NHS Approves "World's Most Expensive Drug" To Save Life Of 10-Month-Old Child
Type 1 SMA begins to affect babies before 6 months of age. Image Credit: Sopotnicki/Shutterstock.com
The NHS have granted approval for the use of the world’s most expensive drug, Zolgensma, on a 10-month-old child with severe spinal muscular atrophy (SMA). If successful, the drug could prolong baby Edward’s life expectancy from just two years to significantly longer, and allow him independence never before seen for patients with the lifelong disease.
The news comes after a long and arduous campaign by Edward’s mother, Megan Willis, to allow her child to be treated with the new gene therapy. However, sitting at an astonishing $2.48 million (£1.79 million) for just one treatment, it has been a struggle to gain approval on the NHS. In diseases such as SMA, time is a key predictor of prognosis – get treatment early in disease progression, and the outcome is far better, so each day counts.
“I’m exhausted. It’s been such a long ride and a rollercoaster,” said Megan, speaking to the Guardian.
“When I think back to myself in November, newly diagnosed, I didn’t think there was an option for him – I thought he was dying because that’s that all it said when I read up on SMA, that 95% of children die, or are severely disabled. I didn’t think he had a future.”
Spinal muscular atrophy is a genetic disorder that varies in severity, but at its most severe it causes muscle weakness, movement problems, difficulty breathing, and eventual death. The symptoms get worse with age, and affected children often pass due to respiratory failure as young as two years old. Most commonly inherited from the parents, SMA is thought to be caused by mutations within the SMN1 gene, which codes for the survival motor neuron protein (SMN), a protein that is vital for maintenance of motor neurons. As the gene is mutated, SMN production is decreased and motor neurons begin to die, preventing signals from passing from the brain to the muscles.
In an attempt to combat the missing SMN, Zolgensma introduces a healthy copy of SMN1 using virus vectors into nerve cells. Once incorporated, this gene allows the cells to produce SMN once more, allowing the affected person to maintain nerve cells and halt disease progression.
Zolgensma was approved for use by the FDA in May 2019, and by NICE for use in the UK in March 2021. As SMA is a degenerative disorder, long-term data on the efficacy of Zolgensma is lacking, but early results suggest the single-dose therapy is able to vastly improve motor function in young children with type 1 SMA. Current therapies involve regular injections every four months for the lifetime of the patient, but Zolgensma claims to produce similar quality of life improvements from just a single treatment.
It is currently only approved for babies less than six months old, but cases are dealt with individually to allow use for older children such as Edward.
My patients deserve to know why drug costs keep going up | Opinion
By Dr. Max Cooper
Not long ago, a patient collapsed just steps away from the emergency department where I work as a physician. The patient’s heart stopped pumping due to dangerously low blood potassium levels. When the body lacks potassium, a condition known as hypokalemia, electrical signals that are critical to muscle functions, especially the heart muscle, can short circuit.
Thankfully, our team was able to quickly diagnose the problem and save our patient’s life. When he woke and we were able to speak to him, we learned the patient was rationing his medications because he couldn’t afford them. He was taking multiple medications to manage chronic conditions. Some of these medications lower potassium levels. He was prescribed a potassium supplement, but due to the overall cost of his prescriptions he decided to skip this important one — and it almost killed him.
This close call and countless other avoidable medical emergencies across Pennsylvania are why we need to rein in high drug costs. An important first step in this direction is more transparency in drug pricing so Pennsylvanians can get a better picture of why their medications keep getting more expensive, even unaffordable in many cases.
For this reason, Senate Bill 579 or House Bill 321, which sets up an independent panel to review the costs of prescription drugs, should become state law without delay.
For my patient with low potassium, life-saving medication was simply too costly. With the prices of other prescription drugs adding up, the patient was forced to choose which medication to skip. Potassium was the one to go, and rationing nearly killed him. For Pennsylvaians with common diseases such as cancer, the bill for medications can exceed $10,000 a year, pushing some families to bankruptcy.
