BILL ANALYSIS                                                                                                                                                                                                    Ó



                                                                    AB 1668


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          Date of Hearing:  April 6, 2016


                        ASSEMBLY COMMITTEE ON APPROPRIATIONS


                               Lorena Gonzalez, Chair


          AB  
          1668 (Calderon) - As Amended March 7, 2016


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          |             |Business and Professions       |     |14 - 0       |
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          Urgency:  No  State Mandated Local Program:  NoReimbursable:  No


          SUMMARY:


          This bill sets up a legal structure whereby individuals with  
          serious or immediately life-threatening conditions can gain  
          access to investigational drugs, biological products, or medical  
          devices (investigational products). Specifically, this bill: 








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             1)   Defines investigational drug, biological product, or  
               device as a drug, biological product, or device that has  
               successfully completed phase one of a clinical trial  
               approved by the United States Food and Drug Administration  
               (FDA), but has not been approved for general use and  
               remains under investigation in an approved clinical trial.



             2)   Allows, but does not require, the manufacturer of an  
               investigational product to make the product available to an  
               eligible patient, as defined.  Specifies that the  
               manufacturer may provide the product for free or require a  
               patient to pay for the costs of the product. 

             3)   Defines an eligible patient as one who has met a number  
               of specified criteria, including having a serious or  
               life-threatening condition, as defined, and having  
               considered all FDA-approved treatment options. 



             4)   Allows, but does not require, a health plan or insurer  
               to provide coverage for the investigational product, or the  
               costs of services related to the use of the investigational  
               product. 



             5)   Prohibits a state regulatory board from taking  
               disciplinary action against a physician's license based on  
               the recommendation or prescription of, or treatment with,  
               the investigational product, provided it is consistent with  
               protocol approved by an institutional review board.   
               Similarly prohibits a state agency from altering a  
               recommendation made to the federal Centers for Medicare and  
               Medicaid Services regarding a health care provider's  








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               certification to participate in the Medicare or Medicaid  
               program based on such recommendation, prescription, or  
               treatment. 



             6)   Specifies it does not create a private cause of action  
               against a manufacturer of an investigational product, or  
               against any other person or entity involved in the care of  
               an eligible patient using the investigational product,  
               provided certain conditions are met.





             7)   Requires institutional review boards to report  
               information about requests for investigational products to  
               the California Department of Public Health (CDPH), the  
               Medical Board of California (MBC), and the Osteopathic  
               Medical Board of California (OBMC).
          FISCAL EFFECT:


          1)Minor and absorbable costs to CDPH, MBC, and OMBC to receive  
            and review biannually reports from institutional review boards  
            on the requests for treatment with an investigational product  
            (various funds).


          2)$380,000 in the first year of implementation, and $200,000  
            ongoing to Department of Managed Health Care (Managed Care  
            Fund). First year costs are higher because of probable  
            additional legal analysis and processing Public Records Act  
            (PRA) requests for experimental procedures previously  
            authorized or rejected by health plans.  Ongoing costs are  
            related to patient and provider inquiries to the DMHC's Help  
            Center. DMHC indicates any of the investigational and/or  
            experimental Independent Medical Review (IMR) requests require  








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            thorough analysis to determine if literature and studies on  
            the treatments are available, and to review clinical trials,  
            if completed or available. 


          3)The California Department of Insurance may, similarly to DMHC,  
            experience unknown costs related to IMR requests, PRA  
            requests, or other requests for other assistance to the extent  
            these requests increase. (Insurance Fund).  


          COMMENTS:


          1)Purpose.  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.
          


          2)Background.  Prior to being available for sale, drugs and  
            devices must pass through clinical trials regulated by the  
            FDA.  Phase one of these trials establishes safety 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 stage 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.  











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            There are, however, other ways to gain access to drugs that  
            are not yet approved. Experimental treatments are often  
            available by participating in a clinical trial.  If a patient  
            is unable to enroll in a clinical trial, the FDA expanded  
            access exemption, also called "compassionate use," provides a  
            pathway for patients to gain access to investigational drugs  
            or devices for serious diseases or conditions.  A licensed  
            physician is able to apply for access on behalf of the  
            patient. The physician must be willing to commit to oversee  
            the treatment, work with the manufacturing company and FDA,  
            obtain the drugs, monitor the patient during the course of  
            treatment, and file necessary paperwork.  The FDA states that  
            they receive approximately 1,000 such applications per year,  
            and has approved more than 99% of those applications.  Critics  
            of this process have raised concerns that the application is  
            too cumbersome for physicians and patients to complete. In  
            February 2015, the FDA proposed a new, much shorter form,  
            which the agency has stated is a top priority to finalize.   
            Based on regulatory timelines, it is possible this new form  
            may be available later this year. 





          3)Support. The Los Angeles County Board of Supervisors and the  
            Amyotrophic Lateral Sclerosis Association Golden West Chapter  
            support this bill, they support expanded use of experimental  
            treatments.



          4)Opposition. The California Nurse Association and the Medical  
            Oncology Association of Southern California oppose this bill.   
            The 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.










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          5)Prior Legislation.  
            


             a)   AB 159 (Calderon) of 2015 was identical to this bill.   
               It was vetoed by Governor Brown, who stated, "Patients with  
               life threatening conditions should be able to try  
               experimental drugs, and the FDA's compassionate use program  
               allows this to happen. The proposed changes to this program  
               will streamline access to these drugs. Before authorizing  
               an alternative state pathway, we should give this federal  
               expedited process a chance to work."  This bill is similar  
               to AB 159 and does not appear to address the veto message.



             b)   SB 149 (Stone) of 2015 was largely similar to this bill  
               and to AB 159, and was held on the Suspense File of this  
               committee. 



             c)   SB 715 (Anderson) of 2015 was also similar to this bill,  
               and was referred to Senate Health Committee and not heard. 
          Analysis Prepared by:Lisa Murawski / APPR. / (916)  
          319-2081