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Fighting cystic fibrosisAntimicrobial Resistance by: Katherine Black 10 Dec 2024, 3:18 pm
Antimicrobial Resistance
Written by: Katherine Black
Imagine you become sick with an ear infection, a common experience for many people, but when you go to the doctor they tell you not only that your infection’s bacteria is resistant to the typical antibiotic prescribed, but also that there are no other options available to treat your infection. This terrifying scenario has become a reality for many, and will continue to do so unless Congress takes action to fight against antimicrobial resistant infections.
Antimicrobial resistance occurs when the bacteria that cause infections evolve from exposure to antibiotics, causing the antibiotics to no longer work. The combination of a high use of antibiotics and a low development of new antibiotics creates a frightening reality for Americans. 35,000 people in the United States die each year due to an antimicrobial resistant infection. In fact, antimicrobial resistance kills one person in the United States every fifteen minutes. The World Health Organization has “declared antibiotic resistance as one of the top ten global health threats”. No one is safe from the threat of antimicrobial resistance. One could develop an antimicrobial resistant infection from something as common as an ear infection or a c-section. We never know when we may culture an infection that makes us a part of these startling statistics, making it crucial that we have options available for when that time may come. Unfortunately, many people with rare diseases or chronic illnesses face a heightened risk for culturing antimicrobial resistant infections.
My older sister, Lauren, was diagnosed with cystic fibrosis, or CF, at 2 weeks old. Cystic fibrosis causes a thick sticky mucus to form in the lungs, creating a breeding ground for bacteria. This leads to people living with CF to frequently develop infections and utilize antibiotics at a high rate, meaning they are also exhausting their antibiotic options due to resistance. The bacteria gains frequent exposure to these antibiotics, allowing it to learn how to resist them. When Lauren was in second grade she went on the IV antibiotic Vancomycin for the first time to treat her MRSA infection. In eighth grade, her doctors had to increase her dose because the MRSA had adapted to the drug and was no longer being treated by it. In tenth grade, Lauren’s MRSA became resistant again as the bacteria had evolved to combat the higher dose. Since there is a lack of development of new antibiotics, there are no options that remotely work to treat Lauren’s MRSA besides Vancomycin. Due to this, she now has to go on a very high dose of the drug, which her body oftentimes cannot tolerate leading to heightened levels of the drug in her bloodstream. No one should be forced to put themselves in dangerous situations in order to stay healthy. I constantly fear for my sister’s future and what treatment options will be available for her as she continues to struggle with her health and is in need of new antibiotics. It terrifies me to think that I could lose my sister to an infection that could have been treated had there been proper funding. This issue persists because our current antibiotic market is failing.
The deaths and fears Americans face as a result of antimicrobial resistance make it clear that new antibiotics are needed. The current state of our antibiotic market does not allow for companies to survive through development or incentivize new companies to enter the space. As Gunnar Esiason stated, “the only way to curb the spread of resistance is limiting antibiotic use” (Esiason, 2022). However, when less antibiotics are prescribed, the companies who make them lose money. As a result, companies are forced to drop out of the market.
Our country needs a new system in order to allow for the development of new antibiotics, a necessity to saving lives in America. The PASTEUR Act would allow for just that. The PASTEUR Act “would establish a subscription-style model which would offer antibiotic developers an upfront payment in exchange for access to their antibiotics, encouraging innovation and ensuring our healthcare system is prepared to treat resistant infections” (Bennet, 2023). The act would support FDA-approved drugs only, but it would be based on value, not sales volume. This would provide drug companies an incentive to develop new antibiotics as there would be foreseeable profit in the space. Change is undoubtedly necessary in the antibiotic market in America. We cannot expect companies to enter a market in which they know they will lose money, but we also cannot leave Americans without treatment options. The bacteria that cause these infections have developed and evolved over time, but our antibiotic market seems to have only moved backwards. The PASTEUR Act would be a catalyst in saving the lives of many Americans.
With the high number of deaths caused by the growing issue of antimicrobial resistance, it is imperative for Congress to pass the PASTEUR Act in order to incentivize the development of novel antibiotics in America. This issue only grows as antibiotic use continues. We cannot wait until it is too late. When lives are at stake it is important and necessary for our government to take action.
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Incentivizing Innovation: A Better Approach to Drug Affordability by: Mac Rung 10 Dec 2024, 3:09 pm
Incentivizing Innovation: A Better Approach to Drug Affordability
Written by: Mac Rung
If you take a look at the world’s leading pharmaceutical companies, even among the vast diversity of products a striking commonality emerges: the majority are American. Despite this, public sentiment towards pharmaceuticals seems to veer on the side of skepticism, leading to anger at corporations for prioritizing profits over public health, and calls to reform the dynamics of the industry as a whole. The anger is reasonable – financial stability and health should not be mutually exclusive – but often misdirected at the pharmaceutical industry. Instead of placing emphasis on cost reductions through price controls, advocates should prioritize lowering costs through insurance reform that benefits patients directly.
