Will the Next Blockbusters be Treatments for Rare Diseases?
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The era of blockbuster drugs was officially declared over several years ago by many pharmaceutical analysts and pundits. Nevertheless, as the old adage goes “it’s difficult to treat old dogs’ new tricks!” After all, the blockbuster drug model has been the major driver of pharmaceutical and biotechnology markets for close to 50 years. Consequently, big pharma and biotech companies haven’t truly abandoned the possibility of finding potential new blockbusters. And, it appears that the blockbuster heir apparent may be drugs to treat rare aka orphan disease indications.
At first blush, this strategy may not make a lot of sense. This is because rare diseases afflict only small numbers of patients (at least in the US and other Western nations). However, what may be considered a rare disease in Europe or the US may actually be less rare in countries with large populations like China and India. Further, while current rare diseases patient populations may be small, the cost of the drugs developed to treat them is extremely high. In some instances, the annual cost per patient can exceed several hundred thousand dollars. If you do the math, it becomes apparent that developing rare disease treatments or so-called niche busters can actually be very big business.
The rare diseases business model has been perfected by Genzyme and many big pharma companies are trying to emulate it. To that end, big pharma’s push into rare diseases continues to gather momentum. So far this year Sanofi-Aventis has made an $18.5bn move for Genzyme, while Pfizer and GlaxoSmithKline have both created rare disease business units.
According to Glaxo’s estimates, 7,000 rare diseases have been identified and collectively this affects 6-8% of the world’s population; in the US and Europe alone rare diseases affect 25 million people. In addition mortality rates are very high, often at a very young age, and less than 10% of these diseases are treated with approved drugs.
To date, over 7,000 rare diseases have been identified. Companies involved in the new rare diseases treatment race have whittled the list down to roughly 200-250 disorders that represent a clear path for clinical, regulatory and commercial success. The criteria used to select these indications include identifying rare disorders with: 1) a relatively high prevalence rate, 20 an early age of onset, 3) a large unmet medical need and 3) a known molecular target. The indications have been broadly classified into four distinct groups: metabolic disorders, autoimmune/inflammation, central nervous system and blood disorders.
While Genzyme identified, developed and commercialized its rare diseases treatments, it is likely that big pharma companies like Pfizer, Glaxo, and Merck will either in-license potential new treatments or acquire companies with platform technologies or drugs in various stages of clinical development. For example, Merck’s acquisition of Glycofi several years ago has allowed the company to enter into the rare diseases and biosimilar markets.
One of the major problems with extant rare diseases treatment is their excessive and oppressive costs. One can only hope that the increased competition in the rare diseases space will help to lower drug prices and make them more affordable for patients who suffer from these devastating and life-threatening disorders.
For more insights in to the orphan drug disease market, check out an article that I wrote for Life Science Leader this month
Until next time...
Good Luck and Good Job Hunting!!!!!
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Yesterday, I posted a piece on "hit' throughput screening (see below). At the time, I learned about 'hit" throughput screening, I mentioned that I had never heard of "hit" throughput screening but I did know about high throughput screening. As it turns out, there was a problem in translation and in fact, there is no such thing as hit throughput screening and it is actually high throughput screening. Mea Culpa! I apologize for the error and in the future I will be assiduous in my fact checking before I post (a lesson that the boneheads in the Obama administration learned the hard way in the recent Sherrod brouhaha).jpg)
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