04 Eylül 2016
İlk 10 Yetim İlaç ve ekonomik önemi.
Top 10 Orphan Drugs and Their
Posted on 26
August 2016 by Luca Dezzani
Rare diseases are known
to bring significant impact on the quality of life of thousands to millions of
people across the globe. Rare diseases are also referred to as orphan diseases
because pharmaceutical companies refuse to adopt them to develop treatments. In
fact, according to the National Center for Advancing Translational Sciences,
there are approximately 7,000 rare diseases affecting around 25 to 30 million
Americans. Unfortunately, there is still a long way to go to find solutions to
end the suffering because treatment exists for only 200 to 300 orphan diseases
and little is known about the cause of most rare diseases.
companies oftentimes look at large disease populations as the main focus of
their investment and show little interest in developing orphan drugs due to a
number of reasons. One of these is that rare diseases only affect a small
population, which equates to very low profitability. Furthermore, recruitment
of patients for research and development (R&D) is also another challenge
because it is difficult to locate patients and may eventually incur additional
R&D costs. Fortunately, the industry has shifted its perception. In fact,
some pharmaceutical companies are willing to take the risk of developing orphan
drugs, which they now consider as potential money makers that can bring big
revenue and a promising return on investment.
this, to address the unmet treatment needs of various rare diseases, laws such
as the Orphan Drugs Act of 1983 in the United States that gives financial incentives,
were promulgated in order to encourage companies to venture on developing
orphan drugs. Moreover, according to Thomson Reuters, other benefits given to
drug companies for the R&D of orphan drugs are tax credits, favorable
reimbursement, R&D grants, fewer hurdles to approval, waived FDA fees,
longer exclusivity, shorter development timelines, lower marketing costs,
greater regulatory success, faster uptake and premium pricing.
Though orphan drugs
cater to a small patient pool, the high price of the drugs offsets the
aforementioned challenge. In fact, in the year 2010, Soliris, which is known to
treat paroxysmal nocturnal hemoglobinuria (PNH) that affects 1 out of 500,000,
was actually considered as the industrys most expensive drug amounting to $409,000
per year of treatment, which generated a total of $541 million revenue for
Alexion Pharmaceuticals. In addition, in the year 2014, the top selling orphan
drug in the USA in the year 2014 was Rituxan by Roche, a chronic lymphocytic
leukemia drug, which yielded to $ 3.646 billion in total sales.
orphan drugs can be one of the biggest breakthroughs in the pharmaceutical
industry. It is projected that by the year 2018, the market will reach $127
billion or approximately 16% of the total prescription sales. Aside from the
profitability associated with the commercialization of orphan drugs, patients
with rare diseases will be given greater chances of surviving and hope as they
face the battle against their illness. The figure below will give us an
overview of the top 20 orphan drugs that will have the projected highest
revenue in 2018.