to prepare. We suggest you have a conversation with your provider and current site of care.

  • Ask your provider what other brands are good options if your brand is in short supply, and ask your care site if they are able to obtain one of these brands if needed.
  • Ask the site of care if they will prioritize patients if there is a shortage. Will it be based on disease, IgG levels, and/or rationing product by the number of grams or frequency?
  • Ask how the site of care handled past shortages.

If it turns out that your site of care does not prioritize patients with your disease or they are unable to obtain your brand of IG, you may want to consider how important is it to you that you continue IG therapy. (Some patients feel comfortable skipping their treatment for a month or two if it means they don’t have to make changes.)

If you’re like Kip, however, and can’t function without regular IG infusions, you’ll need to think about what options are available to enable you to access this treatment. Are you willing to switch brands? Are you willing to move to home infusion? Are you willing to switch to SCIG?

If home infusion becomes your alternative, we urge patients to make that move now. Specialty pharmacies are more flexible and were less affected during the last shortage, but that may not be the case this time.

If you are a patient of a specialty pharmacy when this crisis hits, you may be able to get access to IG since they do not ration based on disease and have access to more brands. However, we do not know how the shortage will affect sites of care. 

“We want to see all patients have access to all brands in their preferred site of care,” Michelle says. “But we just don’t know what the impact is going to be. We expect this shortage will be across all manufacturers. Patients should understand that this is not going to be a perfect scenario. You may still run into hurdles like access to your preferred brand.”

CSI Pharmacy’s patient advocates are available to discuss your options related to IG therapy, regardless of whether or not you are our patient. If we can’t service your needs, we will gladly help you find other resources. No patient should suffer alone.

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Health Information

Get Your Flu Shot Now

For some, getting a flu shot is just part of the fall routine every year. Immunization against the virus that causes influenza prevents millions of people from getting the disease, makes the disease less severe if you do get it, and can prevent death from severe disease and its complications. According to the Centers for Disease Control and Prevention (CDC), influenza causes an estimated 12,000 to 61,000 deaths every year in this country.

This year, because of the COVID-19 pandemic, physicians and public health officials say it’s even more important that everyone over the age of six months get a flu shot. This is especially important if you have an underlying health condition that makes you more at risk for developing complications if you get the flu. While the influenza vaccine will not prevent you from getting COVID-19 (researchers are still working on a vaccine for this different virus), it will help you avoid getting seriously sick with or—heaven forbid—dying from the flu. And the fewer people who get the flu, the more it saves healthcare resources that are still urgently needed for treating COVID-19 patients.

Those who are at high risk for flu complications are also at greater risk for getting COVID-19 and having serious outcomes. If you have an autoimmune condition or immune deficiency disease, such as myositis, myasthenia gravis, pemphigus and pemphigoid, chronic inflammatory demyelinating polyneuropathy (CIDP), and primary immunodeficiency (PI), this means you. You are at a much greater risk for getting sick with the flu and at greater risk for developing complications like pneumonia if you do. If you have heart disease, cancer, or diabetes, getting the flu can also make these conditions worse.

Getting the flu vaccine, however, is not a straightforward decision for some who have these conditions. For example, some people with a history of Guillain-Barré Syndrome (GBS) may have developed this form of muscle paralysis after receiving an influenza immunization. According to the GBS|CIDP Foundation International, the association between GBS and flu vaccines is inconclusive, but they suggest these individuals avoid the vaccine in the future. If you have had GBS, especially if it developed four to six weeks after getting a flu shot, you should talk with your doctor about the risks and benefits of getting the vaccine again.

Another consideration is the aerosol form of the flu vaccine. This is a live attenuated (weakened) influenza vaccine that is given through the nose. While the injected vaccines are made with inactivated virus, the nasal spray is made with live organisms that have been weakened but are still able to activate the body’s immune response against the disease.

