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Infographic: Which Rare Diseases Are The Most Common?

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Rare Diseases Infographic

Infographic: Which Rare Diseases Are The Most Common?

Pharmaceuticals have come a long way since the apothecary days of prescribing cocaine drops for toothaches, or dispensing tapeworm diet pills.

Today, medical breakthroughs like antibiotics and vaccines save millions of lives, and contribute to the industry’s mammoth size. Yet even with rapid advancements, a select group of rare diseases still fly under the radar — and together, they affect over 350 million people worldwide.

What Are Rare Diseases?

Today’s infographic from Raconteur breaks down occurrence rates of notable rare diseases, and their collective impact on pharmaceutical drug sales. But first, let’s look at how they’re defined.

Diseases are considered rare, or “orphan” if they affect only a small proportion of the population. In general, it’s estimated that 1 in 17 people will be afflicted by a rare disease in their lifetime. At the same time, as many as 7,000 rare diseases exist, with more discovered every year.

A report by the global investment bank Torreya looks at the most common types of rare diseases that are a focus for therapeutic companies around the world:

  • Multiple sclerosis emerges above all others, at 90 patients per 100,000 people.
  • Narcolepsy—intermittent, uncontrollable episodes of sleepiness—affects 50 patients per 100,000.
  • Primary biliary cholangitis, the damage of bile ducts in the liver, affects 40 people in 100,000.
  • Rounding out the top five orphan diseases are Fabry disease (30 patients per 100,000), and cystic fibrosis (25 patients per 100,000).

One catch behind these stats? There’s actually no universal definition of what constitutes a rare disease. This means prevalence data like the above is often inconsistent, making it difficult to record the precise rate of natural occurrence.

The Cost of Rare Diseases

This gap in knowledge comes at a price—many rare diseases have constrained options for treatment. Orphan drugs are often commercially underdeveloped, as their limited end-market usage means they aren’t usually profitable enough for traditional research.

In the United States, government-backed incentives such as tax credits for R&D costs and clinical trials are speeding up the pathways from drug to market. Other places like the EU, Japan, and Australia are also following suit.

In total, it’s estimated that pharma companies focused on rare diseases are worth about half a trillion in enterprise value, roughly equal to 17.5% of the value of Big Pharma:

  • Non-oncology value: $315.7B
  • Oncology value: $193.1B
  • Total enterprise value: $508.8B

Source: Torreya Report. Market values are for the top 31 pure play rare disease therapeutic companies.

The average cost of an orphan drug per U.S. patient annually can climb to near $151,000 (a whopping 4.5 times that of a non-orphan drug, at $34,000). That’s why the pharma industry is urgently advancing rare disease therapeutics across different categories.

Dominant Orphan Drug Sales

According to other estimates, orphan drugs are set to capture over one-fifth of global prescription sales by 2024. Blood, central nervous system, and respiratory-related drugs are currently the top therapeutic categories and are expected to keep this status into the future.

The figures below break down global orphan drug sales by therapy category, and their present and estimated future market share. Note that oncology-related orphan drug sales are excluded from this table.

Therapy Category2018 SalesMarket Share2024E SalesMarket ShareChange in Market Share
Total Sales$67.9B$118.5B
Blood$21.3B31.4%$33.1B27.9%-3.4%
Central nervous system (CNS)$11.1B16.3%$20.3B17.1%0.8%
Respiratory$7.8B11.5%$13.6B11.5%0%
Immunomodulators$7B10.3%$12.5B10.5%0.2%
Cardiovascular$6.7B9.9%$8.5B7.2%-2.7%
Endocrine$3.8B5.6%$5.6B4.7%-0.9%
Musculoskeletal$3.5B5.2%$11B9.3%4.1%
Systemic anti-infectives$3.1B4.6%$4.2B3.5%-1%
Gastro-intestinal$2.9B4.3%$6B5.1%0.8%
Genito-urinary$0.6B0.9%$1.5B1.3%0.4%
Sensory organs$0.1B0.1%$1.5B1.3%1.1%
Dermatology$0B0%$0.7B0.6%0.6%

Source: EvaluatePharma. Industry sales are based on the top 500 pharma and biotech companies.

Much is still unknown about rare diseases in the health community. Frequent misdiagnosis, and up to an average of 8 years for an accurate diagnosis, continue to be a problem for patients.

There are two sides to the situation. On one, tech giants like Microsoft are providing digital health solutions to speed up diagnosis, through machine learning and blockchain-based patient registry.

On the other, many skeptics question whether the industry is interested in finding cures for rare diseases at all, especially when they account for a significant portion of industry revenues.

Is curing patients a sustainable business model?

Goldman Sachs

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Healthcare

The Future of Nanotechnology in Medicine

This infographic highlights some of the most promising nanotechnology breakthroughs in medicine, from ‘smart pills’ to targeted cancer treatment.

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nanotechnology medicine

The Future of Nanotechnology in Medicine

Around the world, researchers are increasingly thinking smaller to solve some of the biggest problems in medicine.

Though most biological processes happen at the nano level, it wasn’t until recently that new technological advancements helped in opening up the possibility of nanomedicine to healthcare researchers and professionals.

Today’s infographic, which comes to us from Best Health Degrees, highlights some of the most promising research in nanomedicine.

What is Nanotechnology?

Nanotechnology is the engineering of functional systems at the molecular level. The field combines elements of physics and molecular chemistry with engineering to take advantage of unique properties that occur at nanoscale.