Take it from me. I was born with Cystic Fibrosis in the year 2000, when very little treatment was available to address the disease. I grew up healthy by CF standards, putting heavy emphasis on my treatment and training my lungs through exercise. Throughout lower school, I struggled to gain weight and eventually saw my lung function begin to gradually decline. It was this – the progressive nature of the disease – that always caused my family to worry about my next steps, while planning for a future that was never certain.
By some measure of luck, I was born at the right time. In the same year, a small start-up biotechnology company began tinkering with compounds it envisioned would fundamentally change the course of CF treatments – a vision shared by the Cystic Fibrosis Foundation to a tune of forty million dollars. While I was taking pills and inhaling treatments, Vertex Pharmaceuticals was executing studies and devising plans to bring their investment to fruition. Fast forward to 2024, I can say with unquestionable certainty that investment paid off in a big way, for the company and the patient population. As I was preparing to leave for college – at a time when my lung function could have continued to decline – Trikafta became available, parting the clouds that once obscured my future.
While inspiring, my journey should not be unique. In the United States, approximately 25 million people suffer from rare disease, with only 5% having FDA approved treatments¹. Government’s role in this aspect of the pharmaceutical industry should be to create the atmospherics of prosperity – allowing innovation to thrive and patients to benefit. Unfortunately, recent actions have moved the needle in the opposite direction.
Certain states have recently taken action to implement prescription drug affordability review boards (PDABs) to assess and address the affordability of prescription drugs. While well-meaning in nature, these boards tend to incorrectly address the value drugs have on patients’ lives. They fail to take into account the lifetime of hospitalizations, ER visits, and further medical bills that are avoided with effective treatment. Moreso, effective treatment brings value in the form of relief to family, economic productivity, and the aspirations of a newly functional patient that’s no longer inhibited by health from becoming a productive member of society. Not to mention the daily quality of life changes that occur for the patient – changes most would describe as priceless. No health technology assessment or complex pricing model can quantify that.
Furthermore, implementing price controls on a drug before its exclusivity window is up will have a drastic effect on the profit the company reaps, while decreasing accessibility for the patients that need it most. In 2017, 40% of retail prescription drug spending came from Medicare and Medicaid². If a drug is price controlled and therefore lowered in one state, this would have a cascading effect on what Medicaid plans pay for the drug across the country, which is 23.1% lower than the nationwide lowest price³. Rather than take the revenue hit, pharmaceutical companies will opt to pull the drug from the state to avoid a perilous drop in Medicaid revenue. This is a dangerous path to go down, at best trading access for affordability. States should leave drugs within their patent period out of pricing discussions and let the system run its course. This results in cheap and accessible generics for the patient population, and an incentivized company bent on diversifying profits into other drug candidates.
Recently, the passage of the Inflation Reduction Act aims to act on some of the frustration Americans have with the pharmaceutical industry. While many people will save money on Medicare plans due to caps on certain out of pocket costs, there are concerning elements of the act that will hinder future drug discovery initiatives by stacking the odds even higher against drug developers. The act makes small molecule drugs – such as Trikafta – eligible for government price negotiation at only 7 years post launch, with controls going into effect 2 years later. By some estimates, this will nearly cut profits in half, with 52% of total drug sales happening in year 10-13 of the traditional ~14 year patent period⁴. Laws treating profits as an obstacle to affordability make small molecule drug development nearly uninvestable, hindering the innovation that has long been a hallmark of our country.
There are two separate legislative measures that would avoid the current consequences the IRA is having on early stage pharmaceuticals. The first is to pass the Ensuring Pathway to Innovate Cures Act (EPIC Act). This legislation aligns the price negotiation timeline for small molecule products with that of biologics, beginning 13 years post market entry. This removes the “small molecule penalty” that currently discourages innovation in the drug class due to the shorter profit window compared to biologics.
The second measure is to pass the Optimizing Research Progress Hope and New Cures Act (ORPHAN Cures Act). Under the current IRA conditions, a drug is only exempt from price negotiations if it has an orphan drug designation for a singular condition. Many drugs, specifically cancer drugs, are designed to pursue multiple orphan designations (e.g., multiple types of cancer). Companies should be incentivized to pursue as many orphan designations as possible without losing exemption status. This legislation exempts all orphan drugs, such as Trikafta, from price negotiations, even if the drug is indicated for multiple diseases.