The intranasal vaccine is not recommended for those younger than two, older than 50, or those who have a weakened immune system, including some patients who take immune suppressing medications. If you care for or live with someone who is immune compromised, you should also avoid the nasal spray. And if you have an underlying medical condition that puts you at risk for developing severe complications from the flu (such as chronic lung disease, heart disease, kidney disease, liver disorders, neurologic and neuromuscular disorders, blood disorders, and diabetes), it’s important to check with your physician before taking the nasal flu vaccine.

According to the Immune Deficiency Foundation, those with certain forms of immune deficiency (common variable immune deficiency [CVID], severe combined immune deficiency [SCID] or Bruton’s agammaglobulinemia) are unable to develop protective immunity following vaccination. This means their bodies don’t have the infrastructure to develop the immunity needed to keep them from getting sick, so vaccines will not do them any good. In fact, for individuals with these forms of PI, live vaccines—including the influenza nasal spray—may put them in danger of developing severe disease.

The only reason not to get a flu shot is if you have a severe, life-threatening allergy to the vaccine or any of its ingredients. This might include gelatin, certain antibiotics, or other ingredients. If you get hives when you eat eggs, studies have shown that most times you can still get the flu shot. If you have a more serious reaction to eggs or you are worried about this, you should of course talk with your doctor. Egg-free alternative vaccines are available.

The CDC has more information about seasonal influenza and how to prevent it.

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Clinical Trials for Rare Diseases

The idea of clinical trials is much in the news these days as medical researchers work hard to identify new treatments and vaccines for COVID-19. A clinical trial is a formal research study that, among other things, tests new therapies to see how well they work and if they are safe to use in humans. It’s an essential step in the Food and Drug Administration’s (FDA) process for approving new therapies.

For those who live with rare diseases, though, clinical drug trials have a more direct impact on their day-to-day lives. Many rare disease patients have few or no options when it comes to effective treatment of their symptoms. Even when medications with confirmed effectiveness are prescribed, they often are used without FDA approval (a practice known as off-label use), which sometimes makes it difficult to get insurance to cover the cost.

Getting new medications to market requires extensive testing in order to attain FDA approval. These studies need to have a sizable number of participants in order to show accurate results. That’s not a problem for common diseases like heart disease and cancer. But with so few people with diseases like myositis, myasthenia gravis, or pemphigus, it can be difficult to get enough qualified participants to show clear results.

That’s why many of the patient organizations that we work with encourage their members to consider taking part in a clinical trial if at all possible. This can be a big decision, one that you should discuss with family members and your doctor to be sure they are comfortable with your participation and the role they may need to play in the process. But it’s a great way feel like you are contributing to a cure.

Clinical trials are not limited to testing medications, but here we focus on clinical trials testing safety and effectiveness of new drugs. We’ve outlined answers to some common questions that can help you understand the general process of participation in such trials. Specific questions about a particular trial can be answered by the study coordinator of that trial.

How does a clinical trial work? Each clinical trial follows a strict set of rules outlining who can participate, what processes will be done, how participants are protected against risks, how long the trial is expected to last, and more. This protocol is designed to clearly answer specific research questions about the treatment. These protocols are outlined for all medications being tested in the US on the NIH website ClinicalTrials.gov. Here you can search for a trial based on disease, drug, location, or other criteria. Each trial also has contact information for additional information.

What is informed consent? All research participants have the right to know exactly what they are getting into. Informed consent is the process of providing you with essential information about the study before you decide to take part. Members of the research team will provide written and verbal explanation of the details of the study, including its purpose, how long it’s expected to last, tests or procedures that will be done as part of the research, and who to contact for further information. All known risks and potential benefits are also explained and included in the document. Before you decide to sign this document (which means you agree to participate) you should ask questions and be sure you understand everything included in it. It’s important to note that taking part in a clinical trial is voluntary, and you can leave the study at any time. 

Why do some patients not receive the treatment? Clinical trial protocols often involve comparing outcomes of patients who receive the new drug or treatment with those who are given a placebo (a non-active substitute). Most times neither the researcher nor the participant knows whether the drug or a placebo is being given. Participants are usually randomly assigned to the treatment or the placebo group. In this way, researchers can clearly see that the medication was responsible for the effect and not some other cause, such as participants getting better on their own.

