• Media
  • All
  • Million-Dollar Drugs: Encouraging News for Patients with Rare Diseases, But at What Cost?


  • Home
  • All
  • Million-Dollar Drugs: Encouraging News for Patients with Rare Diseases, But at What Cost?

Million-Dollar Drugs: Encouraging News for Patients with Rare Diseases, But at What Cost?

In recent years, there has been some good news for many of the 30 million people in the U.S. diagnosed with a rare disease, in the form of long-awaited treatment for a number of specific conditions — but this progress has come at a staggering cost for these prescription medications. Fortunately, a new company focusing on life sciences — AEVOLVE AG — has a plan to support medical research that will benefit millions of others with rare diseases, without the alarming costs that are currently associated with drugs of this kind.

The high cost of prescription drugs to treat common medical conditions such as high cholesterol and diabetes have angered many Americans and inspired them to find less expensive options for the treatment they need — some inappropriately rationing their own medication, and millions of others turning to less expensive generic drugs. According to the U.S. Food and Drug Administration, nine out of ten prescriptions filled in this nation are currently for generic drugs. Those who are outraged by the high cost of commonly prescribed medications like these would surely be rendered speechless if they learned of the nearly incomprehensible prices charged to many patients diagnosed with a rare disease. Some of these patients, and their insurance companies, have been forced to pay more than $1 million annually for a drug that will slow the progress of their rare disease — and there is literally no other alternative if they wish to remain alive.

The fact that more and more patients are turning to less expensive generic drugs has posed a challenge to many of the world’s leading pharmaceutical companies and their shareholders. With this motivation, according to a recent article in The New York Times by medical reporters Katie Thomas and Reed Abelson, “pharmaceutical companies are turning to rare-disease treatments and gene therapies as their next profit engine, with major companies like Pfizer and Novartis investing in drugs for tiny pools of patients.”

While, by definition, the number of individuals diagnosed with any specific form of “rare disease” is small, collectively the number of patients in this category is quite large. More than 300 million people worldwide have one of approximately 7,000 diseases classified as “rare,” according to the U.S. medical research organization Global Genes and approximately 30 million people in the U.S. have been diagnosed with a rare disease, according to a coalition of America’s biopharmaceutical companies. For many of these patients, of course, the news that a drug than slow the progression of their disease is literally a matter of life-and-death.

The pharmaceutical industry’s newfound interest in developing drugs for the treatment of rare diseases can be seen in the number of new medications brought to market in recent years. According to The New York Times, “Of 59 new drugs approved in 2018, more than half [34] were for rare diseases. Those treatments are typically the most expensive, helping to drive an increase in overall spending on prescriptions nationwide.”

Those whose lives have been extended by these new medications can thank, in large part, the Orphan Drug Act, passed by the U.S. Congress in 1984. This law arranged for financial subsidies to support clinical trials, and gave the pharmaceutical companies tax incentives and protection against competition that many see as an unfair “monopoly.” In addition, breakthroughs in genetic research techniques and the fact that, under the Affordable Care Act, employers and their insurance companies are now legally required to cover any treatment an individual may need has created “a perfect storm” for skyrocketing prices for drugs to treat numerous rare diseases.

One example of these “million-dollar-drugs” now available for one specific rare disease is Strensiq, produced by the Boston based pharmaceutical company Alexion. The New York Times describes Strensiq, a medication for the treatment of a rare bone disease known as “hypophosphatasia,” as “one of the costliest drugs in the world.” This rare disease is estimated to affect only 1,300 people in the U.S., and like most rare diseases, this condition is genetic in origin. Babies born with hypophospatasia often have bones that are so fragile, due to mineral deficiencies, that they are almost imperceptible in x-rays. Before Strensiq was available, many infants born with this disease rarely lived to see their first birthday.

Adults have been diagnosed with hypophosphatasia, too — and a recent article in The New York Times profiled one Ohio family affected by this rare disease. The total cost of this family’s Strensiq prescription — for two young adults and their mother, all diagnosed with the disease — reached a staggering $6 million in 2018. In this case, the cost of this expensive medication was covered by the family’s insurance plan, coverage obtained through the head of household’s union. Consultants interviewed for the news article estimated that the total “lifetime cost” to provide all three members of this family with Strensiq could reach an astronomical $60 million.

Information about the cost of rare-disease drugs charged by large pharmaceutical companies is often hidden — as some of them do not emphasize the fact that the dosage for these medications is often based on the patient’s weight. This means that a prescription for an adult can be several times the cost estimated for the treatment of an infant.

New treatments for conditions such as hemophilia in children and for lung cancer are also extraordinarily expensive. One new drug for the treatment of Pompe disease — an inherited enzyme disorder that can result in early death in infants, costs approximately $600,000, double the cost estimated by its manufacturer when it was approved in 2010. Alexion, the maker of Strensiq, makes another drug — Soliris, for the treatment of a rare immune and blood disorder — that brought in $3.5 billion for the company last year, and is said to be another one of the world’s most expensive medicines.

The pharmaceutical companies justify these prices by citing the cost it takes to bring them to market. In an official statement, Alexion noted that “developing medicines for rare diseases is complex, costly and carries a significant risk of failure.” In addition, the company said, “there continues to be significant ongoing investment required to bring Strensiq to patients, including in diagnostic and educational efforts and manufacturing.” Some pharmaceutical companies — including Sarepta and Sanofi — say they make it a point to provide patients who are uninsured or can’t afford the out-of-pocket cost with the life-saving medication they need.

At the same time, there can be no doubt that the pharmaceutical companies producing drugs to treat rare diseases see this as a potentially profitable business opportunity. In a report prepared for its shareholders, the company said, “Strensiq is expected to play a major role in enabling Alexion Pharmaceuticals to create shareholder value while competing with other rare disease players.”

Fortunately, there is good news on the horizon for individuals seeking treatment for a rare disease but terrified by the prices demanded by other medications in this field. A bold visionary company — AEVOLVE AG — is about to step into the arena, eager to support medical research project focusing on rare diseases in an entirely new way. Patients themselves will have an opportunity to support medical research projects focusing on the rare disease that affects them personally, and through the use of a blockchain investment platform, this approach will offer transparency not otherwise see in this important field of healthcare.


Aevolve is a protocol that supports medical innovations from R&D stages to full-scale commercialization, by connecting patients, investors and scientific innovators. It offers BioSpheres, an innovation marketplace that connects patients and the global community directly with scientific innovators and experts from all over the world to address various stages of the innovation life cycle.

For more information, visit

Posted in:

All,  Blockchain,  Health,  Innovations

Add comment

Your email address will not be published. Required fields are marked *

You might also like