Looking Back; Thinking Forward
Cancer Drug Development and the Orphan Drug Act by
Michael Scott, Prinicipal and Executive VP,voxmedica
Friday of last week was the 25th anniversary of the signature of the Orphan Drug Act (ODA) by Ronald Reagan -- on January 4, 1983.
Since the signature of this Act, over 300 drugs have been approved in the US with orphan drug designations, and about 60 of those have been cancer therapeutics. If you add the other drugs approved with orphan designations that are used in supportive cancer care, the number is nearer to 80. The first cancer therapy approved with an orphan designation appears to have been methotrexate for osteogenic sarcoma in April 1988; the most recent, a second orphan indication for Nexavar (sorafenib), for hepatocellular carcinoma, in November 2007. Gleevec (imatinib) has now accumulated seven orphan indications in the US alone.
Arguably, the passage of the ODA has been one of the most successful pieces of legislature approved by Congress in the past 30 years. In the decade before its approval, only 10 new drugs developed by the pharmaceutical industry had been approved for orphan diseases. Passage of the ODA has led to an explosion in the number of drugs available for rare forms of cancer, has facilitated the continuing transformation of the global biotechnology industry (with a particularly successful emphasis on the area of oncology), and has led to similar orphan drug legislation in many other countries around the world.
Late last year, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMEA) developed a single application process for orphan drug designation that would be valid for both agencies (see the joint EMEA and FDA press release dated November 26, 2007). This will simplify the process of gaining orphan designation across a dozen of the world’s major pharmaceutical markets. In addition, as we gain greater insight into the precise subtypes of various cancers that are responsive to particular agents, almost every form of cancer is potentially an orphan disease, with major implications for the regulatory development pathways for cancer therapeutics over the next decade.
From the payer perspective, one of the downsides of this success has been the sudden increase in prices for highly effective orphan cancer drugs with relatively small markets. In the past couple of years, the introductions of Erbitux (cetuximab), Revlimid (lenalidomide), Sprycel (dasatinib) and other orphan cancer therapeutics have been associated with high treatment costs for patients and payers. The degree to which this trend is sustainable is one that will tax minds of biopharmaceutical companies and payers for years to come. On the other hand, as effective treatments become more widely available for highly defined patient populations, is it ethical for societies to deny treatment on the basis of cost alone?
How do you think the continuing evolution of the use of the ODA will affect cancer therapy? Can we afford all these niche therapeutics? Does your company really appreciate how to best take advantage of orphan drug designation and relevant regulatory issues? Contribute to the OBR blog on this topic.
And while we are at it, congratulations to the National Organization for Rare Diseases (NORD), whose retiring president, Abbey Meyers, is widely recognized as the primary consumer advocate behind the original approval of the ODA. NORD is also 25 years old this year, and has planned a series of special events to celebrate the joint anniversary.
Michael Scott, Prinicipal and Executive VP,voxmedica
Friday of last week was the 25th anniversary of the signature of the Orphan Drug Act (ODA) by Ronald Reagan -- on January 4, 1983.
Since the signature of this Act, over 300 drugs have been approved in the US with orphan drug designations, and about 60 of those have been cancer therapeutics. If you add the other drugs approved with orphan designations that are used in supportive cancer care, the number is nearer to 80. The first cancer therapy approved with an orphan designation appears to have been methotrexate for osteogenic sarcoma in April 1988; the most recent, a second orphan indication for Nexavar (sorafenib), for hepatocellular carcinoma, in November 2007. Gleevec (imatinib) has now accumulated seven orphan indications in the US alone.
Arguably, the passage of the ODA has been one of the most successful pieces of legislature approved by Congress in the past 30 years. In the decade before its approval, only 10 new drugs developed by the pharmaceutical industry had been approved for orphan diseases. Passage of the ODA has led to an explosion in the number of drugs available for rare forms of cancer, has facilitated the continuing transformation of the global biotechnology industry (with a particularly successful emphasis on the area of oncology), and has led to similar orphan drug legislation in many other countries around the world.
Late last year, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMEA) developed a single application process for orphan drug designation that would be valid for both agencies (see the joint EMEA and FDA press release dated November 26, 2007). This will simplify the process of gaining orphan designation across a dozen of the world’s major pharmaceutical markets. In addition, as we gain greater insight into the precise subtypes of various cancers that are responsive to particular agents, almost every form of cancer is potentially an orphan disease, with major implications for the regulatory development pathways for cancer therapeutics over the next decade.
From the payer perspective, one of the downsides of this success has been the sudden increase in prices for highly effective orphan cancer drugs with relatively small markets. In the past couple of years, the introductions of Erbitux (cetuximab), Revlimid (lenalidomide), Sprycel (dasatinib) and other orphan cancer therapeutics have been associated with high treatment costs for patients and payers. The degree to which this trend is sustainable is one that will tax minds of biopharmaceutical companies and payers for years to come. On the other hand, as effective treatments become more widely available for highly defined patient populations, is it ethical for societies to deny treatment on the basis of cost alone?
How do you think the continuing evolution of the use of the ODA will affect cancer therapy? Can we afford all these niche therapeutics? Does your company really appreciate how to best take advantage of orphan drug designation and relevant regulatory issues? Contribute to the OBR blog on this topic.
And while we are at it, congratulations to the National Organization for Rare Diseases (NORD), whose retiring president, Abbey Meyers, is widely recognized as the primary consumer advocate behind the original approval of the ODA. NORD is also 25 years old this year, and has planned a series of special events to celebrate the joint anniversary.
posted by Don Sharpe - www.oncbiz.com at
3:16 PM
1 Comments:
According to a recent press release, BIO President and CEO Jim Greenwood issued the following statement earlier today:
“Today we celebrate the 25th anniversary of the Orphan Drug Act. The law has led to a vast increase in the number of therapies available to the more than 25 million Americans living with one of the 7,000 recognized rare diseases and significantly contributed to the growth of the biotechnology industry.
“The biotech industry has historically taken financial risks to develop therapies and cures to serve small patient populations suffering from rare, devastating diseases. The Orphan Drug Act established some necessary protection to attract the investment required to research and develop these breakthrough technologies.”
“BIO also recognizes the anniversary of the establishment of the National Organization for Rare Disorders (NORD) which serves patients with rare diseases.”
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