Year: 2023

Categories : HIV PaediatricsTags :

Treating Newborns with HIV in 1st 48 Hours may Result in Medication-free Remission

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Photo by Christian Bowen on Unsplash

An unexpectedly high percentage of children, who were born with HIV and started treatment within 48 hours of life, exhibit biomarkers by two years of age that may make them eligible to test for medication-free remission, according to a multinational study published in The Lancet HIV.

“Moving away from reliance on daily antiretroviral therapy (ART) to control HIV would be a huge improvement to the quality of life of these children,” said Protocol Co-Chair and senior author Ellen Chadwick, MD, at Ann & Robert H. Lurie Children’s Hospital.

Conducted in 11 countries including South Africa, the proof-of-concept study was charged with replicating the case of HIV remission as seen in the “Mississippi baby” that was reported in 2013. In that case, the infant started ART at 30 hours of life, was treated for 18 months, and achieved 27 months of ART-free remission before the virus rebounded. Typically, if ART is stopped, the virus rebounds within a month.

The study included a three-drug ART regimen initiated within 48 hours of life, with the fourth drug added within 2-4 weeks. This is very early treatment compared to the standard of care where three-drug ART may not begin until 2-3 months of age.

In the US, however, based on earlier findings from this study, very early treatment is now the norm for infants at high risk of acquiring HIV infection from their mother.

“With earlier treatment, we hope to limit or prevent the establishment of viral reservoirs in the body. These viral reservoirs hold small amounts of hidden virus which are hard to reach with ART. By shrinking these reservoirs, we expect to increase the amount of time that patients can be in remission, without needing daily ART,” said co-author and Protocol Co-Chair Jennifer Jao, MD, MPH, from Lurie Children’s.

Dr Chadwick adds: “Another benefit of smaller viral reservoirs might be that newer treatments such as long-acting antibody therapies or therapeutic vaccines could potentially be used instead of daily ART.”

“Our results show a higher percentage of children might be eligible to interrupt therapy than we expected, and the next step is to stop ART and see how many children actually achieve remission,” said Dr Chadwick.

“If even one child achieves remission, that would be considered a success. Today, newer more effective and better tolerated HIV medications are available for infants than when the study began, strengthening the prospect of limiting viral reservoirs and testing for possible remission in infants and children with HIV. Overall, this is an exciting advancement and an opportunity to change the course of pediatric HIV infection.”

The study was conducted in 11 countries – Brazil, Haiti, Kenya, Malawi, South Africa, Tanzania, Thailand, Uganda, USA, Zambia and Zimbabwe.

Source: Ann & Robert H. Lurie Children’s Hospital of Chicago

Categories : Immune SystemTags :

Mucus is Snot a Problem for Bacteria, Which Swarm Through It

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Photo by Andrea Piacquadio on Pexels

The increase in mucus from sniffles and runny noses is exactly what bacteria use to mount a coordinated attack on the immune system, according to a new study from researchers at Penn State. The team found that the thicker the mucus, the better the bacteria are able to swarm. The findings could inform treatments to control the spread of bacteria.

The study, recently published in the journal PNAS Nexus, demonstrates how bacteria use mucus to enhance their ability to self-organise and possibly drive infection.

The experiments, performed using synthetic pig stomach mucus, natural cow cervical mucus and a water-soluble polymer compound called polyvidone, revealed that bacteria coordinate movement better in thick mucus than in watery substances.

According to the researchers, the findings provide insight into how bacteria colonise mucus and mucosal surfaces, and also show how mucus enhances bacterial collective motion, or swarming, which may increase antibiotic resistance of bacterial colonies.

“To the best of our knowledge, our study is the first demonstration of bacteria collectively swimming in mucus,” said Igor Aronson, Huck Chair Professor of Biomedical Engineering, of Chemistry and of Mathematics at Penn State and corresponding author on the paper.

“We have shown that mucus, unlike liquids of similar consistency, enhances the collective behaviour.”

Mucus is essential for many biological functions, explained Aronson. It lines the surfaces of cells and tissues and protects against pathogens such as bacteria, fungi and viruses. But it is also the host material for bacteria-born infections, including sexually transmitted and gastric diseases.

A better understanding of how bacteria swarm in mucus could lead to new strategies to combat infections and the growing problem of antibiotic resistance, according to Aronson.

