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Hemophilia is a rare disorder that causes an individual’s blood to not clot properly, because of a lack of clotting proteins, causing excessive bleeding.

In patients with an extremely low level of clotting factor proteins, hemophilia can cause more serious issues and even be fatal.¹

The disease has 2 types: hemophilia A and B. Hemophilia A, which is more common, is a disease where there is a clotting factor VIII deficiency, whereas hemophilia B is deficient in factor IX. A rarer type, hemophilia C, is caused by low levels of factor XI. ²

Hemophilia A affects about 1 in 5000 male births or approximately 400 babies per year. An estimate of 33,000 males in the United States live with hemophilia, mostly type A, according to recent data from 2012 to 2018.³

The first article published on the disorder dates to 1803, when John Conrad Otto, MD, a physician from the Philadelphia, Pennsylvania, area, wrote about a rare, hemorrhagic bleeding disease that affected mostly men. Following this, in 1828, he and Friedrich Hopff developed the term “haemorrhaphilia,” which was shortened to “haemophilia.” In 1947, Alfredo Pavlovsky, a physician from Buenos Aires, discovered that hemophilia can be categorized into A and B. It was not until the 1920s that other factors of the disorder were discovered.⁴

Treating hemophilia properly depends on the kind of clotting factors that available. An ideal way to replace these clotting factors is through continuous replacement therapies, which use infusions to treat patients. Clotting factors can be made from donated blood or in the laboratory. These are also known as recombinant clotting factors.¹

Plasma-derived and recombinant clotting factors remain the most popular for patients. Some factor VIII recombinant products include Advate, Eloctate, Esperoct, NovoEight, and Xyntha. As for humanized factors, Alphanate, Hemlibra, and Hemofil M are just a few examples.⁵

Amicar, Desmopressin Acetate (DDAVP), and Stimate, are other medications that are similar to hormones produced in the body. Amicar can be administered intravenously or orally, while DDAVP and Stimate can be given intravenously or via a nasal spray.⁴

One of the most recent FDA-approved factors for hemophilia A and B is Sevenfact, or coagulation factor VIIA (recombinant), for use in individuals aged 12 years and older. Sevenfact used at 75 mcg/kg dosing, repeated every 3 hours until bleeding stops, has an active ingredient expressed in genetically engineered rabbits.

The demand for hemophilia drug treatment in Europe and North America has increased in recent years, amid increased awareness of the disease. Growth is seen continuing this year.⁶

 Mayo Clinic. August 7, 2020. Accessed June 17, 2021. https://www.mayoclinic.org/diseases-conditions/hemophilia/diagnosis-treatment/drc-20373333

 of hemophilia. Indiana Hemophilia & Thrombosis Center. 2021. Accessed June 17, 2021. 

Hemophilia B. National Organization of Rare Disorders. Accessed June 17, 2021. https://rarediseases.org/rare-diseases/hemophilia-b/

History. National Hemophilia Foundation. 2021. Accessed June 17, 2021. https://www.hemophilia.org/bleeding-disorders-a-z/overview/history

Products licensed in the US. National Hemophilia Foundation. 2021. Accessed June 17, 2021. https://www.hemophilia.org/healthcare-professionals/guidelines-on-care/products-licensed-in-the-us

 drugs market size 2021 is expected to progress at a CAGR of 6% and reach USD 15,830 million by the end of 2025. Intrado GlobeNewswire. May 17, 2021. Accessed June 17, 2021. https://www.globenewswire.com/en/news-release/2021/05/17/2230352/0/en/Hemophilia-Drugs-Market-Size-2021-Is-Expected-to-Progress-at-a-CAGR-of-6-and-Reach-USD-15-830-Million-by-the-end-of-2025.html

Articles

The demand for drug treatments in Europe and North American has increased in recent years, as awareness of the bleeding disorder rises.

News

Hematology

Treating Hemophilia Depends on Available Types of Clotting Factors

From June 22 to June 26, customers who come into a Walgreens pharmacy for a COVID-19 vaccination will receive $25 in Walgreens cash rewards that can be applied to any in-store purchases. The cash rewards incentive follows the announcement of 

President Biden’s National Month of Action

, which was established to support as many US adults as possible getting at least one vaccine shot by July 4.

The White House explained in a statement that the focus of the month is around actions that will make getting vaccinated even easier for individuals across the country. This goal looks to support not just mobilization efforts around vaccine outreach and education, but also incentives that may support more Americans pursuing vaccinations as well.

In Walgreens pharmacies, the offer of in-store cash rewards will be made available immediately following vaccination for those who already have myWalgreens accounts. For those who do not have this account or want to open one, they can obtain a $25 Walgreens gift card by calling 866-614-7029.