While Pennsylvanians ration their care because they can’t afford their medications, putting their health and sometimes lives at risk, pharmaceutical companies continue to hike drug costs: In January alone, 832 drugs got more expensive by 4.5 percent. Since 2014, pharmaceutical costs have ballooned faster than any other cost increases in health care as a whole. With year-over-year wage growth stuck at a paltry 2 percent, paying for medications isn’t just getting more expensive, it’s becoming unaffordable.
As a physician, I want to know why the medications I prescribe to patients become more expensive every year and why the cost increases appear unrelated to efficacy and outcomes. My patients deserve to know this, too.
Senate Bill 579 and House Bill 321 can help Pennsylvanians get answers to these questions. A Prescription Drug Affordability Board would compile information about how drug prices are set, why costs go up and how drug corporations spend their profits. Transparency is the first step toward reining in high drug prices and holding pharmaceutical corporations accountable.
This panel could investigate why drug companies spend more money buying back their stocks than in making their products safer, more effective and more affordable. Stock buybacks are, after all, a financial shell game that enriches CEOs and top executives.
Amgen spent more than $7 billion in 2019 buying back its stocks, and only $4.1 billion that year on research and development. Between 2018 and 2020, Amgen CEO Robert Bradway’s compensation increased from $18.5 million to $20.1 million. Bristol Myers-Squibb spent $6.3 billion on R&D -- $1 billion less than it did on stock buybacks and nearly an additional $1 billion less than it spent on marketing, another spending category designed to increase prescriptions and profits, not safety and effectiveness.
In 2019, the drug industry spent $6.5 billion on advertising and marketing. The most tragic consequence of this dangerous dynamic is the painkiller prescription frenzy: Between 2013 and 2015, the drug industry spent $40 million marketing painkillers, and data links those ad dollars to higher prescription rates and ultimately more opioid deaths.
The billions of dollars changing hands for slick TV ads and stock buybacks shouldn’t hide the simple fact that the biggest beneficiaries of drug prices going up are pharma CEOs, not their average employees, who earned 31 times less than their C-suite bosses. And beneficiaries are definitely not people like my patient who nearly died from low potassium outside my emergency department.
Other states, such as Colorado and Maryland, see the value of greater transparency in drug pricing as a step to bringing down high prices. Pennsylvanians shouldn’t be left behind. Senate Bill 579 and House Bill 321 can help patients and their families torn between paying for medications or groceries.
Just as my team was able to diagnose the problem in our patient and save him, we can diagnose the problem with our health care system: unaffordable prescription drugs. The only question is if we have the courage to fix it and make the pharma giants take their medicine.
Dr. Max Cooper is Pennsylvania State Lead for the Committee to Protect Health Care, a national health care advocacy organization. He is a practicing Emergency Physician in Delaware county, Pa.
How data analytics reduces pharmacy benefits costs in the US
Nathan Holman, Vice President of Information Technology at RxBenefits, tells us how the company is using data analytics, AI and machine learning to reduce pharmacy benefits costs in the US healthcare sector.
What does RxBenefits provide?
RxBenefits is the benefits industry's first and only Pharmacy Benefits Optimizer (PBO). Most employers significantly overpay for pharmacy benefits while receiving suboptimal clinical management and customer service. In fact, over 60% of employers say their prescription drug spending is costly and unsustainable.
Our priority is to help self-insured employers maximise the value they and their members receive from their pharmacy investment. Our pharmacy experts work on behalf of employers, independently of their medical carrier or PBM, to lower their purchasing costs, eliminate wasteful spending, protect their employees’ health and safety, and boost employee satisfaction. Our model reduces pharmacy spend by more than 25% the first year, on average, and insulates the plan against future drivers of trend, like high-cost specialty medications.
How does RxBenefits use data?
Our PBO model leverages advanced Business Intelligence capabilities and data modelling, which is derived from our proprietary analytics platform, RxAnalyzer, to identify risk areas and trend drivers in a company’s claims data - such as chronic health conditions and high pharmacy utilisation - and to forecast cost trajectory and program impact. By applying clinical expertise to this data, we’re able to recommend hyper-targeted strategies to help the employer address any potential issues.
We’ve also started to incorporate AI/machine learning and predictive analytics into a new technology platform called ONE.RxB. This platform, along with our other capabilities, will enable us to analyse a broader array of employer trends, patient demographics, prescription patterns and more, as well as model that data, flag important insights and share recommendations to employers in real time - meeting a growing industry need for better access to data insights.