“Our findings demonstrate how mucus consistency affects random motion of individual bacteria and influences their transition to coordinated, collective motion of large bacterial groups,” Aronson said.

“There are studies demonstrating that collective motion or swarming of bacteria enhances the ability of bacterial colonies to fend off the effect of antibiotics. The onset of collective behaviour studied in our work is directly related to swarming.”

Mucus is a notoriously challenging substance to study because it exhibits both liquid-like and solid-like properties, Aronson explained.

Liquids are typically described by their level of viscosity, how thick or thin the liquid is, and solids are described by their elasticity, how much force it can take before breaking. Mucus, a viscoelastic fluid, behaves as both a liquid and solid.

To better understand how mucus becomes infected, the team used microscopic imaging techniques to observe the collective motion of the concentrated bacteria Bacillus subtilis in synthetic pig stomach mucus and natural cow cervical mucus, which for this purpose are analogous to human mucus.

They compared those results with observations of Bacillus subtilis moving in a water-soluble polymer polyvidone at a wide range of concentrations, from high to low levels of polyvidone.

The researchers also compared their experimental results to a computational model for bacterial collective motion in viscoelastic fluids like mucus.

The team found that mucus consistency profoundly affects the collective behaviour of bacteria: the thicker the mucus, the more likely the bacteria would exhibit collective movement, forming a coordinated swarm.

“We were able to show how the viscoelasticity in mucus enhances bacterial organisation, which in turn leads to coherently moving bacterial groups that cause infection,” Aronson said.

“Our results reveal that the levels of elasticity and viscosity in mucus are a main driver in how bacterial communities organize themselves, which can provide insight into how we can control and prevent bacterial invasion in mucus.”

Aronson explained that the team expects human mucus to exhibit similar physical properties, meaning their findings are also relevant for human health.

“Our results have implications for human and animal health. We’re showing that mucus viscoelasticity can enhance large-scale collective motion of bacteria, which may accelerate how quickly bacteria penetrate mucus protective barrier and infect internal tissues.”

Source: Penn State

Categories : Allergies CancerTags :

Existing Allergy Medication Unleashes Antitumour Immunity against Lung Cancer

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Photo by Anna Shvets

Researchers from Mount Sinai report in Nature that they have identified an allergy pathway that, when blocked, unleashes antitumour immunity in mouse models of non-small cell lung cancer (NSCLC).

And in an early parallel study in humans, combining immunotherapy with dupilumab – an Interleukin-4 (IL-4) receptor-blocking antibody widely used for treating allergies and asthma – boosted patients’ immune systems, with one out of the six experiencing significant tumour reduction.

“Immunotherapy using checkpoint blockade has revolutionised treatment for non-small cell lung cancer, the most common form of lung cancer, but currently only about a third of patients respond to it alone, and in most patients, the benefit is temporary,” says senior study author Miriam Merad, MD, PhD, at the Icahn School of Medicine at Mount Sinai.

“A big focus of our program TARGET is to use single cell technology and artificial intelligence to identify molecular immune programs that can dampen tumour immune response to checkpoint blockade.”

Also known as a PD1 inhibitor, checkpoint blockade is a type of cancer immunotherapy that can unleash the cancer-killing activity of T cells.

“Using single cell technologies, we discovered that the immune cells infiltrating lung cancers, as well as other cancers we studied, exhibited characteristics of a ‘type 2’ immune response, which is commonly associated with allergic conditions like eczema and asthma,” says first study author Nelson LaMarche, PhD, a postdoctoral research fellow in the lab of Dr Merad.

“These results led us to explore whether we could repurpose a medication typically used for allergic conditions to ‘rescue’ or enhance tutor response to checkpoint blockade,” says Thomas Marron, MD, PhD, co-senior author of the study.

“Strikingly, we found that IL-4 blockade enhanced lung cancer response to checkpoint blockade in mice and in six lung cancer patients with treatment-resistant disease. In fact, one patient whose lung cancer was growing despite checkpoint blockade had nearly all their cancer disappear after receiving just three doses of the allergy medication, and his cancer remains controlled today, over 17 months later.”

The researchers are encouraged by the initial results but emphasise the need for larger clinical trials to validate the drug’s efficacy in treating NSCLC.

