Historic Time in Treatment of Rare Disease as Promising Drugs Reach Phase 3 Trials
Legislation to Help Speed Research for Cystic Fibrosis and Other Rare Diseases
Cystic Fibrosis Foundation Drug Development Model Spawns More Than 30 Promising Therapies
Foundation and Legislative Supporters Pushed for Bill
Bill Would Remove Financial Penalties for Participating in Research Studies
Revised Law Includes Designated Funding for Rare Diseases
Ten patient and provider groups, representing millions of Americans, issued the following statement in response to the release of the nonpartisan Congressional Budget Office's (CBO) scores of two pieces of health care reform legislation currently being considered by the U.S. Senate.
Cost effectiveness analysis must be used carefully and as part of a comprehensive evaluation of the value a treatment, such as highly effective modulators, provide to people living with cystic fibrosis.