Rare Disease Treatments Becoming Main Focus Point For Biotech Companies


    The pharmaceutical industry spends billions of dollars into research and development treatments for uncommon diseases. A rare disease is defined as one that hits less than 200,000 Americans and in some cases as few as a dozen. In the United States it’s estimated that 1 out of 10 Americans are diagnosed with a rare disease.

    In past years, drugmakers were hesitant to invest in rare-disease treatments with their focus mainly on common problems such as drugs for heart trouble and cholesterol. This all started to change about 10 years ago when patents on the industry’s most profitable drugs began to expire, and generic medicines started wiping out billions of dollars in annual revenue. After that happened, companies started investing their money and focus towards rare disease medicines, reason being is because these drugs cost less to develop and the competition is minimal. In 2014, the FDA approved a record 17 drugs for rare diseases.

    It’s estimated more than 450 other medicines are in development to treat a variety of diseases such as; rare cancers, sickle-cell disease, the hormonal disorder Cushing’s disease and a bleeding ailment called thrombocytopenic purpura, as well as hemophilia, Hodgkin’s lymphoma and pulmonary fibrosis. Another main difference between common and rare treatments is the how the study is operated. Meaning with common diseases are tested on thousands of patients in big groups, while rare diseases are tested on a much smaller groups that vary from a coupled hundred to as little as a dozen. Analyst Steve Brozak of WBB Securities states, “There’s more money, fewer patients and it’s 10 times easier to defend high prices to payers.”

    Biotech companies are making new advances with medicine daily. Big Pharma Companies as big as Pfizer Inc (PFE), and Bristol-Myers (BMY) have profited billions from selling rare-disease treatments. According to a forecast by a research firm GlobalData estimate that by 2019, 34 rare-disease drugs have the potential to exceed annual sales of $1 billion dollars for each drug. As medicine continues to advance globally, companies are working diligently to try and develop the next breakthrough drug for rare-diseases.


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