Beyond the clinical trial findings reported in the current Nature paper, the investigators have now expanded the clinical trial, adding dupilumab to checkpoint blockade for a larger group of lung cancer patients, and are starting to investigate its use in early-stage lung cancer as well. Through these trials, they are searching for biomarkers to identify those cancer patients who might benefit from dupilumab treatment.

Source: The Mount Sinai Hospital / Mount Sinai School of Medicine

Categories : Addiction Pain ManagementTags :

One in Six Patients with Opioid Use Disorder Leave the Hospital too Early

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The number of patients admitted with opioid use disorder (OUD) and injection-related infections who left the hospital before completing treatment increased significantly between 2016 and 2020 (from 9.3% to 17%) according to analysis from researchers at the Perelman School of Medicine at the University of Pennsylvania. One in six of these patients now leave the hospital before medically advised (BMA).

The findings, published in JAMA, also reveal that the rate at which patients with any opioid-related issues (patients presenting with other issues but exhibiting opioid dependence) left the hospital BMA increased more than 50% (from 7.5 to 11.3%). In both of these groups, nearly half of BMA discharges occurred before the third day, when withdrawal symptoms are most severe. Now that fentanyl has become the dominant opioid causing overdoses, the findings illustrate the need for patient-centred care that adequately manages pain and withdrawal symptoms so that patients can complete treatment.

Approximately 500 000 patients are discharged against medical advice, or in the United States annually, and those circumstances are associated with increased likelihood of death and hospital readmission. Previous research shows that patients with addiction cite withdrawal and pain as their reason for BMA discharge.

“The rapid increase in early discharges is alarming; in 2016, less than one in ten patients admitted for OUD and injection-related infections left the hospital before their care team considered it safe. By 2020, one in six were leaving early,” said lead author Ashish Thakrar, MD. “What’s more, since the study period ended, the COVID-19 pandemic caused the opioid crisis to escalate, underscoring just how urgent it is to understand how we might be able to reverse this trend and get patients the treatment they need.”

Using nationally representative data from the National Readmissions Database, researchers compared the rate of discharge BMA in patients admitted for OUD to the BMA discharge rate for non-opioid admissions. They also evaluated changes in the proportion of BMA discharges before the third admission day, when opioid withdrawal is most severe, and changes in the proportion of discharges BMA in patients with stimulant use disorder.

They identified opioid-related admissions as those with opioid use, dependence, abuse, or overdose. To account for patients who were more likely to have severe OUD and fentanyl use, they also included patients with OUD and an injection related infections, such as bacteraemia, endocarditis, or osteomyelitis.

Between 2016 and 2020, they found that the number of patients admitted with OUD and injection-related infections who left the hospital BMA increased 82%, from 9.3% to 17%. They also found that the discharge BMA rate for all opioid-related admissions increased 50% during this period, from 7.5% to 11.3%. The proportion of BMA discharges occurring before the third day also increased for individuals with OUD and an injection-related infection, from 42.6%, to 48%.

In contrast, the BMA rate increased only marginally for non-opioid mental health or substance use admissions, and all non-opioid admissions (from 3.1 to 3.5%, and 1.1 to 1.5%, respectively).

“These data didn’t allow us to discern which type of opioid that individuals were using when admitted for OUD, but we know that fentanyl, an opioid 25 to 50 times more potent than heroin, has spread in unregulated drug supplies and is now involved in 88 percent of opioid overdoses in the US. Withdrawal symptoms from fentanyl are more difficult to manage than from other opioids like heroin and oxycodone,” said Thakrar. “This study illustrates why we need more research on how to manage individuals withdrawing from fentanyl and other substances in the unregulated drug supply.”

“The drugs that individuals are using have changed over the past decade, and how we treat them should change, too,” said senior author M. Kit Delgado, MD, MS. “Health systems can expand the use of interventions that are already proven to treat withdrawal and reduce but not widely used, such as medications like buprenorphine and methadone.”

Thakrar and Delgado also suggest that hospitals could be incentivised to reduce discharges BMA and to support specialty services such as addiction consult services that have been proven to reduce BMA discharges and that can reduce the risks of future readmission or death.

