BILL ANALYSIS Ó AB 159 Page 1 CONCURRENCE IN SENATE AMENDMENTS AB 159 (Calderon) As Amended July 6, 2015 Majority vote -------------------------------------------------------------------- |ASSEMBLY: |74-0 |(May 18, 2015) |SENATE: |40-0 |(September 1, | | | | | | |2015) | | | | | | | | | | | | | | | -------------------------------------------------------------------- Original Committee Reference: HEALTH SUMMARY: Authorizes the manufacturer of an investigational drug to make an investigational drug that is not yet approved by the United States Food and Drug Administration (FDA) available to a patient, under certain circumstances. The Senate amendments limit the patients able to participate to those with an immediately life-threatening disease or condition, adds types of information about the treatment that is reported by the institutional review board to agencies collecting the data, removes language prohibiting an official, employee, or agent of this state from blocking or attempting to block an eligible patient's access to an investigational drug or device, and clarifies language stating that this bill does not create a private right of action including but not limited to, product liability, medical negligence, or wrongful death against a physician or manufacturer involved in the use of an investigational drug pursuant to this bill. AB 159 Page 2 EXISTING STATE LAW prohibits, under the Sherman Food, Drug, and Cosmetic Law, the sale, delivery, or giving away of a drug or device unless it has been approved to market by either the FDA or the California Department of Public Health. EXISTING FEDERAL LAW prohibits any new drug from being introduced into interstate commerce unless an application has been approved by the FDA. FISCAL EFFECT: According to the Senate Appropriations Committee, one-time costs of $20,000 and ongoing costs of $10,000 per year to review plan filings by the Department of Insurance (Insurance Fund). Additionally, one-time costs of $380,000 in 2015-16 to respond to requests for information about prior independent medical review decisions and ongoing costs of $210,000 per year to respond to consumer complaints and requests for independent medical review for access to investigational drugs by the Department of Managed Health Care (Managed Care Fund). Uncertain impact on state-funded CalPERS health care coverage (General Fund and special funds). COMMENTS: According to the author, this bill removes barriers to accessing potentially life-saving drugs for terminally-ill patients and doctors who believe an investigational drug or device could be their last hope for survival. These patients may seek compassionate use exemptions from the FDA, but the process is cumbersome and approval comes too late for many. This bill removes barriers for patients who need to immediately obtain investigational treatments, while also protecting physicians, hospitals, and manufacturers from retribution. A clinical trial is a study that is carefully designed to test the benefits and risks of a specific medical treatment or intervention, such as a new drug. The FDA requires a multi-phase clinical trials process to be completed before deciding if an investigational medicine is safe and effective AB 159 Page 3 for a broader patient population. Experimental drugs and devices go through clinical trials, stage one of which establishes very basic safety level and assesses toxicity. Once a drug or device passes stage one, there are two more phases before being approved for sale. This bill would allow manufacturers to make available investigational products, that have passed phase one trials, to patients meeting specified criteria, and would protect from liability doctors who recommend or prescribe the investigational products, as well as manufacturers who make them available. Patients who are not eligible for a clinical trial because of where they live, their age, or some other disqualifying factor have the option to seek expanded access if they have serious or life-threatening conditions and no comparable or satisfactory alternative is available. The FDA has in place processes to allow patients access: expanded access, which is sometimes called "compassionate use," supplements the clinical trials process. A licensed physician is able to apply for expanded access on behalf of the patient. The FDA states that they receive approximately 1,000 expanded use applications per year, and has approved more than 99% of those applications. In the last four years, the FDA has denied only 33 of nearly 6,000 expanded access requests. Emergency requests are often granted immediately over the phone. For non-emergencies, the FDA states that they strive to respond promptly and, in general, approve the request within 30 days. Critics of the FDA process have raised concerns that the expanded access application is too cumbersome for physicians and patients to complete. Critics claim that it currently requires an application that takes doctors 100 hours to fill out. On February 4, 2015, the FDA announced that it would be streamlining the expanded use application process, and stated that the proposed application will allow physicians to apply for experimental drugs in just 45 minutes. The FDA draft guidance on the new application process is currently in the public comment period and the new final rule should be released shortly. AB 159 Page 4 The Los Angeles County Board of Supervisors supports this bill, stating that seriously ill patients who have exhausted their options to find a cure and who have identified physicians and a pharmaceutical company willing to assist them, deserve the right to experimental treatments that could improve their health conditions or prolong their lives. The California Nurses Association/National Nurses United (CNA) and the Association of Northern California Oncologists (ANCO) oppose this bill, citing patient safety and other concerns. ANCO states that this bill threatens the viability of adult clinical trial enrollment and would result in slowing the development of cancer care in the future. CNA points out that "nothing in this legislation impacts the availability of drugs to terminally ill patients unless the manufacturers of the drugs allow it to be used in advance of FDA approval". If a manufacturer provided an experimental drug or device to a patient without FDA approval under this law, they would be in direct violation of federal law. Analysis Prepared by: Dharia McGrew / HEALTH / (916) 319-2097 FN: 0001905