To achieve the desired goal of drug affordability, focus should be shifted from the pharmaceutical companies to insurance reform. The costs that weigh on patients are out-of-pocket costs, which are the expenses individuals must pay beyond what their insurance covers. These take form in high deductible payments, as well as high co-pays on visits, procedures, and drug prescriptions. If legislative efforts would be focused towards passing acts such as the HELP Copays Act – which aims to require health insurance plans to factor any
reduction in out of pocket payments made by or on behalf of the enrolled patient towards a plan’s cost-sharing requirements – patients would experience immediate relief.
As current legislation stands, the forty million dollar investment the Cystic Fibrosis Foundation made in Vertex Pharmaceuticals small molecule therapy back in 2000 would no longer make sense in today’s climate. While Trikafta entered my life at the right time, looming price controls stand between my experience and other rare disease sufferers, who deserve the same renewed outlook on life I have been afforded. If the end goal is to truly help the patient, the company driving change shouldn’t bear the brunt of the industry’s flaws. It’s time we shift our focus towards actions that benefit patients’ long term viability, and preserve our country’s culture of incentivization that continues to drive advancements in global health today.
Sources:
1:
https://rarediseases.org/wp-content/uploads/2019/01/RDD-FAQ-2019.pdf
2:
3:
4:
https://www.nopatientleftbehind.org/blog/95r1kiynt79tpamvn2z3b7ms54v6nq
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Shared Decision Making & Care Guidelines by: Mallory Holloway 10 Dec 2024, 2:51 pm
Shared Decision Making & Care Guidelines
Written by: Mallory Holloway
As someone living with cystic fibrosis, I’ve spent countless hours of my life in and out of doctor’s offices. While my journey has been shaped by numerous healthcare professionals as well as my care team, I have learned that the most empowering experiences have come from moments where I was actively involved in my treatment decisions. This collaborative approach, known as shared decision-making, has allowed me to take ownership of my healthcare and has improved my physical health and overall well-being.
Shared decision-making is a model where healthcare providers and patients come together to make decisions regarding treatment plans. This involves considering medical evidence as well as the patient’s personal goals, values, and experiences. I believe that the shared decision-making process must be implemented in clinical settings to foster better communication, improve patient outcomes, and provide holistic care.
The concept is not just about understanding a patient’s medical history—it’s about considering their mental health and preferences while they are going through medical hardships. I was lucky enough, and still am today, to have a care team that has been with me my whole life and knows almost more about me than I do. Because of this, it’s easy for them to work with me and easy for me to communicate my goals with them.
The process gives patients a voice in their healthcare. It’s a holistic approach that shapes informed decisions drawn from medical evidence and values. Colorectal cancer is a high risk for CF patients due to the challenges caused by the CFTR protein. This information should be transparently shared between patients and doctors. They should be told why early screening is important, what symptoms to watch for, and how screenings can fit into their health plan. As patients living with a chronic condition, we are the ones who have to live with the outcomes of treatment decisions, so it only makes sense that we have a say in the process.
One of the most impactful moments of shared decision-making in my journey was choosing between continuing my existing CF medication regimen or switching to a newer treatment with promising results. Although my doctors knew that the new medication was working well with other patients, they still allowed me to decide if I thought it was for me. I was
able to have an open conversation with my care team about the benefits and downsides. We discussed how the newer treatment could reduce my daily pill count and eliminate breathing treatments but also addressed potential side effects and long-term unknowns. Ultimately, the decision was mine and my parents, and because I had been an active participant in the discussion, I felt a sense of power in my medical journey.
Shared decision-making not only improves communication but also enhances patient’s compliance and commitment to treatment. In a traditional healthcare model, doctors often provide patients with treatment options and the assumption that they know what’s best for them. However, it is a fact that no one knows their body better than the inhibitor. In situations where there isn’t a clear “best” option, this process allows for someone to weigh the pros and cons of different treatments based on what matters most to them.
For example, it is common for people living with CF to have elevated levels of cholesterol. Because of this, they may be presented with the option of adding a statin—which is a drug that reduces levels of fats in blood—to their medical regimen. Some patients may choose the statin, others may focus solely on lifestyle changes, while others may choose a mix of both. In this case, there can be benefits from all approaches and similar outcomes, so the choice must be up to the patient. This will help patients do a better job of sticking to their goals and following their treatment plans. Every patient has a different lifestyle and is working towards different goals and this needs to be acknowledged and prioritized by healthcare providers.
In addition to autonomy, shared decision-making strengthens the bond between patients and their doctors. Throughout my CF journey, I’ve built many strong relationships with my doctors because they’ve treated me as a partner in my care. My doctors understand almost every aspect of my journey and what matters to me most. This connection is crucial for someone with a chronic illness, where medical decisions are ongoing and often emotionally taxing. Feeling heard and valued by my care team has made a significant difference in how I view my healthcare.