Source: Penn University Medicine

Categories : Injury & Trauma PaediatricsTags :

Vitamin D Supplements don’t Prevent Fractures in Children

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Photo by cottonbro studio

A major clinical trial has found that vitamin D supplements do not increase bone strength or prevent bone fractures in children with vitamin D deficiency. The findings, published in Lancet Diabetes & Endocrinology, challenge widely held perceptions relating to the effects of vitamin D on bone health.

Around one-third of children have at least one fracture before the age of 18. This is a major global health issue, as childhood fractures can lead to life years of disability and/or poor quality of life. The potential for vitamin D supplements to improve bone strength has attracted growing interest in recent years, based on vitamin D’s role in promoting bone mineralisation. But there have been no clinical trials to test whether vitamin D supplements can prevent bone fractures in children.

Working with partners in Mongolia, a setting with a particularly high fracture burden and where vitamin D deficiency is highly prevalent, researchers from led by Queen Mary University of London and the Harvard T.H. Chan School of Public Health conducted a clinical trial to determine if vitamin D supplementation would decrease risk of bone fractures or increase bone strength in schoolchildren.

This study is also the largest randomised controlled trial of vitamin D supplementation ever conducted in children. Over the course of three years, 8851 schoolchildren aged 6-13 living in Mongolia received a weekly oral dose of vitamin D supplementation.

Testing revealed that 95.5% of participants had vitamin D deficiency at baseline, and study supplements were highly effective in boosting vitamin D levels into the normal range. No effect was seen on fracture risk or on bone strength, measured in a subset of 1438 participants using quantitative ultrasound.

The trial findings are likely to prompt scientists, doctors and public health specialists to re-consider the effects of vitamin D supplements on bone health.

Dr Ganmaa Davaasambuu, Associate Professor at the Harvard T.H. Chan School of Public Health, said:

“The absence of any effect of sustained, generous vitamin D supplementation on fracture risk or bone strength in vitamin D deficient children is striking. In adults, vitamin D supplementation works best for fracture prevention when calcium is given at the same time – so the fact that we did not offer calcium alongside vitamin D to trial participants may explain the null findings from this study.”

Professor Adrian Martineau, Lead of the Centre for Immunobiology at Queen Mary University of London, added:

“It is also important to note that children who were found to have rickets during screening for the trial were excluded from participation, as it would not have been ethical to offer them placebo (dummy medication). Thus, our findings only have relevance for children with low vitamin D status who have not developed bone complications. The importance of adequate vitamin D intake for prevention of rickets should not be ignored, and UK government guidance recommending a daily intake of 400 IU vitamin D remains important and should still be followed.”

Source: Queen Mary University of London

Categories : Healthcare Politics and RegulationsTags :

BHF Responds to the Imminent Approval of the NHI Bill

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The National Health Insurance (NHI) Bill was approved by the National Council of Provinces today and is due to be signed into law by the president shortly after. The Board of Healthcare Funders (BHF) is deeply disappointed. We are not happy with the various sections of the Bill. Despite submissions to government in this regard, the recommendations of the BHF and other stakeholders have largely been ignored and the bill is being passed virtually unchanged from its originally drafted form.

While the BHF fully supports the concept of universal health coverage (UHC) as defined by the World Health Organization (WHO) and believes that it must be a strategic imperative for all those directly or indirectly involved in healthcare, it does not support the NHI Bill in its current form. The bill restricts medical schemes to the provision of complementary cover potentially rendering them unsustainable, further to which the enormous economic value that medical schemes currently add to the health sector would be lost to South Africa if the bill goes ahead unchanged, BHF strongly believes this section should be removed as well as all references to complementary cover contained in the bill.

Additionally, a number of the Bill’s provisions are unconstitutional. These were detailed in the BHF’s submission to government. South Africa needs a strong, vibrant private health sector because government resources will never be unlimited. . The incredibly wide powers it bestows on the Minister of Health grossly undermine the board of the NHI fund and its accountability. The power of the Benefit Advisory Committee to determine health benefits under NHI similarly undermines this accountability. In addition, the BHF is perturbed by the demonstrated inability of the state to adequately operate national public entities and state-owned enterprises, as well as the endless levels of relentless corruption in the public sector.

Other concerns

The Bill allows the Minister of Health and the NHI fund to issue directives that override all other legislation, except the PFMA and the Constitution, including legislation specifically mandated by the Constitution.

