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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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Demographics

The Global Inequality Gap, and How It’s Changed Over 200 Years

This visualization shows the global inequality gap — a difference in the standards of living around the world, as well as how it’s changed over 200 years.

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How the Global Inequality Gap Has Changed In 200 Years

What makes a person healthy, wealthy, and wise? The UN’s Human Development Index (HDI) measures this by one’s life expectancy, average income, and years of education.

However, the value of each metric varies greatly depending on where you live. Today’s data visualization from Max Roser at Our World in Data summarizes five basic dimensions of development across countries—and how our average standards of living have evolved since 1800.

Health: Mortality Rates and Life Expectancy

Child mortality rates and life expectancy at birth are telltale signs of a country’s overall standard of living, as they indicate a population’s ability to access healthcare services.

Iceland stood at the top of these ranks in 2017, with only a 0.21% mortality rate for children under five years old. On the other end of the spectrum, Somalia had the highest child mortality rate of 12.7%—over three times the current global average.

While there’s a stark contrast between the best and worst performing countries, it’s clear that even Somalia has made significant strides since 1800. At that time, the global average child mortality rate was a whopping 43%.

Lower child mortality is also tied to higher life expectancy. In 1800, the average life expectancy was that of today’s millennial—only 29 years old:

Life Expectancy in 1800 by Continent

Today, the global average has shot up to 72.2 years, with areas like Japan exceeding this benchmark by more than a decade.

Education: Mean and Expected Years of Schooling

Education levels are measured in two distinct ways:

  • Mean years: the average number of years a person aged 25+ receives in their lifetime
  • Expected years: the total years a 2-year old child is likely to spend in school

In the 1800s, the mean and expected years of education were both less than a year—only 78 days to be precise. Low attendance rates occurred because children were expected to work during harvests, or contracted long-term illnesses that kept them at home.

Since then, education levels have drastically improved:

 Mean Years of SchoolingExpected Years of schooling 
Global Average8.4 years12.7 years
HighestGermany 🇩🇪: 14.1 yearsAustralia 🇦🇺: 22.9 years
LowestBurkina Faso 🇧🇫: 1.5 yearsSouth Sudan 🇸🇸: 4.9 years

Research shows that investing in education can greatly narrow the inequality gap. Just one additional year of school can:

  • Raise a person’s income by up to 10%
  • Raise average annual GDP growth by 0.37%
  • Reduce the probability of motherhood by 7.3%
  • Reduce the likelihood of child marriage by >5 percentage points
  • Source

    Education has a strong correlation with individual wealth, which cascades into national wealth. Not surprisingly, average income has ballooned significantly in two centuries as well.

    Wealth: Average GDP Per Capita

    Global inequality levels are the most stark when it comes to GDP per capita. While the U.S. stands at $54,225 per person in 2017, resource-rich Qatar brings in more than double this amount—an immense $116,936 per person.

    The global average GDP per capita is $15,469, but inequality heavily skews the bottom end of these values. In the Central African Republic, GDP per capita is only $661 today—similar to the average income two hundred years ago.

    A Virtuous Cycle

    These measures of development clearly feed into one another. Rising life expectancies are an indication of a society’s growing access to healthcare options. Compounded with more years of education, especially for women, this has had a ripple effect on declining fertility rates, contributing to higher per capita incomes.

    People largely agree on what goes into human well-being: life, health, sustenance, prosperity, peace, freedom, safety, knowledge, leisure, happiness… If they have improved over time, that, I submit, is progress.

    Steven Pinker

    As technology accelerates the pace of change across these indicators, will the global inequality gap narrow more, or expand even wider?

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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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