What are “phases” of clinical trials? New medications and other treatments take place using a step-by-step process that begins in the laboratory (preclinical phases) then moves on to testing in humans. Each of these phases seeks answers to different questions.

These are the questions posed during each of the phases:

  • Phase 1 – Different doses of the drug are tested for the first time in a small group of healthy people—usually less than 100.
    • What are the side effects?
    • Is this drug safe to continue testing?
  • Phase 2 – The drug is tested in a larger group of people who have the disease—ideally a few hundred (but usually fewer in rare diseases).
    • What are the side effects in this population?
    • Does the drug work as treatment for this specific disease?
  • Phase 3 – The drug is tested in a much larger group of people who have the disease—ideally several hundred to several thousand.
    • What are the side effects?
    • How well does the drug work to treat the disease?
    • How much of the drug should people take?
    • How does the drug compare to currently available treatments?
  • Phase 4 – This phase happens after the drug is approved by the FDA and is being used by patients.
    • What are the long-term effects of the drug?
    • What is the best way to use this drug as treatment?
    • Are there other risks to using this drug?
    • Are there other benefits not previously identified?

How safe are these experiments? While all medical interventions carry some amount of risk, those who participate in clinical trials are not “guinea pigs.” The FDA requires researchers to include in their protocols certain measures to ensure the study is ethical, that the rights and welfare of participants are protected, and that the risks are reasonable when compared to potential benefits. And all research organizations require any protocol that involves human subjects to be reviewed, monitored, and approved by an independent Institutional Review Board (IRB) of scientists and ethicists.

You can learn more about clinical trials at the National Institutes of Health (NIH) website.

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Advocacy Patient stories

Disability Benefits in Danger

Andrea Williams had a panic attack when she opened her mail earlier this year. Her hands were shaking even before she tore into the envelope from the Social Security Administration (SSA). The letter inside informed her that, according to SSA, she was no longer disabled and would no longer receive disability benefits, including Social Security Disability Income (SSDI) and Medicare.

Andrea is one of thousands of American citizens who are unable to work because of severe, chronic health problems who can expect to receive these notices in the coming year. This is because SSA wants to cut $2.6 billion dollars from SSDI and its sister program Supplemental Security Income (SSI) by changing the rules they use to terminate people like Andrea, who have depended on the disability safety net for years.

Six years ago, Andrea was diagnosed with a rare, debilitating disease of the muscles called myositis. She had trouble lifting her head, she couldn’t pick up her newborn baby, she couldn’t climb stairs, she couldn’t even lift her arms enough to wash her hair. For months she went back and forth to the doctor saying, “I feel like I’m dying.” It wasn’t until she was referred to a specialist and ended up in the hospital for a week that she was finally diagnosed.

Myositis causes chronic pain, disabling weakness, and extreme fatigue. Treatment has helped Andrea, but she has lost too much muscle tissue that she will never regain. She can’t stand for any length of time, she drops things, and the brain fog from her medications makes it difficult for her to think. Her doctor told her she would never work again. When she applied for SSDI benefits in 2016, her application was accepted on the first try, which almost never happens.

“I’m scared to death,” she says. “My biggest fear is not having the medications, and I already can’t afford the doctors.”

An “Explanation of Determination” letter like the one Andrea received is a notice an SSDI or SSI recipient gets when they have been identified by SSA for “Continuing Disability Review” (CDR). This is the agency’s review process to see if beneficiaries are still medically eligible for the program. After this review, if SSA believes the person no longer meets their criteria for disability, their benefits are terminated.

A medical CDR is done at least once every three years, unless the SSA expects your medical condition to improve sooner. Those who have a medical condition that is not expected to improve undergo a CDR every seven years.

A rules change that took effect earlier this year, however, dramatically increases how often a person must undergo a CDR review. This change adds a new category for those whose condition is “likely to improve.” Hundreds of thousands of people now in less frequent CDR categories will be moved into this new category to be reviewed every two years.