There are proposed amendments to the Medical Schemes Act that unfairly discriminate against pregnant women. 

In many instances, the language of the bill creates significant legal uncertainty, which is itself unconstitutional due to the principle of the rule of law upheld by the Constitution. The BHF provided specific examples of this in the body of its submission.

The NHI Bill allows the national sphere of government to encroach on the geographical, functional and institutional integrity of provincial governments. This is not permitted by Section 41 of the Constitution.

The bill tries to dictate to the President in Cabinet (the National Executive) what new legislation must be made or how to amend existing legislation. This is also unconstitutional, as the Constitution itself grants the National Executive its powers. Nothing can change this except an amendment to the Constitution.

The registration system proposed by the bill creates unconstitutional barriers to access to health care that do not currently exist. The certification, accreditation and contracting system proposed by the bill is unwieldy, and it too will create unconstitutional barriers to access to health care that currently do not exist. Both these points are explained further in the body of the BHF’s submission.

Dr Katlego Mothudi, BHF CEO, underscores these serious implications of the bill’s being passed unchanged. “We have consistently given input into this proposed law and are disappointed that our concerns and those of other stakeholders appear not to have been considered or even tested. The bill in its current form will have a negative impact on healthcare access for everyone. There are many areas of uncertainty that have not been clarified, not least with regard to funding and affordability. We are also concerned specifically that the bill may prejudice the rights of women,” he says. “The proposed amendments to the Medical Schemes Act exclude access to pregnancy-related healthcare services for women who are medical scheme members. This means that these women would have to access reproductive health care from the public sector at their own cost, which is in conflict with the provisions of the National Health Act.

“The bill also has the potential for a wider negative economic impact. There is still uncertainty around how the NHI will be funded, but it will very likely be through additional taxation, something that will unavoidably have a detrimental effect on the economy at large – companies, individual employees and the general public – in the form of job losses. This phenomenon has already been discussed in various papers, including one published by the World Bank in 2001. It cited Colombia’s experience in this regard. A 10% increase in payroll taxes resulted in a 4.9% reduction in employment. Those who remained employed experienced a reduction in their disposable income, while the decrease in the overall number of employees saw a reduction in revenue from personal tax. Should the bill pass in its current form, South Africa will almost certainly experience something very similar.

“More specifically, this phenomenon will also impact the health sector. With medical schemes reduced to providing only complementary cover, not only will the schemes industry itself shrink, but all the other private entities it does business with, including hospitals, pharmaceutical companies and health practitioners,” he concludes.

Provided by the Board of Healthcare Funders

Categories : Neurodegenerative DiseasesTags :

Light Therapy may Improve Symptoms of Alzheimer’s Disease

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New meta-analysis included 15 randomised controlled trials involving 598 patients with Alzheimer’s disease and found improvements in sleep and psycho-behavioural symptoms.

Photo by Matteo Vistocco on Unsplash

Light therapy leads to significant improvements in sleep and psycho-behavioural symptoms for patients with Alzheimer’s disease, according to a new study published this week in the open-access journal PLOS ONE by Qinghui Meng of Weifang Medical University, China, and colleagues.

The cognitive decline associated with Alzheimer’s disease is often accompanied by sleep disturbances and psycho-behavioural symptoms including apathetic and depressive behaviour, agitation and aggression. Photobiomodulation is a non-pharmacological therapy that uses light energy to stimulate the suprachiasmic nucleus (SCN), a sleep modulator in the brain. Despite light therapy receiving increased attention as a potential intervention for Alzheimer’s, a systematic evaluation of its efficacy and safety has been unavailable.

In the new study, researchers searched multiple research databases to identify all randomised controlled trials related to light therapy intervention for Alzheimer’s disease or dementia. Fifteen high-quality trials with available methods and relevant outcomes were selected for further analysis. The included trials were written in English, published between 2005 and 2022, and performed in seven countries. They included a combined 598 patients.

The meta-analysis of all fifteen trials found that light therapy significantly improved sleep efficiency, increased interdaily stability (a measure of the strength of circadian rhythms), and reduced intradaily variability (a measure of how frequently someone transitions between rest and activity during the day). In patients with Alzheimer’s disease, light therapy also alleviated depression and reduced patient agitation and caregiver burden.