Additionally, the parents or guardians of eligible adolescents 12 to 15 years of age who get vaccinated will be eligible to receive the $25 Walgreens gift card on the minor’s behalf.

With this incentive, Walgreens is showing its support for the goals set by President Biden’s National Month of Action to incentivize more shots in arms. Also, in light of the potential for an increased demand of vaccinations due to the efforts of the organizations involved in the National Month of Action, 4000 Walgreens locations will be extending their pharmacy hours during Fridays in June in order to support additional flexibility for walk-in vaccinations.

Currently, Walgreens has more than 400 pharmacy locations that are open 24 hours where individuals can get vaccinated based on their schedules and availability. To learn more about these hours, interested individuals can visit the online store locator or call the Walgreens pharmacy nearest to them for information regarding hours of operation.

In pharmacies nationwide, COVID-19 vaccinations have been and continue to be widely accessible, with walk-in appointments available. At Walgreens pharmacies, individuals can schedule appointments, including same-day appointments, online or by calling 800-Walgreens, with options available for both English and Spanish language speakers.

Individuals Getting COVID-19 Vaccines at Walgreens to Receive $25 in Walgreens Cash Rewards. Deerfield, IL: Walgreens; June 22, 2021. 

https://news.walgreens.com/press-center/news/individuals-getting-covid-19-vaccines-at-walgreens-to-receive-25-in-walgreens-cash-rewards.htm

From Tuesday, June 22 to Saturday, June 26, customers getting a COVID-19 vaccination at Walgreens pharmacies will receive $25 in Walgreens cash rewards for in-store purchases.

Retail

Coronavirus

Walgreens Offers $25 In-store Cash Rewards for Individuals Who Get Vaccinated Against COVID-19 

Officials with the FDA have approved dabigatran etexilate (Pradaxa; Boehringer Ingelheim Pharmaceuticals, Inc) oral pellets for the treatment of children aged 3 months to less than 12 years of age with venous thromboembolism. The therapy is indicated for administration directly after these patients have been treated with a blood thinner given by injection for at least 5 days.

The FDA also approved dabigatran etexilate oral pellets for the prevention of recurrent clots in patients aged 3 months to less than 12 years of age who have completed treatment for their first venous thromboembolism. Additionally, dabigatran etexilate was approved in capsule form to treat blood clots in patients 8 years of age and older with venous thromboembolism directly after they have been treated with a blood thinner given by injection for at least 5 days, and to prevent recurrent clots in patients 8 years of age and older who have completed treatment for their first venous thromboembolism.

According to the FDA, dabigatran etexilate is the first approved medication for blood thinning that children can take orally. The only other approved blood thinning medication for children is given by injection.

Dabigatran etexilate was originally approved in 2010 to reduce the risk of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation.

According to the FDA, blood clots can pose a serious problem in children and adults. Children are most at risk for blood clots if they have cancer, congenital heart disease, a central venous catheter, or are admitted to an intensive care unit. Venous thromboembolism can lead to complications, including swelling and discomfort near the clot, chest pain, lung damage, anddeath.

The safety and efficacy of dabigatran etexilate as a treatment for blood clots in patients younger than 18 years of age was evaluated in a study of 267 pediatric patients. In this open-label study, patients were randomly assigned to receive either dabigatran etexilate or standard of care. The study compared the 2 groups for the number of patients who met the composite endpoint, having survived a blood clot, having their blood clots completely resolved, and having no additional blood clots. Results showed that 81 of the 177 people taking dabigatran etexilate met the composite endpoint compared to 38 of the 90 patients who received standard of care.

The safety of dabigatran etexilate to prevent recurrent blood clots in the same pediatric population was evaluated in an open-label, single-arm study in 214 patients with a history of blood clots. The primary endpoints of the study were recurrence of blood clots, major and minor bleeding events, and death, overall and related to blood clots. Recurrence of blood clots occurred in 3 of 214 patients, which was comparable to prior standard-of-care treatments.

The most common adverse effects (AEs) of dabigatran etexilate include digestive system symptoms and bleeding. This drug can cause serious and fatal bleeding, and it is not recommended for patients with bioprosthetic heart valves or triple-positive antiphospholipid syndrome. Additionally, dabigatran etexilate has a boxed warning cautioning that early treatment discontinuation may increase the risk of blood clots and that blood accumulation within parts of the spinal cord in patients undergoing spinal procedures may cause serious AEs.

