Tag: drug approval

Categories : Expert Opinion Healthcare Politics and RegulationsTags :

Addressing Barriers to Accessing Innovative Medicines in South Africa — A Critical Moment Amid Funding Cuts

On By ModernMedia

By Matimba Ngobeni, Country Head: Value & Access, Novartis South Africa


28 July 2025, Johannesburg South Africa – South Africa’s healthcare system stands at a crossroads. Despite the promise of progress outlined in the Budget Speech and the Presidential Health Compact, the reality on the ground reveals persistent and growing barriers to accessing innovative medicines.

Economic pressures, funding constraints, and infrastructure gaps continue to undermine equitable healthcare delivery, particularly for vulnerable communities. What’s more, recent international developments—such as U.S. President Donald Trump’s cuts to funding that supported healthcare initiatives in South Africa—threaten to exacerbate these challenges, potentially limiting access to life-saving advanced therapies.[1]

Economic pressures

The cost of advanced therapies remains out of reach, and the structural inequalities in our healthcare system persist. While top-tier medical plans still provide access to advanced medicines, we are seeing a shift. Patients are moving to lower-tier plans or into the public system, simply because they cannot afford more. And with that shift, their access to advanced therapies disappears. [2]This is not a uniquely South African problem.

Globally, we see the same story repeat: private healthcare becomes a fortress that only those who can pay the toll may enter. Everyone else is left to rely on an overburdened public system, strained by funding shortfalls, infrastructure gaps, and critical workforce shortages. The public healthcare system, already overburdened, struggles to absorb this increased demand. Rising healthcare costs combined with limited household budgets create a perfect storm where affordability becomes the biggest barrier to accessing cutting-edge treatments.

Funding constraints and infrastructure challenges

Both private and public sectors face severe funding constraints. Innovative medicines, especially advanced therapies, come with high price tags that strain budgets and limit availability. At the same time, infrastructure and skills gaps hinder the effective delivery of these treatments. Investments in healthcare infrastructure, workforce training, and data management are urgently needed to support the growing demand for advanced therapies.

While it may seem like all hope is lost, the Presidential Health Compact offers a promising framework aimed at transforming South Africa’s healthcare landscape through infrastructure development and improved data surveillance[3]. However, it stops short of directly addressing access to innovative medicines. This gap underscores the need for stronger collaboration between public and private stakeholders to ensure that patients do not bear the financial burden alone.

Towards equitable access: Collaboration is imperative

Another way forward is through a robust, transparent Health Technology Assessment (HTA) process, where medicines are evaluated not only on their cost but on their ability to save lives, improve quality of life, and reduce the long-term burden on the health system.

Inclusive HTAs, where payers and pharmaceutical companies work together, are essential for reimagining access to advanced therapies. If we only look at the upfront cost of innovation, we miss the bigger picture of societal value.

Globally, risk-sharing models and outcome-based pricing agreements are helping bridge the affordability gap[4]. South Africa could benefit from more flexible legislation to enable these models, ensuring that innovation doesn’t remain locked behind prohibitive price tags.

South Africa’s healthcare future depends on what we choose to prioritise: short-term financial gains or long-term societal wellbeing. Too often, systems have been designed around protecting profits rather than protecting lives. Healthcare should never be a luxury. Yet in South Africa, and across much of the world, the reality is stark: exclusion is the norm, not the exception.

If we want a future where access to life-saving medicines is a reality for all, we need to break down the barriers of affordability, infrastructure, and policy inertia. And we need to do it together — governments, healthcare companies, funders, and civil society — because lives are at stake.

All hope is not lost. But we cannot wait for crisis to be our catalyst. The time for bold, collaborative action is now.

**About Novartis:**  

Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach more than 250 million people worldwide.

Reimagine medicine with us: Visit us at https://www.novartis.com/za-en/ and connect with us on LinkedIn, Facebook, and YouTube.

Novartis South Africa (Pty) Ltd, Magwa Crescent West, Waterfall City, Jukskei View, 2090. Co. Reg. No. 1946/020671/07. Tel. No. +27 (0) 11 347 6600. 

Disclaimer: The presentation may include data on formulations, products, indications, and dosages not yet approved by the South African Health Products Regulatory Authority. This information is not intended to be promoting nor recommending any formulation, indication, dosage, or other claim not covered in the approved Professional Information. Novartis South Africa (Pty) Ltd recommends the use of their products in accordance with the locally approved Professional Information. Views and opinions of speakers do not necessarily reflect those of Novartis.