Given the limited sample sizes in studies included in this meta-analysis, the authors advocate for larger future studies, which could also explore if bright light exposure could cause any adverse behaviour in patients. They conclude that light therapy is a promising treatment option for some symptoms of Alzheimer’s disease.  

The authors add: “Light therapy improves sleep and psycho-behavioral symptoms in patients with Alzheimer’s disease and has relatively few side effects, suggesting that it may be a promising treatment option for patients with Alzheimer’s disease.”

Provided by PLOS One

Categories : Cardiovascular DiseaseTags :

Endovascular Catheter Safely Treats Acute Pulmonary Embolism

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Credit: Scientific Animations CC4.0

The BASHIR™ Endovascular Catheter (THROMBOLEX, Inc.), recently approved by the US Food and Drug Administration (FDA), is paving the way to more effective and safe treatment for acute pulmonary embolism. Already demonstrated to be effective for reducing blockages in lung arteries, a new study in JACC: Advances shows that the BASHIR™ catheter also reduces blockages in the smaller segmental pulmonary artery branches. These branches are ultimately responsible for oxygenating the blood in the lungs.

The new study from Temple University which was part of the National Institutes of Health-sponsored multicentre RESCUE clinical trial, further showed a correlation between decreased numbers of blockages in the small lung arteries and functional recovery of the right ventricle of the heart, which pumps blood into the main pulmonary artery of the lungs.

Compared to other devices, the BASHIR™ catheter also had significantly lower bleeding rates, a key advance in acute pulmonary embolism treatment.

“Blockages in these smaller, distal pulmonary arteries have never previously been explored in patients treated for acute pulmonary embolism,” explained Riyaz Bashir, MD, FACC, Professor of Medicine, Director of Vascular and Endovascular Medicine in the Section of Cardiology, Department of Medicine at the Lewis Katz School of Medicine and Temple University Hospital, co-inventor of the BASHIR™ Endovascular Catheter, and first author on the new report.

The BASHIR™ catheter is a small tube-like device that consists of a helical basket with six mini-infusion catheters at its farthest end.

When the infusion basket is expanded within a clot in a large blood vessel, new channels open, enabling blood to flow through the clot. The blood carries the body’s clot-dissolving chemicals into the channels, accelerating clot breakdown.

Researchers at THROMBOLEX™ Inc. were involved in developing and commercialising this platform technology.

The occlusion of small lung arteries is the major reason for the reduction in blood flow in patients with acute pulmonary embolism.

“The more occlusions a patient has, the lower their survival,” Dr Bashir said.

“Among the treatment goals is relieving obstructions in both large and small arteries.”

Patients who survive may, over more extended periods, be at high risk of developing chronic thromboembolic pulmonary hypertension (CTEPH), which is a life-threatening condition caused by increased blood pressure in the lungs.

In the new study, Dr Bashir and colleagues observed reductions in occlusions in segmental and proximal branches of the pulmonary artery 48 hours following treatment with the BASHIR™ catheter.

The blockages decreased even in those arteries that were distant from where the infusion basket was located, enabling improved blood flow and healing of the right ventricle.

“We suspect that the improvements in blood flow are due to both the expansion of the basket and the flow of the body’s clot dissolving molecules into the clot, which cause the blockage to shrink,” Dr. Bashir said.

“As the volume of blood flow improved, right ventricular function also improved, which could translate to better survival.”

In future work, Dr Bashir and colleagues plan to investigate mechanisms behind the observed reductions in arterial blockage.

Further trials are also needed to understand better the impact of treatment with the BASHIR™ catheter on patient survival and incidence of CTEPH.

Source: Temple University Health System

Categories : Cardiovascular DiseaseTags :

The South African Heart Association Prioritises Access to Cardiovascular Medicines and Devices

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On 29 October, during its annual scientific meeting at the Sandton Convention Centre in Johannesburg, the South African Heart Association (SA Heart®) met with members of the National Department of Health (NDoH), Council for Medical Schemes (CMS), South African Medical Association, medical aid industry, pharmaceutical and medical device industry, as well as the Global Heart Hub – which supports patient advocacy for cardiovascular disease – with the intention to develop an advocacy forum to create a more empowered and engaged community of patients living with cardiovascular disease.