FDA Approves First Oral Blood Thinning Medication for Children [news release]. June 21, 2021; FDA. Accessed June 22, 2021. 

https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-blood-thinning-medication-children

The FDA also approved dabigatran etexilate oral pellets for the prevention of recurrent clots in patients aged 3 months to less than 12 years of age who have completed treatment for their first venous thromboembolism. 

Hospital

First Oral Medication for Blood Thinning in Children Granted FDA Approval 

To recognize National PTSD Awareness Month in June, 

 interviewed Jordan Tishler, MD, president of the Association of Cannabis Specialists, faculty at Harvard Medical School, and a cannabis specialist at inhaleMD, on how medical cannabis can be used to treat post-traumatic stress disorder (PTSD) among veterans and the general public.

 So, what are some areas in which you see the medical community may benefit from further education on how medical cannabis can be used as a legitimate treatment?

Well, I think that the medical community at the moment has largely ignored this issue, which I think has been problematic politically because if there's any group that should be sort of leading the charge and making sure that this gets done well and safely, it's physicians.

For me personally, the reason I do this is purely based on my own super ego, my moral need to provide this kind of care for people. I actually worked for the VA for 15 years before doing this as an emergency room physician, and I left that stable environment to do this because I felt that once I had gained a certain amount of knowledge about this, that I needed to kind of go out there and provide that.

I think that the broader community needs to sort of acknowledge that they've been fed a line of hui about how reckless and irresponsible cannabis is, and to acknowledge that we've got 70 years worth of not bad data—it's not as good as we'd like it to be, but it's not bad—that supports its use for various treatments, and in acknowledging that, then they need to just simply understand what it's good for and for whom it would be helpful.

I speak to my physician colleagues on this topic several times a week—I'm doing one this afternoon, I'm doing one tomorrow morning, and I am universally greeted by engaged curiosity. My physician colleagues almost never say this is baloney or any of those sorts of things. What they say is wow there's so much to know, I'm really interested, thank you for teaching me about this, and I'm so glad there are some people like you doing this.

I think that we really have to think of this as a team effort and getting those primary frontline people to be aware and understand enough that they can understand not only the medicine but also the availability of specialists for whom they can refer—that sort of creates a workable dynamic.

 Absolutely, yeah. What is your outlook on the future of both care for patients with PTSD and acknowledgement of the role of cannabis in its treatment?

Well, I think that our country in particular, though I don't think it's limited to the United States, is certainly seeing an explosion of mental health issues, much of which pre-dated the pandemic, but certainly have been amplified by the pandemic.

We know that our treatments for a lot of these mental illnesses, like anxiety and PTSD, are adequate but not great, or something to that effect. So, we need to keep pushing forward on that research.

We need to acknowledge the mind-body connection that people with psychological illnesses may have medical manifestations, and perhaps equally if not more importantly, people with medical illness are going to have psychological manifestations, and we need to say think about the psychological health of people who say have major illnesses. If you have a heart attack and follow that by a bypass surgery, you can be sure that you're going to have a certain amount of rehab after that, and we need to start thinking a little bit more about what are the psychological rehabs that need to happen.

So, I think that we need to grow in terms of our country's view of psychological illness as well as medical and the way these things interface, and I think we're going to get there. The real question in my mind is are we going to put our money where our mouth is as a country.

Basically, are we going to really devote the kind of resources that are necessary to both research and also provide the care to the people who need it. If it were up to me, I would buy a whole lot fewer F-35s and fund a lot more mental health clinics.

But frankly speaking, unfortunately nobody's asking me. Well, you asked me, but nobody in Congress has asked me.

 Yeah, I wholeheartedly agree with that sentiment. What is the value of the pharmacist in supporting the care and treatment of patients with PTSD?

Wow. Look, I think the pharmacist is in a unique role, and I think that when the relationship between the physician and pharmacist is working well, I think it's an amazing process.

I have had some of the most engaging and interesting discussions with some of my pharmacist colleagues, so I think that there's a huge role that they play in this entire process.

Certainly, we know that patients ask pharmacists lots and lots of questions, and I think that pharmacists do a very good job of answering them, and also knowing when that question needs to be turfed back to the doctor, so I think that's phenomenal.

When we start to think about the cannabis landscape, the problem is that that kind of education, and I don't mean just sort of factual education, but sort of beyond factual into sort of the professionalism and collegiality, I think that—I didn't go to pharmacy school, so I can't speak to how it's taught—but pharmacists generally come out, it would seem knowing something about who physicians are and what they do and how that relationship is supposed to work, and I just don't see any of that in the cannabis world at this point.

