How Does Drug Pricing Drive Therapeutic Choice?

An expert panel convened in Atlanta, Georgia, December 9th during the 49th annual meeting of the American Society of Hematology. The Biogen Idec-sponsored symposia, titled, “How does drug pricing drive therapeutic choice?” featured presentations from Frank Lichtenberg, PhD, Professor of Business at the Columbia University Graduate School of Business, New York, NY; Sir Michael Rawlins, MD, Chairman of the National Institute of Health and Clinical Excellence, London, United Kingdom; and Peter Bach, MD, Memorial Sloan-Kettering Cancer Center, New York, NY. Linda Bosserman, MD, FACP, President, Wilshire Oncology, Los Angeles, Calif. moderated the panel discussion on the issue of drug pricing.

Pharmaceutical Innovation and Cancer Survival
According to Dr. Lichtenberg, cancer survival rates have increased substantially in the last 50 years. He hypothesized that the development and use of new cancer drugs has made an important contribution to the increase in cancer survival. He tested this hypothesis by examining the relationship between drug vintage (approval year) and cancer survival in four methods of analysis, using four different sets of data.

His analysis revealed that the cancer sites whose drug vintage (measured by the share of post-1990 treatments) increased the most during the 1990s (indicating use of newer drugs) tended to have larger increases in observed survival rates, controlling for other factors…that drug vintage (the share of post-1985 treatments) had a positive and statistically significant effect on both 1-year and 5-year survival rates...that, typically, countries that adopted new cancer agents more rapidly experienced larger declines in their age-adjusted cancer mortality rate…that state reimbursement policies may play a part in cancer survival rates.

The NICE Experience: A total cost of care approach
Sir Michael Rawlins acknowledged Dr. Lichtenberg’s research that 5-year survival rates appeared lower in the UK than in other European countries; however, he emphasized that the UK has recently doubled its investment in healthcare—and these results have intrigued US payers.

Faced with half of the US’s per capita healthcare investment, the UK has chosen to establish a rationing system for allocating healthcare resources. To set limits, NICE weighs a drug’s cost-effectiveness in its coverage decisions and in some cases has said no to paying for certain treatments.

“Sometimes we need to show selective use of a drug, because we’ve felt it’s not cost effective for certain uses,” said Sir Michael.

Of the five oncology treatment-condition pairs NICE has rejected for use since 1999, three were cost-ineffective. “In Britain, the truth is the population doesn’t like the idea of cost coming into the decision, [but] it’s clear we have to do it,” concluded Sir Michael.

How to Pay for Cancer Drugs
Peter Bach, MD, acknowledged that drug prices can be perceived as too high. He offered a mix of regulatory and policy solutions to control prices, while maintaining choice, including:

• Create a forum similar to NICE’s approach to value-based payment.
• Give patients more of an incentive to spend healthcare dollars efficiently.
• Regulators could set prices and pay a fixed amount per healthcare gain.
• Change the sole-source definition for Medicare’s average sales price purposes.

Note: A full length version of this article was published in the January issue of OBR.

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View a webcast of this symposium sponsored by Biogen Idec

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The Appeal of Genasense: The drug, the data, and the tangled web that its FDA review has become

At some point—probably in the very near future—there will be a final decision by the U.S. Food and Drug Administration (FDA) about whether Genta’s antisense agent Genasense® (oblimersen sodium) will be approved for the treatment of relapsed/refractory chronic lymphocytic leukemia (CLL). In the meantime, the review process has become an increasingly tangled web of data, assertions, and upset. Many healthcare professionals feel that the drug warrants approval and that the FDA’s treatment of Genasense has been too harsh.

Among the concerns raised by the ODAC members was their preference for progression-free survival (PFS) as an endpoint. The committee also asserted that the patients likely to benefit had not been clearly identified, and that the potential number of patients who would benefit did not warrant the approval of an expensive drug.

According to Genta, and many CLL experts, the FDA’s rejection of Genasense at that time was, simply, wrong. Patients have voiced their concerns, too.

