Microtrends, Orphan Drugs & Evidence-Based Medicines

Here’s a political statement for the new year:

Mark Penn, Hilary Clinton’s campaign strategist, has a book in which he identifies the power of Microtrends to start a political movement or launch a movement. Penn says you only need three million people or one percent of the population. His premise is that by detecting small changes in behavior has a large impact on business, politics and political lives. www.microtrending.com

Okay, now what about orphan diseases. Talk about Microtrends. Orphan diseases affect less than 200,000 people in the US or less than 5 people in a community of 10,000.

Some of the diseases include various types of cancer, TB, cystic fibrosis, Karposi’s sarcoma, Huntington disease, smallpox, idiopathic pulmonary fibrosis, lupus.

Not a bad list, not a good list. Depends on how you look at it from a public health need, disaster, commercial opportunity or roll the dice for yourself, your loved one or your pharmaceutical company.

Sure soccer moms, according to Penn, can be a potent voting block. Others include extreme commuters (90+ minutes to work), Protestant Latinos, Caffeine Crazies.

Now think about some of the people suffering from these orphan diseases.

The granting of the orphan drug status by the FDA and the EU is to encourage the development of drugs to address these diseases, but, normally, these drug developments would be prohibitively expensive/un-profitable to develop under normal circumstances. www.fda.gov/orphan/oda.htm

The idea behind the US Orphan Drug Act is to encourage companies to invest money in research. Under the act many drugs have been developed, including drugs to treat glioma, multiple myeloma, cystic fibrosis, phenylketonuria and snake venom. In the US, from January 1983 to June 2004, a total of 1,129 different orphan drug designations have been granted by the Office of Orphan Products Development (OOPD) and 249 orphan drugs have received marketing authorization in the US. In contrast, the decade prior to 1983 saw fewer than ten such products come to market.

Leading orphan drugs include Amgen’s Erythropoietin and Novartis’ Gleevec. And these products aren’t cheap.

According to a 2003 article in the British Journal of Cancer, Gleevec® (imatinib mesilate), a tyrosine kinase inhibitior, emerged as the most effective non-transplant treatment available for patients with chronic myeloid leukemia (CML).

Yet researchers in the United Kingdom reported that the costs per quality adjusted life year (QALY) is approximately $40,000 more than conventional therapy for patients treated in accelerated phase and almost $60,000 more for patients treated in blast crisis.

Gleevec® has been evaluated as salvage therapy for patients in the accelerated or blast phase of CML., and studies determined that initial treatment of CML patients with Gleevec® is superior to treatment with alfa interferon plus chemotherapy.

Yet paying for Gleevec is expensive. Researchers in England compared the economic impact of Gleevec® compared to standard chemotherapy for the treatment of patients in accelerated or blast phase of CML. They used a computer model which took into account 5 years of treatment from the start of treatment. They estimated that a patient in accelerated phase would accrue an additional 2.09 QALYs with Gleevec® compared to conventional therapies, while patients in blast crisis will accrue an additional 0.58 QALY.

They also report that this improvement comes with a price, approximately $40,000 per additional QALY more than conventional treatment for patients in accelerated phase. For patients in blast phase, the cost was almost $60,000 more per additional QALY. These projected costs were highly dependent on the price of Gleevec®, improvements in quality of life, and duration of haematological response.

Now if you REALLY want to think about microtrends. Think about the impact of the really beneficial use of this drug. And then think about its impact on patients, their families, their doctors, their governments, their payors.

What we talk about when we talk about evidence-based medicines is really this.

And no political candidate, let alone pharmaceutical company or payor or legislator is fully undertaking the challenge of addressing these issues. The government recognizes the need. The law is there. But the research and the issues are still being addressed on an ad hoc basis.

The evidence is there and it is continuing to emerge and evolve. The basis for a sound policy and a sound strategy still needs to be developed.