This new rules change is especially disturbing because it targets people like Andrea who have chronic conditions that flare up unpredictably. Those who are approaching retirement age and those with mental illness are some of the other people this rules change aims to remove from benefits.

“If you get one of these letters, the first thing you need to do is notify Social Security that you want to appeal this decision,” says Michelle Vogel, CSI’s vice president for patient advocacy.

To appeal, you need to submit a Request for Reconsideration form within 60 days of receiving the notice of denial of benefits. If you want to continue receiving benefits while your case is being decided, you will need to submit this form withing 10 days of the denial, and you need to specifically ask that benefits continue. Be aware, however, that if your case is unsuccessful, SSA can require you to return the overpaid benefits.

Andrea has followed this advice and is awaiting a response from SSA. She is hopeful that, with the support of her doctors and her patient advocate, her appeal will be granted and she will once again be able to sleep at night.  

For others like her, Andrea offers this advice:

“You have to reach out and get some help from someone like a patient advocate,” she says. “You have to have your medicine. You have to see your doctors. You can’t take no for an answer.”

CSI Pharmacy has patient advocates who can help you navigate this and other health care access challenges. We offer this service regardless of whether you are a CSI Pharmacy patient.

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CSI Pharmacy’s Plasma Donor Superhero

Maddie was in high school when she developed juvenile dermatomyositis, a rare autoimmune disease of the muscles that made her so weak she couldn’t walk. Bill developed myasthenia gravis, another autoimmune neuromuscular disease, after having anesthesia for bypass surgery. Amanda and her daughter have immune deficiency diseases that make them susceptible to all kinds of infections. Immune globulin (IG) has been a life-saving therapy for all of these individuals.

IG is one of a number of treatments that can only be made from donated human plasma. This year, as a result of coronavirus restrictions, plasma therapeutics companies have experienced a significant drop in donations, which will limit supplies of plasma products such as IG by the end of the year. For patients, this is their worst fear. It means they may not be able to get the medications that allow them to live a normal life.

When Justin McNeill learned that plasma donations were down by as much as 40%, he thought of patients like Maddie, Bill, Amanda, and her daughter. Much of CSI Pharmacy’s business involves providing home infusion services for those who depend on IG therapy. As a delivery technician for CSI Pharmacy, it’s Justin’s job to pack up shipments of immune globulin and the supplies needed to administer it and make sure it all gets to the patient’s home in time for their infusion. 

In the spring, CSI Pharmacy joined the Immunoglobulin National Society in an effort to raise awareness about plasma donation and to inspire more healthy donors to contribute. As part of that effort, the company initiated an internal contest to encourage employees to become plasma donors. Justin was among the first to respond.

“We were told that with all the coronavirus restrictions, people aren’t donating plasma as much,” Justin says. “That means patients aren’t going to be able to get the medicine they need. I figured I’m able to give, so there’s no reason not to.”

Justin started donating in May and has given twice a week ever since—the maximum weekly donations allowed. To date, he has donated plasma 24 times. And even though he works full time and goes to school in the evenings, showing up at the BPL Plasma donation center is part of his weekly routine. He plans to keep on giving as long as they’ll let him.

Justin may have run away with this contest, but he’s not the only CSI Pharmacy employee to participate in the plasma donor drive. Eleven other members of the staff have also donated at least twice. (Regulations require two donations before the plasma can be used to make plasma protein therapies like IG.)

The rules governing who can qualify as a plasma donor are very strict. Justin, who is 24 years old and healthy, had no problem qualifying. When several other employees attempted to donate, however, they were turned away because they have chronic health conditions or other restrictions. This only made Justin more committed to continue donating.

“I knew a lot of the people here in the office couldn’t donate because of various health issues or medications, so I said, why not me?”

“Justin is very modest,” says James Sheets, CEO of CSI Pharmacy. “I know he doesn’t like to call attention to himself. But for us he is a superhero. We are pleased that our employees take this so seriously and are willing to donate plasma. And we’re extremely proud of Justin for his ongoing commitment to making plasma donation a part of his life.”