Of course, shared decision-making isn’t without its challenges. Healthcare appointments are already short, and including additional time for detailed conversations can be daunting. While this can be tedious and annoying, I believe it will make a difference. It will ensure the patient leaves the appointment understanding their options and feeling confident in their choice.
Another challenge is that not every patient feels comfortable or confident making decisions about their healthcare. Healthcare providers need to recognize this and support their patients. Effective communication, compassion, and patience are the keys to guiding patients, especially those dealing with chronic or life-threatening conditions, like CF.
Language barriers and communication issues can also make this process difficult. I’ve been fortunate to have providers who explain things in ways I can understand, but I know that isn’t always the case for everyone. It’s critical for healthcare providers to be mindful of their communication style, ensuring that patients from all backgrounds feel empowered to engage in their healthcare.
This patient-centered approach to healthcare has transformed my life and made my trips to the doctor that much easier. Involving me in the decision-making process has allowed me to take ownership of my treatment, leading to better outcomes and a deeper sense of control over my condition. Shared decision-making should be embraced as a necessary shift toward patient-centered care. It serves as the ultimate key to recognizing providers’ expertise while also empowering patients to be active participants in their health.
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The Value of Medicines and Who Decides It by: Kashvi Kapoor 10 Dec 2024, 2:41 pm
The Value of Medicines and Who Decides It
Written by: Kashvi Kapoor
Medicines save lives. They improve health, extend lifespans, and allow people to live fuller, richer lives. But the true value of a medicine is often much more than the pill in a bottle or the price on a label. For patients, a life-changing drug might mean the ability to breathe freely, to hug loved ones, or simply to wake up to another day. For society, medicines can mean fewer hospital beds filled, fewer days lost at work, and a healthier, happier population.
Deciding how much a drug is worth is complicated. Who gets to make that decision? Governments? Pharmaceutical companies? Patients themselves? This paper explores the human and societal value of medicines, the challenges in determining their worth, and real world examples, focusing on the impact of cystic fibrosis treatments and a poignant case study from Australia.
Every medicine has a story—a journey of discovery, testing, and, ultimately, the hope it brings to patients. From a societal perspective, medicines are invaluable. They allow us to prevent diseases, reduce suffering, and cure once-incurable conditions. But when it comes to assigning a dollar amount, things get tricky.
Pharmaceutical companies invest billions in research and development to create new drugs. Governments and healthcare systems, on the other hand, work to ensure medicines are affordable and accessible. Tools like cost-effectiveness analysis help weigh a drug’s price against its benefits, such as how much it improves patients’ quality of life or reduces healthcare costs.
But these calculations often leave out the human element. Imagine telling a parent that a drug costing thousands of dollars per month is “too expensive,” even though it could save their child’s life. For that parent, no cost is too high. Striking the balance between affordability and innovation while keeping humanity at the center of the conversation is one of the greatest challenges in valuing medicines.
The Importance of Medicines for Cystic Fibrosis
Cystic fibrosis (CF) offers a powerful example of how medicines change lives. CF is a genetic disorder that clogs the lungs and digestive system with thick mucus, making every breath a struggle. For decades, treatments managed symptoms but didn’t address the root cause. Then came CFTR modulators—breakthrough drugs that target the disease at its source.
For people with CF, these medicines are nothing short of miraculous. They extend life expectancy, reduce hospital stays, and, most importantly, give people back their futures. A young adult with CF who once planned for a life measured in decades can now dream of college, a career, and a family.
But the cost of these medicines is staggering, sometimes exceeding $300,000 per patient annually. Governments and insurance companies must weigh the undeniable benefits against their budgets. For patients, the waiting and negotiating feel agonizing. For many, access to these drugs is a race against time.
A Case Study from Australia
Australia’s struggle with providing CFTR modulators, particularly Trikafta, reveals just how high the stakes can be. For two years, Australians with CF waited while the government and the drug manufacturer negotiated over the price of Trikafta. During this time, patients experienced worsening symptoms and watched their health deteriorate, knowing a potentially life-saving drug was just out of reach.
Eventually, a deal was struck, and Trikafta was added to the Pharmaceutical Benefits Scheme, making it affordable for thousands. While this was a victory, the delay had lasting impacts. Families felt abandoned, and many patients’ health deteriorated to a point where the drug could no longer fully reverse the damage.
This case highlights a critical truth: delays in access can be as harmful as outright denial. Medicines like Trikafta are more than numbers on a spreadsheet; they are lifelines for people fighting daily battles with their health.
The value of medicines goes far beyond their price tag. They hold the power to change lives, extend futures, and transform society. But deciding how much they are worth and who can access them is a delicate, deeply human challenge.
As we’ve seen with cystic fibrosis treatments and the Australian Trikafta case, delays in decision-making can have real, human consequences. Governments, pharmaceutical companies, and healthcare systems must work together to strike a balance—ensuring that innovation is rewarded but not at the expense of accessibility.
