BILL ANALYSIS                                                                                                                                                                                                    Ó



                                                                       AB 159


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          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,   |                    |








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          |                |      |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.  









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          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  








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          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  








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          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  








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          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