BILL ANALYSIS                                                                                                                                                                                                    Ó



                                                                     AB 159


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          CONCURRENCE IN SENATE AMENDMENTS


          AB  
          159 (Calderon)


          As Amended  July 6, 2015


          Majority vote


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          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


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          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


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          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.









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          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