To report an adverse event, email: patientsafety.sacg@novartis.com or report it directly through our website: www.novartis.com/report. Alternatively, call 0861 929 929 (PharmaCall).   To report product quality complaints, email: qa.phzais@novartis.com.  Alternatively, call 0861 929 929 (PharmaCall).

Content ID: FA-11474225       Approval date:  7/25/2025       Expiry Date: 7/25/2027

[1] US funding cuts threaten 39 research sites in South Africa

[2] Your Healthcare Financial Plan For 2025

[3] SONA 2025: Quality Healthcare for All

[4] Pricing and Reimbursement: Innovative Risk-Sharing Strategies

Categories : CancerTags :

FDA-Approved Drug Halts EBV-Driven Lymphoma by Disrupting a Key Cancer Pathway

On By ModernMedia
Swollen lymph nodes. Credit: Scientific Animations CC0

Scientists at The Wistar Institute have discovered that a class of FDA-approved cancer drugs known as PARP1 inhibitors can effectively combat Epstein-Barr virus (EBV)-driven lymphomas. The findings, published in the Journal of Medical Virology, demonstrate that these drugs, which work by blocking the activity of the PARP1 enzyme, can halt tumour growth by interfering with the EBV’s ability to activate key cancer-promoting genes.

“We’ve uncovered a completely different mechanism for how PARP inhibitors work in EBV-positive cancers,” said Italo Tempera, PhD, associate professor at Wistar’s Ellen and Ronald Caplan Cancer Center and senior author of the study. “Instead of preventing DNA damage from repairing itself in the tumours, like these drugs do in other cancers, they essentially cut off the virus’s ability to hijack cellular machinery to drive cancer growth. This opens up exciting possibilities for repurposing existing FDA-approved drugs to treat EBV-associated cancers.”

EBV infects over 90% of the global population. While most people with the virus remain symptom-free, immunocompromised individuals such as people with HIV and transplant recipients have an increased risk of EBV causing several types of cancer, including various lymphomas and carcinomas. Despite the virus’s clear role in driving these malignancies, no specific therapies currently target EBV-driven cancer.

In search of such a therapy, Tempera and his research team focused on PARP1, a cellular protein that is known primarily for its role in DNA repair. In cancer treatment, PARP inhibitors typically work by preventing cancer cells from repairing their DNA, causing them to die. However, Tempera’s team had previously discovered that PARP1 plays a very different role in EBV infection: It helps control which genes are accessible and active, essentially acting as a master regulator of gene expression.

“Think of PARP1 as a key that opens up DNA to make certain genes readable,” explained Tempera. “EBV uses this key to unlock cancer-promoting genes. When we block PARP1, we’re essentially taking away the key so the virus can’t get in and use our DNA for its own purposes.”

Using a mouse model of EBV-driven lymphoma, the researchers treated the animals with BMN 673 (talazoparib/talzenna), a PARP inhibitor that has already been approved for breast cancer treatment. Compared to controls, the treated mice showed an 80% reduction in tumour growth, and the cancer’s ability to spread to other organs was significantly reduced. Further, when the team analysed the tumours, they found no increase in DNA damage in the treated animals – the hallmark of how PARP inhibitors typically work. Instead, they discovered that PARP1 inhibition disrupted a critical partnership between the viral protein EBNA2 and the cellular oncogene MYC.

“EBNA2 is like the conductor of an orchestra, directing cellular genes to play a cancer symphony,” said Tempera. “It specifically turns on MYC, which is one of the most important cancer-promoting genes. When we inhibit PARP1, EBNA2 can’t effectively activate MYC anymore, and the whole cancer program falls apart.”

The findings have significant therapeutic implications. Because PARP inhibitors are already FDA-approved and their safety profiles are well established, the path to clinical application could be accelerated compared to developing entirely new drugs.

The research also suggests this approach might work beyond EBV-associated lymphomas. The team is now investigating whether PARP inhibitors could be effective against other EBV-driven cancers, including nasopharyngeal and gastric carcinomas. Additionally, given EBV’s suspected role in autoimmune diseases, the researchers are exploring whether PARP1’s regulation of viral gene expression might contribute to these conditions.

“This work really showcases the power of understanding fundamental viral biology,” said Tempera. “We’re taking insights from basic virology research and translating them into potential therapies. With further development, this approach could provide new hope for patients with EBV-associated cancers who currently have limited treatment options.”