In fact, unfortunately, the overriding message to the public seems to be oh forget about the doctor, just come to us and we'll tell you what to buy and how to use it, and I can tell you from both observing the population as well as the effects that that message has had on my own patient population, that that can lead to problems of overuse, to tolerance, and ultimately potentially to use disorders.

Obviously, we don't want people to come to harm from the medicine that we're giving them, so that's sort of a primary concern for me. It gets me right back into that discussion of having a binding prescription because if the prescription says for cannabis like it does for other medicines, sell 30 tablets or sell an eighth of an ounce of botanical or whatever, then that whole incentive to sort of upsell and side cell goes away, and we can really then be concentrated and confident that the patient is going to get what they need. Whether they use it though, that's a different question. But at least then that's a relationship question between that doc and that patient.

 Right, absolutely. Thank you so much for taking the time to speak with me today, Dr. Tishler.

Oh, it's my pleasure. Let me take 2 seconds here just to remind everybody that if they're looking for resources about cannabis medicine, they should visit the association of cannabinoid specialist website which is cannabis-specialist.org. Thank you again.

Videos

Jordan Tishler, MD, president of the Association of Cannabis Specialists and faculty at Harvard Medical School, discusses his outlook on the future of both care for patients with PTSD and acknowledgement of the role of cannabis in its treatment.

Mental Health

Expert: Medical Community Should Be Leading the Charge on Cannabis Treatment, Dispensing to Avoid Cannabis Overuse, Use Disorders

New research has found that the percentage of American adults with diabetes who achieved glycemic control worsened between 2007 and 2010, and again between 2015 and 2018, according to Johns Hopkins Bloomberg School of Public Health.

These findings emphasize the ongoing challenges of controlling diabetes, which the study authors said is one of the most prevalent health conditions. The team used data from an annual government-sponsored health study to evaluate trends in blood sugar control among adults with diabetes, as well as blood pressure and cholesterol control.

Type 2 diabetes is the most common form of the disease and is strongly related to diet and lifestyle factors. It impacts more than 13% of the adult population in the United States and increases the risks for other serious diseases, especially cardiovascular disease, according to the study. The traditional approaches to diabetes care are to reduce chronic high blood sugar, to keep blood pressure below hypertensive levels, and to control cholesterol.

According to the study, the proportion of adults with glycemic control improved between 1999 and 2007, but then dropped from 57.4% to 50.5% between 2007 and 2018. The study authors also observed a decrease in the proportion of people achieving blood pressure control, whereas the proportion achieving cholesterol control leveled off.

“These are concerning findings,” said senior author Elizabeth Selvin, PhD, MPH, a professor in the Bloomberg School’s Department of Epidemiology, in the press release. “There has been a real decline in glycemic control from a decade ago, and overall, only a small proportion of people with diabetes are simultaneously meeting the key goals of glycemic control, blood pressure control, and control of high cholesterol.”

For their study, the investigators had access to data that included interviews and clinical exams of approximately 5000 people across the country. The sample consisted of 6653 participants in the surveys from 1999 to 2018 who were at least 20 years of age, not pregnant, and reported having been diagnosed with diabetes outside of pregnancy.

Between 1999 and 2010, the percentage of respondents achieving glycemic control, defined as HbA1c levels below 7.0%, rose from 44% to 57.4%. That percentage then decreased sharply between 2015 and 2018. Similarly, the percentage achieving blood pressure control increased steadily from 64% between 1999 and 2002 to 74.2% between 2011 and 2014, then dropped to 70.4% between 2015 and 2018.

Finally, the percentage of people with diabetes who controlled non­–high-density lipoprotein (non-HDL) cholesterol rose from 25.3% between 1999 and 2002 to 52.3% between 2007 and 2010, but then reached only 55.7% between 2015 and 2018. The proportion of people achieving control of all 3 risk factors rose from 9% between 1999 and 2002 to 24.9% between 2007 and 2010, then slipped to 22.2% between 2015 and 2018.

“These trends are a wake-up call, since they mean that millions of Americans with diabetes are at higher risk for major complications,” said lead study author Michael Fang, PhD, a postdoctoral fellow at the Bloomberg School, in the press release. “Our study suggests that worsening control of diabetes may already be having a detrimental effect at the national level.”

Furthermore, the findings suggest that something changed since 2010 to slow progress in controlling these risk factors for diabetes. In the press release, Selvin said 2 large clinical trials published in 2008 may suggest a reason for this slowed progress. The trials found that intensive lowering of HbA1c to very low levels did not show the cardiovascular benefits that people had hoped for, and some trial participants saw increased risks of hypoglycemia.