For Justin, it’s all about Maddie, Bill, Amanda and her daughter, and others for whom he packs up the products and supplies for their home infusions. He urges anyone who qualifies to consider becoming a plasma donor.

“We’re probably about to get hit with a really bad shortage of IG products,” he says. “Our patients need this medicine that’s made from human plasma. We’ve got a lot of people who are really sick and really need this medicine. Even donating just twice will help save lives. You can make a big difference.”

As the winner of CSI Pharmacy’s Plasma Donation Incentive Program, Justin McNeill was presented with a trophy and a monetary gift during a ceremony in September.

Find a plasma donation center near you.

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Be an Advocate

COVID-19 was the last thing on Michelle Vogel’s mind the day she raced to South Florida to care for her elderly mother who had fallen in her home. Her mom hadn’t been feeling well that week. She thought she had a urinary tract infection again. As it turned out, Judy Vogel had COVID pneumonia. She died a week later in ICU.

Michelle is the head of CSI Pharmacy’s Patient Advocacy team. Since the beginning of the pandemic, she has tirelessly insisted that everyone needs to wear a face mask, wash their hands or use hand sanitizer, and maintain social distancing. And she walks the talk. She knows that the lives of the immune compromised patients she cares for depend on this.

It never occurred to her, however, that she would need to protect herself from her own mother. Judy lived alone. She rarely left the house. And she took precautions. No one knows how she might have contracted this highly contagious condition, but she gave it to her daughter.

Five days after learning of her mom’s diagnosis, Michelle herself tested positive for COVID. As someone who lives with several rare, chronic conditions, she knew her chances of developing severe COVID pneumonia were high, and over the next few days she did become very sick.

“I’ve had migraine headaches, but they’re nothing compared to COVID headaches,” Michelle says. “And I’ve never been so tired. I’ve never had so much pain in my legs that they just give out. The coughing is worse than any bronchitis. And then it’s just odd to lose your smell and taste. My stomach, the diarrhea, the chills and fevers…it just hits every part of you. I even have skin lesions.”

With the support of daily telemedicine checks, Michelle battled the disease at home for a week. Her doctors started her on a corticosteroid (prednisone), which has been shown to reduce lung inflammation in COVID patients. They tried azithromycin (Z-Pak), which has antiviral properties, although it has not been reliably tested in COVID patients.

They also ordered cough suppressant medicine and a portable nebulizer that helped her to breathe in a bigger dose of medication to open her lung passages. From an online supplier, she ordered a pulse oximeter (a medical device that fits over your finger) to be sure her blood oxygen levels stayed adequate. Still, she ended up in the hospital with COVID pneumonia.

“There’s a lot doctors can’t see on a telemedicine call,” she says. “They don’t see you when you’re gasping for air. They can’t listen to your lungs to hear how congested you are. And how do you get a chest x-ray or labs drawn when you’re too exhausted to drag yourself out of the house?”

Michelle is the person many rare disease patients turn to for advice on navigating the maze that is our healthcare system, overcoming health insurance obstacles, and accessing the expensive therapies that keep them alive. She is an expert who loves sorting out these challenges.

So during the week she spent in the hospital, Michelle became her own advocate. She knew, for example, that remdesivir (an antiviral medication) and convalescent plasma (blood serum with antibodies from COVID patients who have recovered from the disease) had shown some positive results, so she requested these. She also asked about other treatments and was offered a clinical trial to test a new biological therapy.

While no one expects to come down with COVID—or any other disease, for that matter—Michelle’s experience shows the importance of educating yourself about whatever condition you find yourself burdened with. Know what drugs and therapies are used to treat the disease and ask if they might be right for you (or your loved one). And if you don’t understand what the doctors are saying, ask questions until you do.

“COVID-19 has affected all of us in one way or another,” Michelle says. “I have stayed vigilant in wearing my mask and isolating to stay safe. I never imagined that it would touch my family, take my mother, and leave me battling with COVID pneumonia. Please stay safe so this doesn’t happen to you or someone you love. And if it does, be an advocate for yourself or your loved one.”