Ultimately, the value of a medicine should reflect not only its scientific and economic impact but also the hope it brings to the people who need it most. By putting patients at the heart of these decisions, we can ensure that the medicines of today—and tomorrow—reach those whose lives depend on them.
References
European Federation of Pharmaceutical Industries and Associations (EFPIA). (n.d.). Value of medicines. Retrieved from https://www.efpia.eu/about-medicines/use-of-medicines/value of-medicines/
Neumann, P. J., Cohen, J. T., & Weinstein, M. C. (2014). Updating cost-effectiveness — the curious resilience of the $50,000-per-QALY threshold. New England Journal of Medicine, 371(9), 796-797. https://doi.org/10.1056/NEJMp1405158
O’Neill, A., McNamara, M., & Wainwright, C. E. (2019). The cost of cystic fibrosis—an update and the need for innovative health economic solutions. Current Opinion in Pulmonary Medicine, 25(6), 611-617. https://doi.org/10.1097/MCP.0000000000000624
RealClearHealth. (2022). For two years, Australia failed people with cystic fibrosis. It can’t happen again. Retrieved from
https://www.realclearhealth.com/articles/2022/02/18/for_two_years_australia_failed_peop le_with_cystic_fibrosis_it_cant_happen_again_111317.html
The Value in Health Journal. (2020). Valuing medicines in the 21st century. Value in Health, 23(5), 615-623. https://doi.org/10.1016/j.jval.2020.02.001
Wouters, O. J., McKee, M., & Luyten, J. (2020). Estimated research and development investment needed to bring a new medicine to market, 2009-2018. JAMA, 323(9), 844-853. https://doi.org/10.1001/jama.2020.1166
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How Avery Flatford Manages Cystic Fibrosis While Playing for Tennessee Softball 17 May 2024, 3:43 pm
By Jack Church, The Daily Beacon
“When Ryleigh White got injured early in conference play, Karen Weekly was in a jam.
“We just didn’t want to get ourselves in a situation once Ryleigh White was hurt,” Weekly said, “where if another pitcher woke up sick on a Friday morning — now we didn’t want to go into a weekend with two.”
She reached out to Avery Flatford, a former Farragut pitcher and student at the University of Tennessee. Flatford played one season at Tennessee Tech but came to UT to focus on school.
When the opportunity came to join the Lady Vols, Flatford couldn’t pass it up.
“It’s been my dream ever since I was five years old playing at CBFO out in Farragut,” Flatford said. “It’s always been my dream, and I am just ecstatic to be here and very happy to be a part of this family.”
Flatford doesn’t have it easy. She has cystic fibrosis, a disorder affecting her lungs and digestive system. Despite that, she excelled at Farragut and won two state championships, throwing three no-hitters during her junior season.
While managing her medical condition, she has found a way to excel in softball. Whether at Farragut, Tennessee Tech or now with the Lady Vols, Flatford has found success playing the sport she loves.
“Softball and CF have gone hand in hand my whole life,” Flatford said. “So, over the years, I’ve been able to keep adapting and make sure I’m on top of it.” [Source: Jack Church, The Daily Beacon]
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Running is a Remedy for Mallory Holloway 1 Apr 2024, 6:19 pm
Running is a Remedy for Mallory Holloway
BY AIDAN LAVIN, CENTER FIELD
“Marist freshman distance runner Mallory Holloway was born into running.
Holloway grew up as an only child with parents who ran in college at the University of South Carolina and Cal Poly Humboldt, and then went on to become track coaches as adults.
The path to becoming the promising distance runner she is today, however, was not as straightforward as it appeared on the surface.
According to the Cystic Fibrosis Foundation, there are 40,000 individuals living with cystic fibrosis in the United States and 105,000 people across 94 countries. At 18 months old, Holloway was diagnosed with the disease.
Cystic fibrosis, a double recessive rare genetic lung disease, is progressive and life-threatening due to the formation of thick mucus built up in the lungs, digestive tract and other parts of the body. It leads to severe respiratory and digestive problems as well as other complications. An individual running long distances with cystic fibrosis would seem to be out of the question.” [Source: Aidan Lavin, Center Field]
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2023 Year in Advocacy Blog Post 21 Dec 2023, 11:39 am
Here’s a recap of some highlights from the Boomer Esiason Foundation’s 2023 Year in Advocacy and other cystic fibrosis news.
In March, BEF signed on to a letter supporting the PASTEUR Act, a bipartisan, bicameral legislation that calls for the development of a sustainable antibiotic pipeline to meet the growing threat of antimicrobial resistance. The pipeline is based on a novel payment model that fosters innovation and creates financial incentives for new drug development. People living with CF are especially vulnerable to resistant infections and need access to new antibiotics now.