“As a result of these trials, what we may be seeing is that doctors of people with diabetes may have backed off a bit on glycemic control, with potentially damaging results,” Selvin said in the press release.

She noted that since those results were published in 2008, many new and improved diabetes drugs have entered the market, allowing HbA1c to be lowered without causing hypoglycemia.

Major Study of Diabetes Trends Shows Americans’ Blood Sugar Control is Getting Worse [news release]. Johns Hopkins Bloomberg School of Public Health. June 9, 2021. Accessed June 22, 2021. 

https://www.jhsph.edu/news/news-releases/2021/major-study-of-diabetes-trends-shows-americans-blood-sugar-control-is-getting-worse.html

Researchers noted decreases in control of blood pressure as well as non¬–high-density lipoprotein cholesterol, both of which are risk factors for diabetes. 

Diabetes

Weight Management

Study: Blood Sugar Control is Worsening Among Americans With Diabetes

discussion, Suzanne Soliman, PharmD, BCMAS, and Sally Arif, PharmD, BCPS, BCCP, address how the COVID-19 pandemic has impacted women in the pharmacy field.

I know women make up 60% of our workforce. Another issue that I think often comes up in our profession is that women only make up 35% of leadership positions in pharmacy and that's even pre-pandemic been something that's been a limit for women.

Often, women are kind of straddling the taking care of children as they advance in their careers. They're still taking care of kids, but now even the additional responsibility of maybe caring for elderly parents, causing them to be overlooked for C-suite positions as leaders.

Do you have some advice for women with regards to how to get that support so they can move up in the profession as well?

 I think that you hit the nail on the head. When I've presented on this, I've seen that for the past 40 years, women have been the majority of graduates in our profession, yet there hasn't been any change in leadership. The leadership numbers have remained the same for the past 40 years.

The other interesting fact is that mid-career-wise, about 36% of pharmacists are working part-time who are female between the ages of 30 and 45. This shows really what they're doing between the ages of 30 and 45 is probably having their children and raising their families. That particular study showed that these women were all working less than 40 hours a week.

So, I think that this has been an issue, for me at least personally, and for a lot of other women, is when you're raising your family, how do you manage and what do you do? I think you can get creative, and there are opportunities where you can job share. I've done that before as well, where I'm sharing a position, an academic position, with a colleague who is also a mom, and we both kind of work part-time and build in a full-time role.

There are other ways to get very creative. I think having mentors and people who can guide you, for me that's always been important. Some of my greatest mentors were also women who had similar personal paths to mine as well and helped guide me along the way, and I think that having a mentor or a sponsor is very important as well.

I think knowing that it's okay to take a step back for a while, if that's what you choose to do, and it's okay not to as well. There are many women who decide to go a different route, and they decide to jump into leadership early on in their career, stay in leadership, and continue that path. So, I think knowing that it's okay to do either one is fine. But I think we really need to support one another a little bit more, to help one another in those positions.

Yeah, thank you so much. I think everything you just said is so important. It's this idea of rising up but also lifting up as women. I didn't even know the difference between mentors and sponsors until mid into my career where I've had mentors that have helped me with my career choices or decisions, but having a sponsor, I think especially for women of color or minority status that often don't have someone advocating for them, and that could be a man or someone that looks very different than you that is in a leadership position and is intentionally using their privilege and power to help advance women in the profession into positions that they are very well suited for but often overlooked for because of the presenteeism not being there, although productivity and being present are very different things, and we struggle with that in pharmacy.

 I think women get hesitant. I know personally speaking, I went to a job interview when I was 7 months pregnant and my stomach was like out there, and you could tell, and I was so scared.

I remember thinking I'm going to be overlooked for this position—it was a full dean position, and I said I'm going to be overlooked because of the size of my stomach, but I'm going to put my suit on, and I'm going to go and take that chance, and I think that sometimes we have to still take the chance.

But I love the idea of a sponsor. I've seen it become very successful, and I think that it's extremely important if you can have a sponsor, that's a great way to help improve the status of women in pharmacy.

Yeah, and I think for women everywhere to just ask as we find things that we might need, or resources or people to help support us in our careers is to ask and see. Often, I've said hesitancy is something we're socialized to kind of do because we don't want to rock the boat or seem like we're too needy, but I think we as women definitely have to get to a place where we can empower each other to say it's okay to ask for what your needs are. Often, I've been surprised who's come out at different points in my career to help kind of support me.

So, all women out there in the pharmacy profession—not to say that we don't have struggles and things that we deal with day to day—but if you need some time off for your mental health, or you need a proper break because you’re a mom that just came back and is nursing and needs a lactation room at your facility, you should be asking for these things, and policies in organizations should be very present in their inclusive language around supporting women in the workplace.