During the symposium, SA Heart® emphasised the cardiac profession’s difficulty in obtaining approval and full reimbursement for the evidence-based treatments, shown to improve patient outcomes. The rules and regulations surrounding the availability of treatments, variously determined by the NDoH, the CMS and funders, currently inhibit access to effective cardiovascular disease management. SA Heart®, an affiliate member of the European Society of Cardiology (ESC), adopts the ESC’s guidelines as its own, supplemented by consensus statements that take prevailing local circumstances into account. Instead of following the up-to-date recommendations of the ESC, both the NDoH and various private sector funders rather give preference to the CMS algorithms that are years out of date. These anachronisms determine standards of care, despite ongoing advances in evidence-based medicine.

Prescribed Minimum Benefits (PMB’s) are a set of defined benefits to ensure that all medical scheme members have continuous access to defined minimum health services, regardless of the benefit option they select. PMB’s are limited in their reach. Private sector funders often retreat behind rules and regulations within the state sector, as to the type and level of treatment that they approve. Each funder independently determines what will be funded and at what level. Based upon their unique Health Technology Assessments, each funder then decides what treatment is approved or declined. It is unfair to restrict treatment to what is available only in the state sector. Novel therapies could be more widely available if there were more constructive agreements between state and industry. The CMS is currently reviewing the PMB package and defining patient entitlement.

SA Heart® pointed out that physicians are at the interface between the patient and the funder. Frequently, the patient misunderstands their own relationship with their medical aid and may deem the practitioner responsible for limitations imposed on his/her treatment. The administrative burden on clinicians is often intolerable, requiring repeat motivations, chronic medication forms, PMB applications and telephone calls to funders.

During the symposium pertinent questions were raised around funder and regulator reimbursement decisions, as well as the obscure mechanisms governing the pricing of medicines and devices. SA Heart® also raised concerns about the future of both the public and private healthcare system. A balance between the two sectors is a delicate one, requiring careful policy decisions to ensure that both systems will contribute effectively to the overall health of our country. Debates often focus around issues of equity and quality of service. The idea of centralizing decisions and distribution in the pharmaceutical and medical device sectors can be seen as an effort to ensure equitable access to essential medicines and equipment. However, this approach could negatively impact the ability to cater for South Africa’s diverse and specific needs – with more bureaucracy and inefficiency.

The meeting was called to determine interest amongst the various parties in establishing a forum to find solutions to these problems, and to harmonise the regulations around access to both medications and devices. The panellists were unanimous in their agreement that the current rules, regulations and disparities between the public and private sectors need to be reworked and there was consensus that a forum should be created to continue these discussions and arrive at effective, durable solutions. SA Heart® committed to driving this process, and will regularly engage all stakeholders in future meetings, with the ultimate goal of improving patient access to innovative cardiovascular treatments.

Categories : Medical Industry New Compounds and TreatmentsTags :

Christmas Came Early with These FDA Approvals

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AI art generated by GenCraft.

After the stupendous effort for COVID vaccines and treatments, it may seem like other diseases were being neglected. Nevertheless, the US Food and Drug Administration suddenly had a fire lit underneath it, and got cracking with accelerated drug approvals. Now, 2023 seems to have brought plenty of new drugs to bolster the physician’s armamentarium – some are the first-ever treatment for their indications. Hopefully, with FDA and European Medicines Agency (EMA) approvals, South African approvals should not be too far behind.

Since the pandemic, hotly anticipated drugs have made a big splash or sunk without a trace. In 2021, semaglutide was approved for weight management, unleashing a wave of people using (and some abusing) the GLP-1 agonist for weight loss. Adagrasib, which targets KRAS, previously thought undruggable, was a major advance for the treatment of non-small-cell lung cancer and was one of a few notable new non-COVID pharmaceuticals.

Aducanumab/Aduhelm was the top tip for new drugs in 2021, but turned out to be an absolute debacle: it wound up being an astronomically expensive, mostly ineffective drug with significant side effects. There were even questions raised over how it got approved in the first place.

Alzheimer’s disease

Last year, Aduhelm seemed like yet another false start in the long battle against Alzheimer’s disease. This year though, it looks like help finally arrived for fight against the dreaded neurodegenerative disease with not one but two breakthrough drugs, both  antiamyloid antibodies.

