Orphan drug launches reach 10-year high in 2011

Orphan drug launches for rare diseases reached a 10-year high in 2011, underscored by new breakthroughs in personalized medicine, according to a report by IMS Institute for Healthcare Informatics in New Jersey.

Orphan drugs carry a higher price tag than other drugs because they are intended for a patient population of no more than 200,000.

Among the 12 orphan drugs launched last year was Pfizer’s (NYSE:PFE) drug critzotinib, branded Xalkori, to treat nonsmall cell lung cancer. The drug targets a specific biomarker — the anaplastic lymphoma kinase fusion gene. The therapy is for 3 percent to 7 percent of patients with tumors that carry a unique mutation and can improve survival by nine months. Roche-owned Genentech drug, sumotropin, a human growth hormone, was launched to alleviate the short stature associated with Turner syndrome, a disorder that affects women in which they lack or have a malfunctioning sex chromosome.

The surge on orphan drug launches reflects a decade of research pharmaceutical companies have been doing in this area that “is beginning to bear fruit,” a person familiar with the report said. The reason behind pharmaceutical companies focusing on this area is it gives them some advantages over developing drugs for a large patient population. These drugs are developed for niche patient communities, but because it is usually for an unmet need, it can mean a faster track to market and companies can enjoy longer exclusivity, which means these drugs can generate more revenue for them. On the other hand, they still have to move through clinical trials and could face delays if the companies receive a complete response letter from the U.S. Food and Drug Administration that requires more data.


Five therapy areas accounted for one-third of the $320 billion spent on pharmaceuticals last year — cancer, asthma chronic obstructive pulmonary disease, dyslipidemia, diabetes and mental health — particularly psychosis or bipolar disorders. Spending on branded medication rose 2.2 percent and reflected the impact of expired patents that shaved about $15 billion off the total spending. The figure represents a marginal increase of 0.5 percent over 2010, which reflects a more cautious patient population with senior citizens rationing their healthcare spending.


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