According to the most recent findings (ASH abstract 751), which have been submitted to the FDA as part of the appeal, patients in the triple combination arm achieved a complete response (CR) rate of 17 percent, compared with a CR rate of 7 percent among patients treated with chemotherapy alone, a statistically significant difference.

The new data may be enough to warrant approval by the FDA. But the larger question of whether the agent should have been approved in the first place still looms, as do issues other than data that may have influenced the original decision.

Regardless of the outcome, the saga of Genasense raises crucial questions about the FDA review process. These issues extend well beyond this one drug: as is widely known, the FDA review of Dendreon’s immunotherapy Provenge® is now under serious investigation.

In its genuine efforts to ensure drug safety, is the FDA preventing drugs from reaching the patients who could be helped? Are there political reasons behind the FDA’s decisions to approve or reject drugs, and if so, what are they? Are drug applications such as these being used to set a precedent, rather than being evaluated for their own merit? Is the FDA weighing cost too heavily in its considerations of benefit?

Numerous CLL patients have already been helped by Genasense, and there is a clear desire among experts and patients alike that this drug be made available. At some point, the outcome of the Genasense application will be made clear. But its hard-to-treat side effects—the questions and issues raised during the review process—remain.

Note: A full length version of this article was published in the January issue of OBR.

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Biogen Idec: An Oasis for Employees During the Southern California Wildfires

With wildfires threatening Southern California families and their homes, Biogen Idec opened its campus facilities to ease the burden of their employees who were in the fires’ path of destruction. By Candice J. Bruce, PhD

The wildfires that ravaged southern California in late October 2007 halted business-as-usual at many of the over 500 biotech companies in San Diego County. The fires moved in on Sunday, October 21 and by Monday morning there were eight fires fueled by strong Santa Ana winds that derailed firefighters’ efforts to stop them. Biotech centers in Carmel Valley, Sorrento Valley, and Carlsbad as well as their adjacent neighborhoods, which house many of the 36,000 people employed in the San Diego biotech industry, were in jeopardy.

Several biotech companies and academic institutions were closed due to the looming threat of the approaching wildfires. Recognizing the seriousness of the situation and that many of their employees would be affected by the fires, Biogen Idec’s San Diego facility opened up its campus to displaced employees without hesitation. Over 30 percent of Biogen Idec’s employees had been evacuated and many of them along with their families and pets spent the night at the campus hoping their home would be spared. To try and make everyone as comfortable as possible, “the catering staff stayed on and prepared meals; blankets and pillows were provided as well as books and DVDs for the kids,” said Naomi Aoki, director of corporate communications for Biogen Idec.

Jorg Thommes, director of purification process development at Biogen Idec, evacuated his home during the wildfires; he and his family spent the night at the campus. His children were frightened by the fires, but “when we arrived at Biogen Idec they saw some of their friends and became happier so my wife and I became more comfortable. We were deeply impressed with the company’s commitment and concern for us. A few days after we returned home, my daughter asked when the next sleepover at daddy’s work would be.”

Over 300,000 people in San Diego County had been forced to flee their homes. Biogen Idec kept its campus open for the displaced employees and was able to secure hotel rooms for many of them. Concern for the welfare of the evacuees led to a group being formed to make sure that employees were aware of resources available to help them, and many employees not affected by the fires opened up their homes to those that needed a place to stay. Annie Glidden, head of the relief effort at Biogen Idec, stated, “I was amazed by the unquestioning commitment for employee safety and well-being coming from everyone at Biogen Idec in San Diego as well as our other locations. Everyone came together on behalf of the people affected by the fires, and it was something that I had not experienced before at a company.” Biogen Idec was closed for business the entire week of October 22 and employees were paid in full.

Overall the wildfires burned more than 300 square miles and destroyed over 1000 homes in San Diego. The economic impact is estimated to be over $1 billion in San Diego County alone. Numerous biotech companies including Genentech, Invitrogen, Althea Technologies, and Biogen Idec as well as others have donated hundreds of thousands of dollars to various fire relief funds. Biogen Idec has donated $50,000 to Habitat for Humanity as well as other funds and is organizing a team to work with the organization to rebuild a home.

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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.