April was Donate Life Month, which raises awareness for organ donation and celebrates the lives saved by organ transplantation. One organ donor can save up to eight lives and there are over 100,000 people waiting for organ transplants.
For CF Awareness Month in May, BEF launched a robust CF education campaign to spread CF awareness.. Also in May, the FDA approved Kaydelco for infants as young as one month old, which is the first and only modulator approved for infants in that age group.
In June, BEF highlighted World Infertility Awareness Month with an education campaign about CF and infertility. Most men with CF are infertile and many women with CF struggle with issues that may impact their fertility. The Foundation has made it a key priority to educate community members about CF and infertility, as well as encouraging people living with CF to talk with their care teams to discuss family building options. While there is an alarming lack of accessible information for people with CF about family building, in recent years research, awareness, and educational resources have begun to appear more frequently. The campaign also called attention to the BEF IVF program, which supports people with CF to realize their dreams of starting a family.
BEF spoke out against the Colorado Prescription Drug Affordability Review Board’s decision to review the affordability of Trikafta in August, condemning the board’s decision to consider an upper price limit for the lifesaving drug. Such a price limit would jeopardize Coloradans’ access to Trikafta and disincentivize broader drug development. Later in the month, cystic fibrosis related infertility and BEF’s IVF program was featured on the Man Up podcast. The episode took a deep dive into the stigma and mental health aspects of infertility, as well as advice for people struggling with infertility.
This October, in a victory for patients everywhere, a federal court struck down a court ruling that allowed insurers to use copay accumulator adjusters for drugs that do not have a generic equivalent. This decision was a huge win for the CF community, as copay accumulators unfairly disadvantage people with CF who rely on copay assistance programs to afford their medications.
In November, BEF participated in the 2023 Milken Institute Future of Health Summit, and was featured in a panel on antibiotic development. The panel featured leaders in the nonprofit, research, and biopharma spaces who discussed the devastation that antimicrobial resistance poses to public health both now and in the future, as well as the importance of the PASTEUR Act in the current health policy landscape.
The year ended on a high note– on December 8, the Colorado Prescription Drug Affordability Review Board determined that Trikafta was “not unaffordable” for patients living in Colorado. This victory came after the passionate, steadfast advocacy of the CF community, who voiced their needs and showed the importance of patient centricity in healthcare decision-making.
As we look back on 2023, we’re so proud of all that we have accomplished as an organization and as a community. We are anticipating that 2024 will be an impactful year for advocacy and more as we look forward to making even more progress towards creating a better future for people living with CF.
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Milken Institute Future of Health Summit Recap 7 Dec 2023, 9:40 pm
Last month, BEF attended the Milken Institute Future of Health Summit in Washington D.C. The conference was a who’s who of leaders in public health, biotech, pharma, philanthropy, finance, government, and healthcare. A common theme throughout the conference was the challenge of meeting an exponentially growing list of unmet needs with thoughtful, effective, and efficient solutions. The agenda was thoughtfully crafted and, unlike most conferences, most panels feature a diversity of thought which sometimes erupted in professional debate.
We began our time at the conference listening to a panel on the future of gene and cell therapies, which are at the forefront of an exciting frontier in medicine whose promises are enormous, but so are their price tags. The discussion centered around the affordability of these treatments, which hinges on four major points: manufacturing, technology, data, and policy. Scaling these therapies and developing infrastructure to share information are some future directions that could broaden gene and cell therapies’ accessibility and drive these technologies further into the mainstream. This is imperative for the 10% of people living with CF who do not benefit from existing CF modulator therapies and for the broader CF community. Cell and gene therapies are rapidly progressing toward a treatment for the final 10% and toward a cure for all people with CF. Once these therapies are developed, affordability and accessibility will be key challenges to overcome in order to ensure that they can get to the people who need them the most.
The second panel we attended focused on investing in health care innovation. The panel consisted of leaders in the financial and biotech realms who discussed the challenges facing health start-ups today, particularly regulatory barriers and the high cost of capital. When the cost of capital is high, the risk is magnified for operators and financers. The importance of discipline, flexibility, and a focus on patient centricity in business models were all key themes that emerged from the leaders’ varied perspectives. Although it may be a challenging time for health start-ups, with the right leadership, management, and value proposition, there is potential for greatness. CF is a major success story in healthcare innovation and a shining example of how effective partnerships and regulations can work together to produce a life-changing medical breakthrough. However, we can’t stop there. The same drive and creativity must be applied to ensuring that 100% of people living with CF have access to a treatment and a cure.
Day one ended with a deep dive into meeting patient needs, particularly how to overcome barriers to patient engagement. The panelists weighed in on developing trust between patients and providers, making clinical trials more accessible, treating the whole patient, and recognizing the importance of non-medical factors in health outcomes. To develop trust with patients, providers must participate in the communities that they serve and develop reciprocal relationships by listening to patients’ needs. One key takeaway was that healthcare should take time to learn from other industries like finance, hospitality, and travel when it comes to consumer centricity.