 Definitely, and I think that women are also not represented in different areas of pharmacy. So, independent pharmacies, they're primarily owned by men. So, there are different areas—even managers, so pharmacy managers at chains are typically male.

So, I think that establishing ground rules and maybe even talking about some of these numbers and letting women become more aware of it, that it is possible, and talking more about it I think can just help open the path as well.

It's really important I think even myself owning a pharmacy, it never came into my head as a pharmacy student because I was like there's so many hours and now I've seen women who own their pharmacy, they're bringing their babies to the back, they're nursing their babies there, and they're living these great lives. I think that there are so many opportunities for women in various areas of pharmacy and sometimes they're overlooked because we have an assumption.

So, the other thing that I wanted to mention too when you're talking about the burnout and stress is, right now, it's higher than ever. I know that a lot of people are feeling a little bit better, and that COVID-19 is getting better with the vaccines. But there's a lot of concerns still related to fertility, related to breastfeeding, related to not vaccinating your children for COVId-19, so I think that we're still not out of the woods yet. So, I think there are still a lot more topics that we need to discuss and bring to the forefront.

I agree I think we're always learning, and I think as women we can leverage some of our empathy because we can understand these concerns that our female patients specifically are having out there around some of the topics you just mentioned, and bringing our whole selves into conversations when we're counseling, explaining as a mom, I totally understand your concerns as someone who is trying to get pregnant, I've been there as well.

So, these types of things, I think we talk about empathy a lot, and I think as women we do have that ability to connect with so many of our patients because of our lived experiences.

So, I'm excited to continue talking with you about a lot of these issues as we kind of think about solutions and think about ways to navigate them. There is no easy answer, but I think just knowing, like what we've said today, that we are here for each other and that no one's really alone in their struggle, but that there's optimism, as we've said, as we're kind of getting hopefully out of the pandemic and more people are getting vaccinated.

 Yeah, definitely. I think we have to remain optimistic. I think that that's critical.

I don't know if you saw there was a study—and if anyone listening does research, please contact me, you can use the Pharmacist Moms Group as the number—but there was a study showing women physicians had more empathy, and they displayed more empathy. They spent more time with their patients. I would love to see anything related to pharmacy in that area as well. I'm not sure if there is anything but there's definitely some information out there in medicine on that.

Absolutely, I think you just touched on a great point with scholarship. I think that granted, our time is limited often, but getting some scholarship work around gender differences in the workplace and how that impacts patient care and health outcomes is really important for us because we can learn from that. I think as evidence-based individuals, we often want to see some of that data to kind of support what we anecdotally kind of feel might be happening in our practice.

 Yeah, definitely. Well, thanks Sally for all of this. It was great discussion, and I think we can talk for hours about this topic.

Suzanne Soliman, PharmD, BCMAS, and Sally Arif, PharmD, BCPS, BCCP, discuss how the COVID-19 pandemic has impacted women in the pharmacy field.

Women's Health

Expert: Although Women Are the Majority of Pharmacy Graduates, the ‘Leadership Numbers Have Remained the Same for the Past 40 Years’

A common industry practice of using rebates to reduce drug costs for insurers has sparked concern among federal agencies that out-of-pockets costs for patients and list prices have increased to a new level, according to a study from The University of Washington.

A research team from the university wanted to further investigate whether patients with or without insurance were paying more because of rebates to insurers. After examining the cost and price data on more than 400 branded drugs, the results showed that rebates were closely related to increases in out-of-pocket costs for patients by approximately $6 for those with commercial insurance, $13 for Medicare patients, and $39 for the uninsured, according to the study.

“We know that list prices have been increasing quite dramatically as have rebates, but no one has looked into the association between rebates and out-of-pocket costs,” said study lead author Kai Yeung, in the press release. “Increases in out-of-pocket costs are associated with rebates, however rebates also help keep premium costs down.”

The researchers looked at data on 444 branded drugs without generic equivalents from national datasets, including health care costs and drug prices from 2007 to 2018, such as the federal Medical Expenditure Panel Survey and SSR Health.

The team noted that the cost increases can influence patient health, since a higher cost can cause patients to take their medications less frequently, leading to more visits to the emergency department and increased hospitalization. Individuals analyzed in this study who did not have insurance had the poorest health, in addition to those with lower incomes who were less likely to take medication as prescribed when costs increased, according to the researchers.

The study authors suggest that future research and policies should focus on disassociating list prices from what patients pay out of pocket.