Up first is lecanemab/Leqembi from Eisai/Biogen. It targets the buildup of amyloid proteins in the brain, which otherwise lead to the formation of amyloid plaques and neurofibrillary tangles of tau protein, the hallmarks of the disease.

The other candidate is donanemab, which did not secure FDA approval last year, after pharma company Eli Lilly witnessed the disaster that was Aduhelm. It did show a reduction in decline in one measure of Alzheimer’s disease but not another, so its effects are a mixed bag.

Like Aduhelm, donanemab and lecanemab both have a serious downside: brain swelling, which claimed the lives of at three donanemab trial participants.

RSV

Previously minimised by the pandemic’s social distancing and routine masking, respiratory syncytial virus (RSV) experienced a resurgence in the wake of lifting these restrictions. RSV afflicts primarily those over 60 and young children. Among those 65 and older with RSV in the US, the Centers for Disease Control estimated 120 000 annual hospitalisations, with up to 10 000 of whom dying. Among children under 5, the figures are 58 000 annual hospitalisations and 100 to 300 deaths. Historically, RSV vaccine developments wound up being ineffective. Fortunately, this year saw the first approval for an RSV vaccine. A 120µg dose of their Arexvy vaccine produced statistically significant and clinically meaningful reductions in cases of lower respiratory tract disease caused by RSV in adults aged 60 years and older. Pfizer and Moderna are hot on HSK’s heels with their own vaccine applications.

Age-related macular degeneration

Apellis got an approval for pegcetacoplan this year, for geographic atrophy (GA) secondary to age-related macular degeneration, in its intravitreal injection. This is the first and so far only treatment for this indication. “The approval of SYFOVRE is the most important event in retinal ophthalmology in more than a decade,” said Eleonora Lad, MD, PhD, lead investigator for the phase 3 study. “Until now, there have been no approved therapies to offer people living with GA as their vision relentlessly declined. With SYFOVRE, we finally have a safe and effective GA treatment for this devastating disease, with increasing effects over time.”

Interestingly, Apellis also got an approval for paroxysmal nocturnal haemoglobinuria (PNH) with a patient-injectable version of pegcetacoplan. The disease results from the destruction of red blood cells by the immune system.

Lymphoma

Abbvie and Genmab’s epcoritamab, for certain cases of large B-cell lymphoma (LBCL), got accelerated FDA and EMA approval earlier this year. The FDA has also granted accelerated approval to Roche’s glofitamab. The drugs bind to binding to CD20 on malignant B cells and CD3 on T cells to kill cancer cells, creating an effect like CAR-T cell therapy but without the complexity (and presumably, cheaper too).

Major depressive disorder, postpartum depression

Mental health is full of gaps needing to be filled by effective treatments. Not much has made been added for depression since selective serotonin reuptake inhibitors (SSRIs) came onto the market in the 1990s. Zuranolone, from Biogen and Sage Therapeutics, is the first oral treatment for postpartum depression, which previously was treated only by IV injection in a healthcare facility. Unlike slow-acting SSRIs, this treatment, which targets the GABA-A receptor, is a short course.

Inflammatory bowel disease

There has been a steady drip of new biologic drugs for inflammatory diseases, such as bimekizumab (psoriasis and deucravacitinib which recently received FDA approval. Eli Lilly entered this crowded marketplace with ixekizumab. Now, after trouncing Novartis’ Cosentyx for psoriasis with its own mirikizumab, it pulled its application for that indication and switched it to ulcerative colitis – beating about a dozen competitors to be the first IL-23 inhibitor. It aims to get an approval for Crohn’s disease in 2025. Pfizer’s etrasimod for ulcerative colitis got approval in October 2023, and should receive EMA approval in 2024. Its phase 3 trial achieved 27% remission versus 7.4% for placebo.

Pulmonary arterial hypertension

Last is sotatercept, a new drug for pulmonary arterial hypertension (PAH), which previously had no real treatment. Unlike the current therapy aimed at simply dilating blood vessels, sotaracept targets BMPR-II signalling, addressing the cause of PAH. It earned a priority preview by the FDA based on its phase 3 trial data, with possible approval by March 2024.