,,ii #aafth ,,congress #b;d ,session ,s4 #dhfg ,,in ,,the ,,senate ,,of ,,the ,,united ,,states ,october #bf 7legislative day1 ,october #ai71 #bjbj,mr4 ,coons 7for himself and ,ms4 ,murkowski7 introduced the following bill2 which was read twice and referred to the ,committee on ,health1 ,education1 ,labor1 and ,pensions ,a ,,bill ,to direct the ,secretary of ,health and ,human ,services to support research on1 and expanded access to1 investigational drugs for amyotrophic lateral sclerosis1 and for other purposes4 #a4 ,short title ,this ,act may be cited as the ,accelerating ,access to ,critical ,therapies for ,,als ,act4 #b4 ,grants for research on and expanded access to experimental therapies for ,,als 7a7 ,in ,general ,the ,secretary of ,health and ,human ,services shall award grants to eligible entities for purposes of supporting research on1 and expanded access for individuals to1 investigational drugs for the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis pursuant to an expanded access request submitted1 and allowed to proceed by the ,secretary1 under section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb74 7b7 ,application ,an eligible entity seeking a grant under this section shall submit to the ,secretary an application at such time1 in such manner1 and containing such information as the ,secretary shall specify4 ,such application shall specify a research objective relating to expanding access to investigational drugs 7as described in subsection 7a77 that would be supported by the award of such grant4 7c7 ,selection ,not later than #ij days after the date of submission of an application for a grant under this section1 the ,secretary shall determine whether to award the grant1 taking into consideration whether awarding such grant will support a research objective relating to expanding access to investigational drugs 7as described in subsection 7a771 consistent with the mission of the ,national ,institutes of ,health4 7d7 ,use of funds ,an eligible entity may use funds received through the grant-- 7#a7 to pay the manufacturer or sponsor for the direct costs of such drug 7as authorized under section #cab4h7;d7 of title #ba1 ,code of ,federal ,regulations 7or successor regulations771 if such costs are justified as part of peer review of the grant2 7#b7 for the entity's direct costs incurred in providing such drug consistent with the research mission of the grant2 or 7#c7 for the direct and indirect costs of the entity in conducting research with respect to the drug involved4 7e7 ,definitions ,in this section3 7#a7 ,the term eligible entity means a participating clinical trial site or sites sponsored by a small business concern 7as defined in section #c7;a7 of the ,small ,business ,act 7#ae ,u4,s4,c4 #fcb7;a777 that is the sponsor of a drug that is the subject of an investigational new drug application under section #eje7;i7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee7;i774 7#b7 ,the term participating clinical trial means a phase #c clinical trial conducted pursuant to an exemption under section #eje7;i7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee7;i77 or section #cea7;a7 of the ,public ,health ,service ,act 7#db ,u4,s4,c4 #bfb7;a77 to investigate a drug intended to prevent1 diagnose1 mitigate1 treat1 or cure amyotrophic lateral sclerosis4 7#c7 ,the term participating clinical trial site means a nonprofit or public health care facility1 or network of facilities1 at which patients participating in a participating clinical trial receive an investigational drug through such trial4 #c4 ,,hhs ,collaborative for ,neurodegenerative ,diseases 7a7 ,establishment ,not later than one year after the date of the enactment of this ,act1 the ,secretary of ,health and ,human ,services shall establish and implement a ,collaborative for ,neurodegenerative ,diseases between the ,national ,institutes of ,health and the ,food and ,drug ,administration 7to be known and referred to in this section as the ,collaborative71 which shall-- 7#a7 enter into a cooperative agreement1 contract1 or other instrument with a private entity or entities under which such private entity or entities will operate the ,collaborative2 7#b7 focus on advancing regulatory improvements and scientific research that will support and accelerate the development and approval of drugs for patients with amyotrophic lateral sclerosis2 7#c7 foster the development of effective drugs that improve the lives of people that suffer from rare neurodegenerative diseases2 and 7#d7 share information with the ,secretary to advance the purposes specified in paragraph 7#c71 such as through carrying out the grant programs under sections #b and #e and developing the action plan under section #d4 7b7 ,gifts 7#a7 ,in general ,the ,collaborative may solicit and accept gifts1 grants1 and other donations1 establish accounts1 and invest and expend funds in support of pre-competitive research and research associated with phase #c and phase #d clinical trials conducted with respect to investigational drugs that are the subjects of expanded access applications under section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb74 7#b7 ,use ,in addition to any amounts appropriated for purposes of carrying out this section1 the ,collaborative may use1 without further appropriation1 any funds derived from a gift1 grant1 or other donation accepted pursuant to paragraph 7#a74 7c7 ,advisory panel 7#a7 ,in general ,the ,collaborative shall convene an advisory panel to conduct a review of the design and implementation of the programs