Mesothelioma and Other Rare Diseases: Forging Alliances to Make a Greater Impact

togetherNational Organization for Rare Diseases (NORD) and the Drug Information Association held their 2nd Annual Conference –  the US Conference on Rare Diseases and Orphan Products: Shaping the Future Now. The Meso Foundation was in attendance representing mesothelioma and the meso community.

Sessions focused on advocacy for inclusion of patients and community members in the research process, patient access to health care, and development of relationships with stakeholders.  Special attention was paid to the unique challenges that rare disease patient advocacy organizations face. Several NORD member patient advocacy organizations presented case studies of their advocacy successes. Many members of NORD face some of the same issues that face the Meso Foundation; including little or no treatment options, few clinical trials, and limited research funding.

One issue discussed at length was addressing the rising cost of health care. Mark B. McClellan, former director of the Centers for Medicaid and Medicare (CMS), presented on access to care and cost containment. He made the point that expanding coverage and lowering prices won’t support needed innovation, but we need financing reforms that achieve better quality and lower costs. We are facing even more pressure in the coming years. He suggested public insurance providers, Medicaid and Medicare, will try to contain costs by squeezing payment rates, looking to medical innovation, and use of technology to lower costs. He encouraged the rare disease community to remain vocal advocates, so that rare diseases are not ignored and drugs for orphan diseases continue to be developed. Last year there were more than 400 drugs in development for rare diseases, more than one third of those were for rare cancers, including mesothelioma. The Orphan Drug Act has made strides to provide pharmaceutical companies to invest in orphan drugs for rare diseases. One suggestion to improve development of orphan drugs in the United States would be to increase drug exclusivity to the same level as Europe from 7 to 10 years. The Meso Foundation has supported this advocacy effort.

Another important issue for the rare disease community, as well as the meso community, was the idea of risk tolerance. Both the National Institutes for Health (NIH) and the Food and Drug Administration (FDA) had representatives at the meeting that heard the community’s concerns about assessment of risk tolerance. The Meso Foundation had the opportunity to express the view of a patient facing a fatal illness who may be willing to undertake a greater risk than those with more treatment options. The NIH and FDA representatives assured the community that they do consider the severity of a condition as well as the treatment options when approving clinical trials and approving drug safety and efficacy.

The pending threat of the sequester on January 2, 2013 was discussed as well. Chris Jennings, former White House Senior Health Advisor, presented his thoughts on the issue.  He compared the sequester to business or family budgets, stating that “no business or family would make across the board cuts to their budgets – they would preserve funding for the important things and make targeted cuts.” He also touched on an important theme of this election year, which is the need to see a bipartisan solution to balancing the budget and reducing the deficit.  The Meso Foundation has been a vocal advocate for Congress to act this year to avert a sequester.

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