Source: Wistar Institute

Categories : Cardiovascular DiseaseTags :

BMJ Finds Inaccuracies in Key Studies for AstraZeneca’s Blockbuster Heart Drug Ticagrelor

On By ModernMedia

Investigation finds evidence of serious misreporting, raising fresh doubts over the approval and decade long use of ticagrelor

Photo by National Cancer Institute on Unsplash

In a follow up investigation into the multibillion dollar drug ticagrelor, The BMJ has uncovered fresh concerns, this time in key platelet studies used in its FDA approval.

For more than a decade, ticagrelor (Brilinta in the US and Brilique in Europe) has been recommended for patients with acute coronary syndrome – a range of conditions related to sudden reduced blood flow to the heart.

Last December, an investigation by The BMJ found serious data integrity problems in the landmark clinical trial (PLATO) that was used to gain worldwide approval for ticagrelor, calling into question the drug’s advantage over cheaper rivals.

Now, as generic versions of the drug prepare to launch this year, The BMJ has expanded its investigation, looking at two key platelet studies that AstraZeneca claimed explained ticagrelor’s ability to treat acute coronary syndrome successfully.

It finds that the “primary endpoint” results (the trial’s key measurement) for both clinical trials were inaccurately reported in the leading cardiology journal Circulation, and reveals that more than 60 of 282 readings from platelet machines used in the trials were not present in US Food and Drug Administration (FDA) datasets.

What’s more, one active trial investigator never became a study author, while one author told The BMJ he was not involved in the trial, and most investigators, including the principal investigator, were unreachable or declined to be interviewed.

Victor Serebruany, an adjunct faculty member at Johns Hopkins University and ticagrelor’s most renowned critic, told The BMJ that “there are episodes of skyrocketing rebound and profound platelet inhibition after ticagrelor making patients prone to thrombosis or bleeding. If doctors had known what happened in these trials, they would never have started using ticagrelor.”

Circulation and AstraZeneca did not respond to a request for comment.

Serebruany added: “It’s been obvious for years that there is something wrong with the data. That the FDA’s leadership could look past all these problems—on top of the many problems their own reviewers identified and are now being discovered by The BMJ—is unconscionable. We all need to know how and why that happened.”

Source: BMJ

Categories : Diseases, Syndromes and Conditions New Compounds and TreatmentsTags :

FDA Approves First Drug Designed to Treat Indolent Systemic Mastocytosis

On By ModernMedia
Photo by Rodion Kutsaiev on Unsplash

Systemic mastocytosis (SM) is a rare haematologic disorder that can lead to a range of debilitating symptoms across multiple organ systems and a significant impact on patients’ quality of life, and now the first medicine has been approved to specifically treat the most common form of the disease. On Monday 22 May, the US Food and Drug Administration (FDA) approved AYVAKIT® (avapritinib) to treat indolent systemic mastocytosis (ISM) in adults.

ISM represents the vast majority of SM cases, and AYVAKIT is now available for adults with ISM at the recommended dose of 25mg once daily. AYVAKIT was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease. AYVAKIT has been FDA approved for the treatment of advanced SM since June 2021.

“After decades of caring for people with indolent systemic mastocytosis, I have seen firsthand its profound impact on patients’ underlying mast cell burden, symptoms, physical and mental health, and ability to work and participate in daily activities,” said investigator Cem Akin, MD, PhD, Professor of Medicine at the University of Michigan. “Despite the use of multiple supportive care treatments, a considerable number of patients with indolent systemic mastocytosis continue to experience a substantial disease burden. AYVAKIT advances the treatment of indolent systemic mastocytosis by targeting KIT D816V, the primary underlying cause of the disease, and establishes a new standard of care for a broad population of patients with this disorder. AYVAKIT delivered statistically significant and consistent clinical improvements in the PIONEER trial, and based on these practice-changing data, I feel a tremendous sense of hope for the future for all those affected by the disease.”

The approval of AYVAKIT in ISM is based on data from the double-blind, placebo-controlled PIONEER trial – the largest study ever conducted for this disease – in which patients received AYVAKIT 25mg once daily plus best supportive care (AYVAKIT) or placebo plus best supportive care (placebo). AYVAKIT demonstrated significant improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden.

AYVAKIT was well-tolerated with a favourable safety profile compared to placebo, and most adverse reactions were mild to moderate in severity. The most common adverse reactions for AYVAKIT (≥10%) were eye oedema, dizziness, peripheral oedema and flushing. Serious adverse reactions and discontinuations due to adverse reactions occurred in less than 1% of patients.

Detailed results from the PIONEER trial, including open-label extension study data showing the clinical benefits of AYVAKIT through 48 weeks of treatment, were presented in February 2023 at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting.