By advancing our data analytics capabilities we’ll provide employers with deeper insights into employee health trends and tools to make more informed decisions about their pharmacy benefits.
How important is data analytics in the pharmacy benefits industry?
Without data, an employer’s pharmacy benefits plan is a black box. The pharmacy benefits industry is currently very opaque, with many employers stuck with misaligned pharmacy contract terms or a lack of a pharmacy contract altogether.
It’s essential for employers to have transparent contract terms as well as full visibility into their plan’s performance in order to improve the health of their employee population at the lowest cost. Applying data analytics is critical to gaining this visibility.
When data analytics is applied to pharmacy benefits, employers can better evaluate their pharmacy benefits options, gain insights into their plan’s performance and utilisation, uncover potential risks, and make better decisions about their benefits programs. They can also analyse the cost and employee impact of any decisions before they’re made.
Can you give us an example of this?
With insights derived from our proprietary data analytics platform, RxAnalyzer, we forecast the cost-savings and member impact of each recommended strategy for our clients. With this knowledge, they are empowered to make the best decisions for their plan and their members.
They can construct benefits that are attractive to both current and future employees, optimise their wellbeing, and reduce overall prescription drug costs. In fact, most employers are able to reduce their pharmacy spend by more than 25% on average the first year and protect themselves against future drivers of increased spend, like the rising cost and utilisation of specialty medications.
What key trends are emerging in the pharmacy benefits industry?
There are several trends, some of the top ones to look out for are:
Specialty Carve-Out. We are continuing to see more innovative specialty drug therapies hit the market every year, and use of these drugs increase. Just 1% of prescription claims now account for roughly 50%-60% of an employer’s plan costs, driven in large part by high-cost specialty medications. In order to address the impact on employers, we’re seeing a rise in employers exploring specialty carve-out programs.
Unfortunately, this practice can negatively impact members, who can be left out in the cold with no way of securing their necessary medications and employers are still left footing the bill. There are alternatives to specialty carve-out that protect both employers and their members, however. As specialty drugs continue to rise in both cost and utilisation, employers need to re-evaluate their pharmacy benefits strategy and utilize an independent pharmacy expert who will operate in the best interest of them and their members.
Hospital Costs Rising: Hospital revenues declined significantly last year, and they’re expected to lose up to $122 billion again this year. Meanwhile, their drug spend and utilisation continues to rise. In fact, 70% of a hospital’s plan members take prescriptions each year, which is 25% higher than the typical commercial plan.
On top of that, 1 in 6 hospital plan members take a higher-priced drug. Hospitals play a dual role as both healthcare provider and employer and their employees expect a rich benefit, so it’s important for them to carefully design a drug plan that balances access and cost while ensuring a top service experience. This includes promoting the hospital’s in-house pharmacy, ensuring appropriate prescribing and drug utilisation, implementing processes to eliminate wasteful spending on high-cost brand and specialty medications, and gaining access to Manufacturer Copay Assistance Programs (MCAP) and 340B Drug Pricing Discounts, if eligible and available.
As these trends continue to emerge over the next few years, partnering with an independent advocate who proactively reviews your benefits plan on a regular basis can ensure employers consistently have the best rates, rebates and contract terms.
Do you think "virtual pharmacies" will be a standard way for people to get their prescriptions in the future?
Virtual pharmacies accelerated during COVID-19 as patients were in need of more convenient healthcare offerings. For example, last year Amazon announced a remote retail pharmacy solution that will accept insurance and be in PBM pharmacy networks, leveraging PillPack, an online pharmacy that fills prescriptions by mail.
Costco also recently announced a new online pharmacy, and many traditional “brick & mortar” pharmacies are now offering this option as well. It is likely that more options like this will continue to become available.
However, virtual pharmacies are simply another option employees and members have for filling their brand and generic drug prescriptions. There are often key differences between a virtual and in-person pharmacy, and benefits to either option. Employers should help inform their members of the pros and cons and differences of each kind of offering before changing their pharmacy benefits plans.