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Can’t be Complacent with COVID

Judith Vogel had lovely hands, always perfectly manicured, and beautiful, beautiful eyes with long lashes. She taught fifth grade for most of her life in Montville, New Jersey and saved every letter her daughter Michelle wrote to her from camp as a child.

At 81, she lived alone, but she enjoyed a full social life: playing canasta and mahjong, going to movies, eating out. She had lots of friends. With this year’s pandemic restrictions, though, Judy lost all that. All she could do was sit at home and watch the news and fret. She was afraid she would get sick. And she worried about our country, all the hatred she saw. All the fear.

What she really wanted was to see her grandson get married (his wedding was postponed twice because of the pandemic). And on her birthday, November 3, she wanted to celebrate by voting for Joe Biden. He could turn things around in this country. She was convinced.

Judy will enjoy neither of these dreams. She passed away on August 20. She died, as she feared, of COVID-19.

It was a Thursday morning. Michelle hadn’t been able to get her mom on the phone for two days. She threw some things in the car and drove the 300 miles to South Florida. She made it in record time. On the way, she called her cousin who lived closer and asked her to go check. They found Judy on the floor. She had probably lain there for a full day.

When the paramedics arrived, they decided she was OK. She was conscious. She didn’t have a fever. She wasn’t coughing. They didn’t want to risk taking this elderly lady to the hospital where she might catch COVID.

When Michelle got there, her mom was sitting comfortably in a chair, sipping fluids. She made an appointment for Judy to be seen by her primary care provider the following morning. Judy had a history of urinary tract infections (UTIs). Michelle assumed that’s what was making her confused and fatigued. That and the fact that she’d lain on the floor for the last 24 hours.

Michelle, who serves as CSI Pharmacy’s Vice President of Patient Advocacy and Provider Relations, took her mom to the doctor and got her started on treatment. But that afternoon, when Judy was too weak to stand up from a chair, Michelle knew something more serious was going on.

She had to call 911. It was the only way to get her mom to the emergency room. But she couldn’t go with her; no one is allowed in hospitals these days except the patient. That evening when she called the hospital, she learned that Judy had tested positive for COVID. Judy was in ICU. Soon she would be on a ventilator. Michelle would never see her mom again.

“I was shocked,” Michelle says. “I thought she had a UTI. I didn’t put the symptoms together. I never thought of COVID.”

Looking back, Michelle realizes there were a lot of signs she missed when she talked to her mom every day. She thought, for example, that her mom’s decreased appetite was related to the isolation and depression Judy was feeling. But maybe she wasn’t eating because she couldn’t taste or smell. These are symptoms of COVID.

Judy complained of headaches and muscle aches, but she didn’t have a fever. She just thought she was coming down with a cold. When Michelle talked to her mom’s best friend, though, she said Judy had been coughing for weeks. Maybe she’d been sick for weeks, but no one realized it. Judy didn’t like to bother anyone.

The thought of her mother lying on the floor alone all day and all night before she was finally found will never leave Michelle. But even as she moves through her own grief, Michelle wants her family’s experience to serve as a lesson for others.

“Isolation is hard on everyone,” she says, “but it is especially difficult for our seniors. It affects us both physically and emotionally. It can be really horrible. But as much as people want to be more socially active and get back to their lives, this virus is going to go on for years. And the more complacent we are, the more severe it will be. We can’t assume COVID hasn’t affected anyone in our personal circle. We still need to take precautions. We need to be safe.”

Secondly, she wants people to be aware that COVID is a threat that is with us everywhere, and that coronavirus should be at the top of our minds at all times.

“We don’t really understand all the symptoms of COVID-19,” she says. “A lot of patients never present with a fever, but they have all these other symptoms: severe headaches, body pain, diarrhea, rashes, weakness, tingling toes…all kinds of things. We need to understand that there are many more symptoms than just the cough and fever that you always hear about. And if you are feeling bad, you need to get yourself to the doctor.”