“The biggest takeaway is understanding that the rebates work to reduce the cost of prescription drugs for insurance companies and may reduce premiums,” said co-author Anirban Basu, in the press release. “And while it’s unclear how much the discounts are reducing premiums, they are definitely not translating to lower out-of-pocket costs for the patients who are using the treatment because of this structure of coinsurance and copayments tied to the list price.”

Drug rebates for insurers tied to higher costs for patients, especially the uninsured. University of Washington. Published June 15, 2021. Accessed June 16, 2021. 

https://www.washington.edu/news/2021/06/15/drug-rebates-for-insurers-tied-to-higher-costs-for-patients-especially-the-uninsured/

A research team from the university wanted to further investigate whether patients with or without insurance were paying more because of rebates to insurers. 

Reimbursement

Higher Drug Costs for Patients Tied to Rebates for Insurers

Follow-up results from the ZUMA-5 trial of axicabtagene ciloleucel (Yescarta, Gilead), the first and only chimeric antigen receptor T-cell therapy approved in patients with relapsed or refractory indolent follicular lymphoma (FL), shows that 94% of patients had achieved a response, according to a Gilead press release.

In an analysis comparing ZUMA-5 patients with a minimum of 18 months follow-up with those observed in SCHOLAR-5, axicabtagene ciloleucel showed a superior overall survival (OS) and progression-free survival (PFS) over treatments that are currently available.

“Follicular lymphoma is one of the most common non-Hodgkin lymphomas, and patients can experience frequent relapses, which quickly leaves us short of treatment options,” said study author, professor John Gribben, in the press release. “We are very encouraged by these data that suggest a significant and sustained survival benefit with Yescarta even after multiple rounds of prior treatment.”

The score analysis compared follow-up data from the phase 2 ZUMA-5 study to a weighted sample from the SCHOLAR-5 external control cohort of current therapies balanced for patient characteristics through propensity scoring. In the ZUMA-5 cohort, 94% of patients achieved a response compared to 50% of patients in the control cohort with an odds ratio of 16.2.

Axicabtagene ciloleucel further demonstrated a 58% reduction in the risk of death and 70% reduction in the risk of disease progression, relapse, or death versus the current therapies in the control cohort, according to the study authors.

“In an indolent disease like follicular lymphoma, longer-term data that demonstrate durable responses are critical. After a patient with follicular lymphoma relapses, the duration of response shortens with each new therapy,” said Caron A. Jacobson, MD, MMSc, medical director, Immune Effector Cell Therapy Program, Dana-Farber Cancer Institute and Assistant professor of Medicine, Harvard Medical School, in the press release. “The continued durable benefit demonstrated by axicabtagene ciloleucel at nearly two years is exciting, and the substantial survival benefit over current therapies that we’re seeing in the SCHOLAR-5 analysis is encouraging. These follow-up data reinforce axicabtagene ciloleucel as an important advance for a group of patients who have historically had few options.”

Axicabtagene ciloleucel received accelerated approval

 from the FDA for the treatment of adult patients with relapsed or refractory follicular lymphoma after 2 or more lines of systemic therapy in March 2021.

Longer-term Data for Kite’s Yescarta® in Relapsed or Refractory Follicular Lymphoma Demonstrate Substantial Survival Improvement Over Current Therapies in Comparative Analysis. Gilead. Published June 12, 2021. Accessed June 16, 2021. 

https://www.gilead.com/news-and-press/press-room/press-releases/2021/6/longer-term-data-for-kites-yescarta-in-relapsed-or-refractory-follicular-lymphoma-demonstrate-substantial-survival-improvement-over-current-therapie

 In the ZUMA-5 cohort, 94% of patients achieved a response compared to 50% of patients in the control cohort with an odds ratio of 16.2.

Specialty Pharmacy

Oncology

Longer-Term Data for Axicabtagene Ciloleucel in Relapsed/Refractory Follicular Lymphoma Show Substantial Survival Improvement Over Current Therapies

A recent analysis of patients with obstructive sleep apnea (OSA) found that moderate to severe OSA with no continuous positive airway pressure (CPAP) use is associated with increased cardiovascular disease (CVD) incidence, whereas OSA with CPAP use was associated with decreased CVD incidence relative to no CPAP use, according to an American Academy of Sleep Medicine study.

"Our study contributes to understanding the role of CPAP therapy for cardiovascular risk prevention," said lead author Diego R. Mazzotti, MD, in a press release. "We found the effects of CPAP were stronger in patients with moderate to severe sleep apnea, as well as in patients who used CPAP, on average, greater than 4 hours per night."