Source: Blueprint Medicines

Categories : Ethics and Law Medical IndustryTags :

Did the FDA Break its Own Rules in Approving New Antibiotic?

On By ModernMedia
Photo by Rodion Kutsaiev on Unsplash

In the US, drug approval needs “substantial evidence” of effectiveness – but an investigation by The BMJ into the recent approval of the antibiotic Recarbrio from Merck suggests that these standards are being bypassed.

Recarbrio is a combination therapy made up of a new beta-lactamase inhibitor (relebactam) and a decades old Merck antibiotic (imipenem-cilastatin) to treat complicated infections. It costs $4000–$15 000 for a course, compared with a couple of hundred dollars for the generic version of Merck’s old antibiotic.

Peter Doshi, senior editor at The BMJ, describes how US Food and Drug Administration (FDA) scientists had serious doubts about its highly expensive Recarbrio but the agency approved it anyway.

Did the FDA break its own rules in approving this antibiotic, and what does this case tell us about problems within the agency, he asks? 

In its FDA application, Merck submitted results from two clinical trials comparing Recarbrio with imipenem in adults with complicated urinary tract infections and in patients with complex intra-abdominal infections.

But FDA reviewers noted that Merck had studied the wrong patient population to evaluate the added benefits of the new drug, and said the trial for urinary tract infections showed that Recarbrio was as much as 21% less effective than the older, cheaper imipenem.

The FDA concluded that “these studies are not considered adequate and well-controlled.” And of a third clinical study, the FDA called it a “very small,” “difficult to interpret” “descriptive trial with no pre-specified plans for hypothesis testing.”

Yet despite all three clinical studies not providing substantial evidence of effectiveness, FDA approved Recarbrio.

“Instead of basing its decision on the clinical trials in Merck’s application, FDA’s determination of Recarbrio’s efficacy was justified on past evidence that imipenem was effective, plus – to justify the new relebactam component – in vitro (lab) studies and animal models of infection rather than evidence from human trials as required by law,” writes Doshi.

Others are concerned that Recarbrio’s approval essentially amounts to a return to a way of regulating medicines that the FDA abandoned a half century ago prior to the agency’s “substantial evidence” standard.

Doshi explains that, under specific circumstances, the Director of the Center for Drug Evaluation and Research (CDER) can waive in whole or in part the FDA’s “adequate and well-controlled studies” approval criteria. But the FDA told The BMJ ”there was no center director memo in the file” for Recarbrio.

And when The BMJ contacted Janet Woodcock, CDER Director at the time, and now the FDA’s Principal Deputy Commissioner, she said she was not aware that clinical studies showed Recarbrio did not provide substantial evidence of effectiveness.

Woodcock was also unable to confirm that approvals of new drugs require at least one clinical study of the drug itself that demonstrates substantial evidence – evidence lacking in the case of Recarbrio.

A spokesperson for CDER told The BMJ that FDA “applied regulatory flexibility” in approving Recarbrio. 

It is unclear whether this regulatory flexibility enabled FDA to conclude Recarbrio had met the legal “substantial evidence” standard without “adequate and well-controlled investigations” of Recarbrio, says Doshi. FDA declined to answer the question, saying “We have no additional information to provide.”

The decline of science at the FDA has become unmanageable, argues David Ross, associate clinical professor of medicine at George Washington University, School of Medicine and Health Sciences, and former FDA medical reviewer, in a linked commentary.

He describes Recarbrio’s approval as “shocking” and says while much of the blame must go to the FDA’s reliance on industry paid user fees for around two-thirds of its annual drugs budget, “the corruption of the FDA’s scientific culture remains the primary culprit driving the deterioration of safety and effectiveness standards.”

To address this “dismal situation” he suggests tapering the FDA’s dependence on user fees and improving public access to the information received by the FDA, its reasoning, and its decisions.

“The Recarbrio approval is a sentinel event, warning of a return to an era when drug effectiveness was an afterthought,” argues Ross. “Although the FDA crowed about this approval, it would have been better advised to remember that “for a successful technology, reality must take precedence over public relations, for nature cannot be fooled,” he concludes.

Source: EurekAlert!

Categories : Ethics and Law Medical IndustryTags :

Systematic Bias in Industry-sponsored Cost-effectiveness Studies

On By ModernMedia
Photo by Marek Studzinski on Unsplash

Industry-sponsored studies on a new drug or health technology are more likely to be found ‘cost-effective’ than independent studies, across a range of diseases, according to findings from a study published in The BMJ.