authorized by this ,act4 ,such panel shall include representatives of patients1 treating physicians1 national organizations that facilitate provision of care services1 researchers1 drug sponsors1 drug manufacturers1 and ,federal agencies4 7#b7 ,report ,the advisory panel convened under paragraph 7#a7 shall1 not later than #ah months after the date of the enactment of this ,act1 submit to the ,committee on ,energy and ,commerce of the ,house of ,representatives and the ,committee on ,health1 ,education1 ,labor1 and ,pensions of the ,senate a report that contains-- 7,a7 the findings and conclusions of the review conducted under paragraph 7#a72 and 7,b7 recommendations for carrying out the programs under this ,act during the #b-year period following the submission of such report1 including recommendations relating to the inclusion of additional neurodegenerative diseases or disease areas within the grant programs under sections #b and #e4 7d7 ,duties and authorities ,the ,collaborative shall identify and implement strategies for the ,secretary-- 7#a7 for purposes of expediting the approval of drugs to treat amyotrophic lateral sclerosis1 including through coordination among the centers of the ,food and ,drug ,administration to achieve the goals specified in the draft guidance for drug sponsors entitled ,amyotrophic ,lateral ,sclerosis3 ,developing ,drugs for ,treatment ,guidance for ,industry published in ,september #bjai2 7#b7 to facilitate access to investigational drugs for amyotrophic lateral sclerosis pursuant to an expanded access request under section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb7 in a similar manner as investigational drugs for cancer are provided through ,project ,facilitate of the ,center of ,excellence for ,oncology2 7#c7 with respect to the rare neurodegenerative disease grant program established under section #e2 7#d7 to define or develop the regulatory and translational pathway for emerging therapeutic categories2 7#e7 to share1 within the ,collaborative1 findings and insights related to pre-competitive research and research associated with phase #c and phase #d clinical trials conducted with respect to investigational drugs that are the subjects of expanded access applications under section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb72 and 7#f7 to develop and implement an ongoing mechanism to share feedback and information and develop strategies with the neurodegenerative disease community1 including patients1 treating physicians1 national organizations that facilitate provision of care services1 access1 and research1 researchers1 drug sponsors1 drug manufacturers1 and ,federal agencies4 #d4 ,neurodegenerative disease action plan 7a7 ,in general ,not later than #f months after the date of the enactment of this ,act1 the ,secretary of ,health and ,human ,services shall publish an action plan describing actions the ,department of ,health and ,human ,services1 through the ,national ,institutes of ,health and ,food and ,drug ,administration1 intend to take during the #e-year period following publication of the plan with respect to program enhancements1 policy development1 regulatory science initiatives1 and other appropriate initiatives to-- 7#a7 foster the development of safe and effective drugs that improve the lives of people living with rare neurodegenerative diseases as quickly as possible2 and 7#b7 facilitate access to investigational drugs4 7b7 ,contents ,the action plan published under subsection 7a7 shall-- 7#a7 identify appropriate representation from within the ,food and ,drug ,administration and ,national ,institutes of ,health to be responsible for implementation of such action plan2 and 7#b7 include elements to facilitate-- 7,a7 interactions and collaboration between the ,food and ,drug ,administration1 including the review centers thereof1 and stakeholders including patients1 sponsors1 and external biomedical community2 7,b7 consideration of cross-cutting clinical and regulatory policy issues1 including consistency of regulatory advice and decision making2 7,c7 identification of key regulatory science and policy issues critical to advancing development of safe and effective drugs2 and 7,d7 engagement by staff of the relevant centers of the ,food and ,drug ,administration and other relevant offices of the ,food and ,drug ,administration and ,national ,institutes of ,health with the designated leadership of the ,collaborative4 #e4 ,rare ,neurodegenerative ,disease ,grant ,program ,the ,secretary of ,health and ,human ,services1 acting through the ,commissioner of ,food and ,drugs1 shall carry out a program of awarding grants to1 and contracts entered into with1 public and private entities to cover the costs of research on and development of interventions intended to prevent1 diagnose1 mitigate1 treat1 or cure amyotrophic lateral sclerosis and other life-threatening or severely debilitating neurodegenerative diseases1 including costs incurred with respect to the development and critical evaluation of tools1 methods1 and processes-- 7#a7 to characterize such neurodegenerative diseases and their natural history2 7#b7 to identify molecular targets for such neurodegenerative diseases2 and 7#c7 to increase efficiency and productivity of clinical development of therapies1 including advancing rational therapeutic development and working to establish clinical trial networks4 #f4 ,authorization of appropriations ,for purposes of carrying out this ,act1 there are authorized to be appropriated #ajj1jjj1jjj for each of fiscal years #bjbb through #bjbf4