The research team analyzed adult patients with available-hypopnea index (AHI) between January 2018 and February 2020 in Kaiser Permanente Southern California. Eligible participants at baseline had more than 1 year of continuous insurance coverage, allowing gaps less than 90 days, and were free of CVD 1 year prior to OSA diagnosis. Each participant was separated between 3 groups: no OSA, OSA with any CPAP use, and OSA without evidence of CPAP use.

For this study, CVD incidence was defined as first occurrence of myocardial infarction, stroke, unstable angina, heart failure, or CVD death, based on validated HER algorithms. The team used Cox proportional hazards models to assess the association between OSA with or without CPAP use and CVD incidence, adjusted for baseline age, sex, body mass index, race/ethnicity, Charlson comorbidity index, and use of anti-hypertensives and lipid-lowering medications.

The study included 11,145 patients without OSA, 13,898 with OSA and CPAP use, and 20,884 patients without CPAP use. In adjusted models, moderate-severe OSA without CPAP use was associated with increased CVD incidence compared to no OSA. Further, OSA with any CPAP use was associated with lower CVD incidence compared to OSA patients with no CPAP use, whereas stronger effects were observed when restricting the sample to moderate-severe OSA, according to the study.

"Our study, while observational, suggests that clinical trials focused on understanding how to sustain long-term CPAP adherence in obstructive sleep apnea patients are necessary and could be critical for optimizing comorbidity risk reduction," Mazzotti said in the press release.

Mazzotti D, Chen A, An J, et al. 439 Continuous Positive Airway Pressure and Cardiovascular Risk in a Large Clinical Sample of Obstructive Sleep Apnea Patients, Sleep. 2021; 44 (2): A173–A174. 

https://doi.org/10.1093/sleep/zsab072.438

Treating sleep apnea with CPAP therapy is associated with lower risk of heart problems. EurekAlert! Published June 9, 2021. Accessed June 14, 2021. 

https://www.eurekalert.org/pub_releases/2021-06/aaos-tsa060921.php

Eligible participants at baseline had more than 1 year of continuous insurance coverage, allowing gaps less than 90 days, and were free of CVD 1 year prior to OSA diagnosis.

Sleep

Cardiovascular Health

Study Finds Treating Sleep Apnea with CPAP Therapy is Associated with Lower Risk of Heart Problems

Everyone has neutralizing antibodies (NAbs).

NAbs defend our cells against pathogens by diminishing their infectivity. While binding antibodies attach to a pathogen and activate immune cells to destroy it, NAbs simply inhibit the pathogen’s biological effect.

Scientists use NAbs in both active and passive immunization. Vaccines are the most common active immunization example. Serum therapy is a passive immunization treatment that involves administering a recovered patient’s blood serum to an infected patient. Recently, investigators have been studying NAb-based therapies to treat and prevent COVID-19.

Most NAbs are very specific for one pathogen. Consequently, pathogens that frequently mutate, like HIV, pose a challenge. However, broadly neutralizing antibodies (bNAbs) are a potential solution that can inactive multiple strains of a virus. Currently, investigators have found bNAbs for HIV, hepatitis C, and influenza, among others.

 investigated if a bNAb (VRC01) can prevent HIV-1 infection. One trial enrolled 2699 at-risk cisgender men and transgender individuals in the Americas and Europe, and a second trial enrolled at-risk women in sub-Saharan Africa. Investigators randomized patients to receive either:

The investigators looked at 2 things: acquisition and prophylaxis of infection. Participants received 10 infusions in total and underwent HIV-1 testing every 4 weeks. The results concluded that VRC01 did not prevent HIV-1 acquisition. However, VRC01-sensitive HIV-1 isolate analyses suggest bnAb prophylaxis can be effective. The investigators assert that virus strain’s susceptibility to VRC01 was an important factor in antiviral activity. VRC01 only effectively inhibited 30% of the virus strains in the trial regions but provided 75% protection in participants with VRC01-sensitive strains.

While VRC01 did not prevent HIV-1 acquisition, the study demonstrated that preventing HIV infection with bNAbs is feasible. Researchers are currently conducting early phase clinical trials on various bnAbs, including combination products. If these new bnAbs are effective, they can be used to prevent HIV infections and save countless of lives.

 is a 2022 PharmD candidate at the University of Connecticut in Storrs.

Corey L, Gilbert PB, Juraska M, et al. Two Randomized Trials of Neutralizing Antibodies to Prevent HIV-1 Acquisition. 

. 2021;384(11):1003-1014. doi:10.1056/NEJMoa2031738

Investigators have found bNAbs for HIV, hepatitis C, and influenza, among others. 

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