The second day began with a fascinating discussion of the threat of antimicrobial resistance (AMR) featuring BEF’s Gunnar Esiason, who shared his perspective as a patient advocate and weighed in on the importance of developing novel antibiotics to bolster the US’s currently inadequate antibiotic supply. AMR is not just a future threat– it is a current, pressing problem. Senators Todd Young (R-IN) and Michael Bennett (D-CO),bipartisan cosponsors on a piece of legislation called the PASTEUR Act, followed up the panel with a discussion focused on financing a sustainable pipeline for new antibiotic development. They also pointed to the United Kingdom where a pilot reimbursement model is in place that shows how PASTEUR might work in the United States.
Next, leaders in philanthropy and pharmaceuticals took on the topic of “moonshot” philanthropy, which sets lofty, bold goals to solve seemingly insurmountable problems in health. Strategies for reaching these “moonshots” included using capital in a complementary way to existing programs, using personal stories and patient perspectives to drive a philanthropic narrative, and finding intersections between the mission of an organization and the current zeitgeist. In many way, the Cystic Fibrosis Foundation’s venture philanthropy success has been seen as the playbook for transforming the prognosis of a killer condition, and was on the top of everyone’s mind.
Rare disease research is an essential branch of research that has seen tremendous progress in the past few decades. However, this progress is far from complete, as the vast majority of people living with rare diseases do not have a treatment or a cure. Apart from the need to inspire innovation in drug development to treat these rare diseases, there is also a dire need to improve infrastructure to ensure that these drugs are delivered to the patients who need them. The panelists also emphasized the importance of centering the needs and voices of patients in developing future rare disease treatments.
We had the pleasure of meeting parents of children with rare diseases and learning more about initiatives that empower patients with resources and guidance on their way to discovering cures. Parents are powerful advocates and key drivers of change in the rare disease space. Partnerships between rare disease advocates can provide valuable insight for all involved, which is why it is so important to connect with the rare disease community outside of CF. Rare disease breakthroughs can lead to the development of policies, tools, and infrastructure that can translate to other groups and drive more cures for more people.
The conference ended with a discussion of the balance between innovation and access, a key point of tension among the panels. ARPA-H Director Renee Wegrzyn and the 16th NIH Director Francis Collins discussed the importance of healthcare delivery as a tool to improve access to innovative medicines. Of course, Francis Collins is practically a headlining celebrity in the CF community for his work to identify the CF-causing gene mutation.
In short, the Future of Health Summit was inspiring and intellectually challenging. Although we walked away aware of more problems in health than solutions, the spirit of cross-disciplinary collaboration and bipartisan partnership made us hopeful for the future. While there was a lot of technical talk of ROIs, regulatory hurdles, and biological minutiae, there was also a deep sense of compassion and care. Many of the speakers made it clear that they were driven by their desire to make a real difference and their commitment to end suffering. The emotional core of health must not be overlooked as we forge ahead to find new solutions to both age-old and rapidly emerging problems.
The main takeaways from the Milken Institute Future of Health Summit:
- Patient centricity is the future of healthcare. Patients are active consumers of healthcare, not passive beneficiaries. They need to be central in the current and future development of the healthcare landscape. Patient engagement is key to creating solutions that address the growing list of unmet needs that pervade healthcare.
- Policy needs to meet the moment. Effective policies can address the inefficiencies and gaps of the US healthcare system, but can also bolster its current strengths. Politicians and government officials need to listen to patients when crafting policy that meets their needs and ensures that all people have accessible, affordable, and comprehensive care.
- Collaboration and communication are vital to bridging gaps in healthcare. There are far too many of these gaps and they are often much too wide. Communication between the private and public sectors is key to ensuring both innovation and access in healthcare, while better, more effective communication between health professionals and the general public can empower people to take charge of their health.
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Antimicrobial Resistance: What You Need to Know 1 Dec 2023, 4:31 pm
Most people have taken antibiotics in their lifetime– they treat common bacterial infections like strep throat, UTIs, and whooping cough. Antibiotics are life saving medicines that also treat serious infections like sepsis. People living with CF often rely on antibiotics to treat infections in their lungs. People with CF are at an elevated risk for bacteria infections because of the thick sticky mucus in their lungs.
Bacterial infections, like other microbial infections, are often hard to medically treat because they can evolve to become impervious to commonly used antibiotics designed to kill them. Because people with CF need to be treated with antibiotics from a young age, they are at a greater risk of their infections becoming increasingly resistant over time. Multidrug resistant and pan-resistant infections occur when a bacterial infection is resistant to more than one or all available antibiotics, and are commonly called superbugs. For people living with CF, superbugs can lead to end-stage disease and complicate transplant eligibility.