In a linked editorial, experts make a call for better reporting of results, more transparency, open-source cost-effectiveness models, and more independent studies, to reduce decision makers’ reliance on potentially biased cost-effectiveness analyses.

A cost-effectiveness analysis (CEA) provided the manufacturer is required by some countries to weigh up a product’s costs and effects.

This cost analysis evidence can be used to set the price for a drug or health technology or decide whether insurance policies will cover them. New drugs covered by insurance plans can be much more profitable than those not covered, which could lead to bias in CEAs funded by the drug and technology manufacturing industry.

While previous studies have consistently shown sponsorship bias in CEAs, most studies were limited to specific diseases, and are out of date. To fill in the gaps, Feng Xie and Ting Zhou from McMaster University, Canada, analysed data from all eligible CEAs published between 1976 and March 2021. 

They selected CEAs that reported an incremental cost-effectiveness ratio (ICER) using quality-adjusted life years or QALYs – a ‘value for money’ metric of years lived in good health.

The authors used data from the Tufts Cost-Effectiveness Analysis Registry. In total, 8192 CEAs were included in the study, of which nearly 30% were sponsored by industry. 

The study defined CEA industry sponsorship as an analysis funded by drug, medical device, or biotechnology companies, either wholly or in part. 

The results show that the industry-sponsored CEAs were significantly more likely to conclude that the new medicine or health technology was cost-effective than those not sponsored by industry.

For example, industry-sponsored studies were more likely to report the intervention being studied as cost-effective below the commonly used threshold of $50 000 per QALY gained than non-industry sponsored studies.

Among 5877 CEAs that reported the intervention was more effective but more expensive than the comparator, the ICERs from industry sponsored studies were one third (33%) lower than those from non-industry sponsored studies.

While only having the registry information to work with was a limitation, the authors said their analysis provides a basis for comparison with previous investigations.

As such, they suggested that “sponsorship bias in CEAs is significant, systemic, and present across a range of diseases and study designs.”

In lower and middle-income countries, industry bias can increase drug prices, where fewer resources mean decision-makers often need to rely on published, rather than independent CEAs. 

In a linked editorial, Adam Raymakers at Cancer Control Research, Canada, and Aaron Kesselheim at Brigham and Women’s Hospital, USA, argue that decision-makers “should exercise caution when using published cost-effectiveness analysis in coverage decisions.”

They say finding solutions to tackle bias is more important than ever, and make the case for open-source analysis models, increased transparency, and increased funding for independent analyses, to help minimise reliance on industry-sponsored cost analyses.

Source: The BMJ

Categories : Pain ManagementTags :

New Migraine Prevention Drug Gets The Green Light from FDA

On By ModernMedia
Source: Usman Yousaf on Unsplash

Atogepant (Qulipta) has become the first oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) specifically developed for migraine prevention to win FDA approval, manufacturer AbbVie announced on Monday.

Following on after rimegepant, which is also indicated by the FDA for acute migraine treatment, atogepant became the second gepant approved for prevention of episodic migraine in adults.

The atogepant decision “reflects a broader shift in the treatment and management paradigm for the migraine community,” noted Peter Goadsby, MD, PhD, DSc, of the University of California Los Angeles and King’s College London.

“Qulipta provides a simple oral treatment option specifically developed to prevent migraine attacks and target CGRP, which is believed to be crucially involved in migraine in many patients,” said Dr Goadsby in a statement.
Atogepant has a high affinity at the CGRP receptor, and being a small-molecule drug it can be taken orally, unlike injectable anti-CGRP monoclonal antibodies approved for migraine prevention.
An oral CGRP-receptor antagonist is easier for patients, Goadsby noted when he presented data from atogepant’s pivotal phase IIb/III trial at the 2019 American Academy of Neurology annual meeting. “It could facilitate, with time, the greater use of this mechanism in primary care,” he told MedPage Today. “Primary care doctors will more easily use a medicine that’s relatively simple to use and well-tolerated, and that means more migraine patients can get treated.”

In the phase III ADVANCE trial, 873 participants were randomised to receive a once-daily dose of oral atogepant (10mg, 30mg, or 60mg) or placebo. After 12 weeks, average days with migraine per month dropped from baseline by 3.7 days with atogepant 10mg, 3.9 days with atogepant 30mg, 4.2 days with atogepant 60mg, and 2.5 days with placebo. The most common adverse events with atogepant were constipation and nausea, along with fatigue. 
Patients should notify their healthcare provider if they have kidney problems or are on dialysis, have liver problems, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed, AbbVie said.

Source: MedPage Today