BILL ANALYSIS Ó AB 159 Page 1 ASSEMBLY THIRD READING AB 159 (Calderon) As Amended April 28, 2015 Majority vote ------------------------------------------------------------------- |Committee |Votes |Ayes |Noes | | | | | | | | | | | |----------------+------+----------------------+--------------------| |Health |17-0 |Bonta, Maienschein, | | | | |Burke, Chiu, Gomez, | | | | |Gonzalez, | | | | | | | | | | | | | | |Roger Hernández, | | | | |Lackey, Nazarian, | | | | |Patterson, | | | | | | | | | | | | | | |Ridley-Thomas, | | | | |Rodriguez, Santiago, | | | | |Steinorth, Thurmond, | | | | |Waldron, Wood | | | | | | | |----------------+------+----------------------+--------------------| |Business & |14-0 |Bonilla, Jones, | | |Professions | |Baker, Bloom, Campos, | | | | |Chang, Dodd, Eggman, | | | | |Gatto, Holden, | | | | |Mullin, Ting, Wilk, | | AB 159 Page 2 | | |Wood | | | | | | | |----------------+------+----------------------+--------------------| |Appropriations |15-0 |Gomez, Bigelow, | | | | |Bloom, Bonta, | | | | |Calderon, Chang, | | | | |Daly, Eggman, Eduardo | | | | |Garcia, Holden, | | | | |Jones, Rendon, | | | | |Wagner, Weber, Wood | | | | | | | | | | | | ------------------------------------------------------------------- SUMMARY: Permits a manufacturer of a drug, biological product, or device (experimental drug or device) that has not yet received federal or state approval to market to make the product available to eligible patients with serious or immediately life-threatening diseases, as specified. EXISTING STATE LAW: 1)Prohibits, under the Sherman Food, Drug, and Cosmetic Law, the sale, delivery, or giving away of a new drug or device unless it is either a) A new drug or new device for which the California Department of Public Health (DPH) has approved a new drug or device application, and has not withdrawn, terminated, or suspended that approval; or, b) A new drug, and a new drug application has been approved for it by the United States Food and Drug Administration (FDA). EXISTING FEDERAL LAW: Prohibits any new drug from being introduced into interstate commerce unless an application has been approved by the FDA. AB 159 Page 3 FISCAL EFFECT: According to the Assembly Appropriations Committee, minor and absorbable costs to DPH, the Medical Board of California, and the Osteopathic Medical Board of California to receive and review biannually reports from institutional review boards on the requests for treatment with an investigational product. 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. Background. 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 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. Expanded Access to investigational products through the FDA. Patients who are not eligible for a clinical trial because of where they live, their age, or some other disqualifying factor AB 159 Page 4 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. Court History. In 2005, the Abigail Alliance for Better Access to Developmental Drugs, with the mission of facilitating access to experimental drugs for patients, sued the FDA, claiming a constitutional due process right for terminally ill patients to access unapproved drugs. A United States District court disagreed and the case was heard by the United States Court of Appeals for the District of Columbia Circuit. In 2006, a divided three-judge panel of the District of Columbia Circuit agreed with the Alliance, finding that where there are no other FDA-approved treatment options, a terminally ill patient has a constitutionally-protected "fundamental right" to access investigational drugs. In 2007, the District of Columbia Circuit AB 159 Page 5 reheard the case "en banc" and reversed the panel decision. The Alliance then filed a petition asking the United States Supreme Court to hear the case but the Supreme Court denied the petition, so for now the Court of Appeals decision stands. Support. The Los Angeles County Board of Supervisors supports this bill. Opposition. The California Medical Association, California Nurse Association/National Nurses United (CNA), Association of Northern California Oncologists oppose this bill, citing patient safety and other concerns. The Association of Northern California Oncologists believe 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 violation for federal law. Statement of Concern. The Pharmaceutical Research and Manufacturers Association (PhRMA) filed a letter of concern with this Committee, stating that Right-to-Try legislation, while well-intentioned, could have serious unintended consequences that must be taken into consideration. Robust clinical trials are one of the key elements in bringing new therapies to market. PhRMA states that it is imperative that investigational drugs be used within the boundaries of carefully constructed and monitored clinical trials to ensure patient safety, and to glean key understandings about the safety, efficacy, and uses of investigational drugs. Furthermore, PhRMA is concerned with the lack of oversight and accountability in this legislation. Both clinical trials and the FDA's expanded access program require that patients be given these under the close supervisions of a AB 159 Page 6 physician and other health care providers to closely monitor patient reactions. Allowing blanket access to these drugs outside of monitored trials could yield catastrophic results for the patient. Analysis Prepared by: Dharia McGrew / HEALTH / (916) 319-2097 FN: 0000390