People living with CF have been facing a reality that many more people maysoon experience. The World Health Organization designated antimicrobial resistance (AMR) as one of the top ten global public health threats facing the world. Antimicrobial resistant infections killed at least 1.27 million people and were related to almost 5 million deaths in 2019, while hospital-associated infections and deaths increased 15% from 2019 to 2020 in the US. There are almost 3 million AMR infections in the US each year, 35,000 of which are fatal.
AMR is not just deadly, it’s costly. In 2013, the CDC estimated that AMR costs the U.S. $55 billion per year, $35 billion of which is a loss of productivity. In 2050, that number could reach the hundreds of trillions. Left unchecked, AMR could decrease the size of the labor force, contribute to GDP loss, and significantly reduce livestock output.
There is a shocking lack of new antibiotics available in the U.S. today. Available antibiotics are based on drug classes that were discovered almost 40 years ago and are becoming less and less effective against infections. The shortage of novel antibiotics can be attributed to a lack of resources, interest, and path to profitability for drug developers. The current antibiotic development pipeline has failed people with CF and is woefully inadequate to tackle the growing presence of antibiotic resistance.
The Pionering Antimicrobial Subscriptions To End Upsurging Resistance (PASTEUR) Act is a proposed bipartisan, bicameral legislation that would address the current lack of an effective pipeline for novel antimicrobials. The legislation uses “pull incentives” like a Netflix subscription; the government pays developers a fixed rate in exchange for access to critical need antimicrobials. This subscription model ensures that developers have a sustainable financial incentive and path to profitability, which removes a significant barrier to antibiotic development.
The PASTEUR Act not only funds the development of new antibiotics, but also of diagnostic tools, surveillance, vaccines, and other alternative treatments. Antibiotic stewardship and surveillance are key parts of the PASTEUR Act because key contributing factors to AMR are the overuse and misuse of antibiotics. Irresponsible antibiotic use occurs when doctors prescribe an antibiotic for an infection that does not respond to antibiotics or when patients do not adhere to antibiotic protocols. Antibiotic misuse and overuse in food production is also a large driver of AMR. PASTEUR would create a robust surveillance and governance system and expand upon current frameworks to ensure that new antibiotics are used safely and effectively.
There have been some recent breakthroughs in the fight against AMR. Recently, scientists developed a new antibiotic called zoliflodacin that is an effective cure for gonorrhea, which has become increasingly drug-resistant. Zoliflodacin was developed by the nonprofit Global Antibiotic Research & Development Partnership (GARDP) in collaboration with Innoviva Specialty Therapeutics. This development marks a breakthrough in new antibiotic development and shows the effectiveness of using new models to promote the development of previously unprofitable drugs.
There has also been a breakthrough in antibody therapy for antibiotic resistant infections. In a recent study from the University of Cologne in Germany, researchers isolated antibodies from immune cells from people with CF who were infected with Pseudomonas aeruginosa, a multidrug resistant pathogen. These antibodies effectively targeted bacteria in animal models, which could lead to the development of antibody therapies that target infections that become resistant to antibiotics.
Although the threat of AMR is significant, there is a path forward– we just need to take it. The PASTEUR Act would incentivize new antibiotic development and set up a robust framework for antimicrobial surveillance and governance. New scientific breakthroughs are already underway, but they need the right infrastructures to ensure that new treatments are safe, accessible, and properly used. AMR has already taken too many lives and we cannot wait any longer.
- Learn more about antimicrobial resistance from these resources:
Support the PASTEUR Act by urging your representatives to act using the links HERE and HERE.
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Mallory Holloway breaks PR and earns First Team All-Met Honors 12 Oct 2023, 2:30 pm
“NEWARK, New Jersey – On Saturday morning the women’s cross country competed at Branch Brook Park in the Metropolitan Conference Cross Country Championships. Despite sitting out some key runners, the women raced to a strong sixth place finish overall as a team.
Leading the way was freshman Mallory Holloway, who finished in 12th place with a personal best time of 18:30.58 to earn First Team All-Met Honors.
On the day, 10 women competed for Marist and the results were six personal bests, three collegiate bests, and one season best time.
“I am so extremely proud of how well our women executed today out there on the course. After a sub-par showing at Paul Short last week, it was so nice to see the girls bounce back and have their best team performance of the season,” said Head Coach Chuck Williams. “In our sport we are constantly striving to get five women to put together their best races all on the same day, but to have all 10 women achieve that today gives us a great deal of confidence as we prepare for the MAAC championships.”
Next up for the women’s cross country team will be the Vermont Fall Foliage Invitational on Sunday, Oct. 15 in St. Albans, VT.” [GoRedFoxes.com]
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