On this page you can find important Policy Reports from the nation's leading thought leaders in the Rare Disease Community.
From an article by Joann Donnellan – Today there are 10,000 known diseases in the United States, but only 500 have established treatments. Life-threatening diseases such as Alzheimer's, cancer, heart disease, and many others lack effective treatments or cures. The urgent need to find cures is impeded by the process of bringing promising drugs and devices to market. A new report by the Bipartisan Policy Center (BPC) recommends a series of policy actions that Congress can take to help reduce the time and cost of developing and delivering safe and effective medical products to patients. Such actions could considerably increase the competitiveness of U.S. companies in the global marketplace.
Although called rare or orphan diseases – defined as conditions affecting fewer than 200,000 people in the United States – there are more than 7,000 such conditions, affecting 30 million Americans. Collectively, rare diseases represent a significant public health challenge.
Quite often these are genetic, childhood conditions, in some cases caused by a single gene mutation that may or may not be inherited from a parent's genes. Because they are so rare, and many families may not have prior experience with the condition, simply getting a diagnosis can be an agonizing, years-long process. In addition, because these diseases are so uncommon, many doctors have no experience treating them and there may only be a handful of physicians in the world who have any experience with a given condition.
Even after diagnosis, patients still face barriers to care: 95 percent of rare diseases have not one single U.S. Food and Drug Administration (FDA) approved drug treatment. The absence of treatments for rare diseases actually used to be much worse. While many products and therapies were developed to treat cancer, heart disease and other common conditions, from 1973-1983, fewer than 10 rare disease therapies were developed.
On June 12th, the final rule to amend FDA's 1992 orphan drug regulations was published in the Federal Register. 78 Fed. Reg. 35117 (June 12, 2013). This final rule, which largely finalizes the revisions as proposed in October 2011, will be effective August 12, 2013. We previously posted on the details of the proposed rule, as well as litigation that is still ongoing concerning some of the issues discussed by FDA in the proposed and final rules.
Lexington, Massachusetts, US – April 9, 2013 – Shire plc (LSE: SHP, NASDAQ: SHPG) today launched a Rare Disease Impact Report, which uncovers the health, psycho-social, and economic impact of rare diseases on patient and medical communities in the United States (US) and United Kingdom (UK). The report, developed in collaboration with an external advisory board of thought leaders in the medical, advocacy, health policy and health economics fields, will be distributed at the World Orphan Drug Congress in Washington, DC (April 9-11) and is available for immediate download at www.rarediseaseimpact.com.
Today, the Pharmaceutical Research and Manufacturers of America (PhRMA) released a report entitled "Innovation in the Biopharmaceutical Pipeline: A Multi-Dimensional View." The report was conducted by researchers at the Analysis Group, and examines the drug pipeline to illustrate the type of research conducted by biopharmaceutical companies.
The rule of rescue describes the moral impulse to save identifiable lives in immediate danger at any expense. Think of the extremes taken to rescue a small child who has fallen down a well, a woman pinned beneath the rubble of an earthquake, or a submarine crew trapped on the ocean floor. No effort is deemed too great. Yet should this same moral instinct to rescue, regardless of cost, be applied in the emergency room, the hospital, or the community clinic?