Supplement: The Trials of Keeping Track

1 on the epidemiology of autoimmune diseases, perhaps the most detailed publication on the topic so far. That report shows that in the more common conditions, such as MS, the imbalance tilts so that affected patients are roughly two-thirds female. "In some diseases, however, the degree of disproportion is very extreme," Cooper says. For example, at least 85% of patients with thyroiditis, systemic sclerosis, systemic lupus erythematosus, and Sjögren disease are female. In data from the Centers for Disease Control and Prevention for 1995, well-defined autoimmune diseases were one of the top-10 causes of death for women under the age of 65 years.2 The struggle to track these diseases matches the pharmaceutical industry's failure to treat them. For example, drugs for rheumatoid arthritis evolved from injectable gold in the 1930s to biologics today, but treatment results remain mediocre. According to Anthony Manning, vice president and global head of inflammation, autoimmunity, and transplant research at Roche, only about one-third of patients with rheumatoid-arthritis get "significant benefit," even with today's best therapies. Such lacklustre results probably arise from rheumatoid arthritis' heterogeneity, varying in mechanism between individuals and within the same individual over time. Many other autoimmune diseases include similar variability, which thwarts effective treatment. Still, some members of the pharmaceutical community feel optimistic about improving results in the very near future. "Five years ago, we didn't have the plethora of tools that we have today," says Andrew C. Chan, vice president of research immunology and antibody engineering at Genentech. "If you only have one or two tools, it's hard to go and attack any disease and say, 'I have the answer.' It's like having a hammer, and you're walking around looking for the nail." Economic Costs Source: National Institutes of Health. Progress in Autoimmune Diseases Research, 2005. (3 "The individual and his or her family must cope with the feeling of loss of contribution to society combined with redefined social roles, and the effects of pain, fatigue, low self-esteem, mental distress, and depression," Kvien writes. He specifically describes the burden of rheumatoid arthritis, but the same thing could be said of many autoimmune diseases. Yvonne Norton puts it this way: "It's a complete lifestyle change for those who have got it badly. We just know it's going to be there forever." Most autoimmune diseases affect women more than men. In most of the disease shown here, women get them four or more times more often than men. Only two of the diseases show roughly equal gender percentages, and just one - ankylosing spondylitis - occurs more often in men. Source: Randall Stevens of Roche, presentation in a 6 November 2006 Teleconference The breadth of autoimmune diseases resembles the variation in prevalence. As shown here, some autoimmune diseases such as psoriasis and rheumatoid arthritis, occur much more often than others, such as scleroderma and Wegener's granulomatosis. Source: Randall Stevens of Roche, presentation in a 6 November 2006 Teleconference Patient Groups and Lobbying One of the few things that can be said with certainty about the burden of autoimmune diseases: Policymakers have not yet realized the size of the issue, says Rose. "The US and most developed countries have overlooked the significance of autoimmune diseases," he says. "Partly, this is because historically they have been looked at one at a time, which means they look quite small. It's only when you begin to look at them inclusively that you appreciate the scale of the problem." Patient groups play a crucial role in raising consciousness, pushing for more funding, and lobbying for provision of new treatments. "I think that really is an extremely important part of the whole equation," says Rose, who is involved with the American Autoimmune Related Disease Association (AARDA), which played a key role in convincing the NIH to establish a coordinating committee on autoimmune disease research. Ladd, the association's president, is proud of the role that AARDA played in getting congress to ask NIH for a report on the autoimmune disease research it was funding. "It was very obvious at that time [in the late 1990s] that autoimmune diseases were underfunded, and that 90% of the research was on just one or two of the 80 or so diseases." -Virginia Ladd "It was very obvious at that time [in the late 1990s] that autoimmune diseases were underfunded, and that 90% of the research was on just one or two of the 80 or so diseases," Ladd says. In 1998, NIH established the ADCC, and, says Ladd, "with the formation of the committee about $30 million was made available that served as a kind of springboard for a lot of work." The money helped establish, for example, a blood registry for families with clustering of autoimmune diseases. Across the Atlantic, patient groups generated their own successes. Since 2000, for example, Norton has served on a parliamentary working group with Lupus UK, a group that includes Janet Dean, a member of parliament. In recent years, the group managed to initiate discussions in Westminster and has been in direct communication with health ministers. "We've drawn in a lot of members of Parliament," Norton says. "I think they're beginning to understand about it." Beyond raising awareness, disease-specific groups help patients obtain their medications. The MS Society, for example, fought a sustained battle to get the UK's health-spending watchdog, the National Institute for Clinical Excellence (NICE), to fund new MS drugs through the National Health Service. For a while, it looked as if beta-interferon would not be available under the state-health system, but in 2002, a deal was brokered in which costs were shared between the government and drug firms. "When you're ill, you don't want to have to bang the table at your GP's surgery just to get him to refer you to the right specialist." -Yvonne Norton Other battles lie ahead. For example, NICE is considering whether Tysabri (natalizumab), a new MS drug from Biogen Idec, should be available on the National Health Service. This drug is administered by infusion and will involve significant costs to the healthcare system. The MS Society, among others, is pushing hard for a positive decision. Meanwhile, the struggle to raise awareness among physicians continues. "So many patients come to me and say, 'I've been to my GP time after time with my various symptoms, and he doesn't know what's wrong with me, or who to refer me to,'" says Norton. "It's hard, because when you're ill, you don't want to have to bang the table at your GP's surgery just to get him to refer you to the right specialist." The situation is improving, partly thanks to the work of Lupus UK. This organization hangs posters in hospitals and produced a book for general practitioners, which genuinely seems to be raising awareness, Norton says. It isn't all heartbreak, though, because lobbying benefits patients. The US National MS Society's Web site, for example, carries this comment from Scott Hansen, who has lived with MS for eight years: "Engaging in advocacy gives me optimism. My doctor believes that I'm actually getting healthier with age, and I think that's because I'm involved in fighting for myself and others." Despite the significant victories that autoimmune-patient groups have achieved, Ladd says more could be achieved with a more united front. "Disease-specific groups on their own do good advocacy work, but we need collective numbers to really get the job done," she says. The trouble is that the general public, and some of those who form policy, still think of these diseases singularly, she says. Only five percent of Americans can name an autoimmune disease, Ladd says, and some 29% think AIDS is one. "Just having that awareness among the public would be great," Ladd says. Meanwhile, Norton remains positive, despite everything that lupus has cost her. "I do think we're getting there," she says. "I think awareness is growing. To me, you can't cope with something unless you get out and face up to it." References 1. G.S. Cooper et al., "The epidemiology of autoimmune diseases," Autoimmun Rev, 2:119-25, 2003. 2. S.J. Walsh et al., "Autoimmune diseases: a leading cause of death among young and middle-aged women in the United States," Am J Public Health, 90:1463-6, 2000. 3. T.K. Kvien, "Epidemiology and burden of illness of rheumatoid arthritis," Pharmacoeconomics, 22 (Suppl1):1-12, 2004.

Supplement: The Trials of Keeping Track

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