One practical example of this technology is the use of tiny carbon nanotubes to transport drugs to specific cells. Not only do these nanotubes have low toxicity and a stable structure, they’re an ideal container for transporting drugs directly to the desired cells.

Small Systems, Big Applications

While many people will be most familiar with nanotech as the technology powering Iron Man’s suit, real world breakthroughs at the nanoscale will soon be saving lives in healthcare.

Here are a few ways nanotechnology is shaping the future of medical treatment:

1. Smart Pills

While smart pill technology is not a new idea — a “pill cam” was cleared by the FDA in 2001 — researchers are coming up with innovative new applications for the concept.

For example, MIT researchers designed an ingestible sensor pill that can be wirelessly controlled. The pill would be a “closed-loop monitoring and treatment” solution, adjusting the dosage of a particular drug based on data gathered within the body (e.g. gastrointestinal system).

An example of this technology in action is the recent FDA-approved smart pill that records when medication was taken. The product, which is approved for people living with schizophrenia and bipolar disorder, allows patients to track their own medication history through a smartphone, or to authorize physicians and caregivers to access that information online.

2. Beating the Big C

Nearly 40% of humans will be diagnosed with cancer at some point in their lifetime, so any breakthrough in cancer treatment will have a widespread impact on society.

On the key issues with conventional chemotherapy and radiation treatments is that the body’s healthy cells can become collateral damage during the process. For this reason, researchers around the world are working on using nano particles to specifically target cancer cells.

Oncology-related drugs have the highest forecasted worldwide prescription drug sales, and targeting will be a key element in the effectiveness of these powerful new drugs.

3. Diagnostics

Medical implants — such as knee and hip replacements — have improved the lives of millions, but a common problem with these implants is the risk of post-surgery inflammation and infection. In many cases, symptoms from an infection are detected so late that treatment is less effective, or the implant will need to be replaced all together.

Nanoscale sensors embedded directly into the implant or surrounding area could detect infection much sooner. As targeted drug delivery becomes more feasible, it could be possible to administer treatment to an infected area at the first sign of infection.

Examples like this show the true promise of nanotechnology in the field of medicine. Before long, gathering data from within the body and administering treatments in real-time could move from science fiction to the real world.

10,000 years ago, man domesticated plants and animals, now it’s time to domesticate molecules.

– Professor Susan Lindquist

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Visualizing Healthcare Spending by Country

Healthcare spending can be measured as a proportion of GDP, by admin costs, and per capita—and the United States comes in first in every category.

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How Much Do OECD Countries Spend on Healthcare?

When you start feeling ill, the first line of defense is typically to have a doctor assess the symptoms—but how much you end up paying for a visit differs greatly by region.

Today’s interactive visualization was created by HealthDataViz consultant Lindsay Betzendahl, who also founded #ProjectHealthViz. The data considers how healthcare spending by country stacks up across the 36 Organization for Economic Cooperation (OECD) members, and how it has changed since 2010.

One thing is clear—the United States comes in first place in each category, but that’s not necessarily a good thing:

 🇺🇸 United States🌐 OECD Average
Healthcare Spending (% of GDP)16.9% (#1)8.8%
Admin Costs as % of Health Spend8.3% (#1)*~3%
Per Capita Prices (Current PPPs, USD)$10,586 (#1)$3,992

*Although Costa Rica’s figure was higher in 2016, more recent data is not yet available.

Let’s look at each individual cost category, to see what else we can learn.

What Portion of GDP Goes Towards Health?

Population health is a strong determinant in quality of life. As such, how much a country spends on healthcare as a percentage of gross domestic product (GDP) can be an important indicator.

The U.S. spends 16.9% of GDP on its healthcare, nearly double the OECD average of 8.8%. That’s also over 4 percentage points (p.p.) above Switzerland, which ranks second with 12.2% healthcare spending by GDP.

The problem? While Switzerland consistently ranks as having one of the best healthcare systems in the world, the U.S. lags behind—which means that expenditures are not always translating into better health outcomes for patients.

Where’s the Money Going?

Looking after the health of millions of people is a lot of work, and this is where spending on healthcare administration and financing comes into play. Funds are allocated to medical resource providers, who manage everything from health records to salaries and insurance bills.

The U.S. spends about 8.3% of its total healthcare expenditures on these complex costs today, which is a marginal increase from 7.5% in 2010. Interestingly, Costa Rica’s healthcare spending on the same metric was even higher in 2016, at 9.5% of the total.

On the bright side, Mexico has been making strides in the past few years: administrative spending plunged from 10.3% in 2013, down to 4.6% in 2017.

Globally, advancements in health-tech are helping to reduce costs by streamlining tedious processes. However, it’s still not enough—and these immense costs trickle down to patients.

How Much Does Each Person Shell Out?

Over the past eight years, a majority of OECD countries have seen their healthcare spending per capita climb, with Luxembourg and Greece being the only exceptions. The average OECD country’s spend was $3,992 per capita in 2018, up from $3,080 in 2010—nearly a 30% increase.

However, the U.S. experiences the most dramatic sticker shock by far. At $10,586 per head, the U.S. average is already more than double the OECD average. What’s more, this is a 33.3% increase from $7,939 in healthcare spending per capita in 2010.

As the U.S. healthcare reform debate around prices and quality of care rages on, it’s important to remember that healthy people are the backbone of any country’s long-term economic growth.

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