,,ii #aagth ,,congress #ast ,session ,s4 #ahac ,,in ,,the ,,senate ,,of ,,the ,,united ,,states ,may #be1 #bjba ,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 therapies for ,,als 7a7 ,in general ,the ,secretary of ,health and ,human ,services 7referred to in this section as the ,secretary7 shall award grants to participating entities for purposes of expanded access for individuals to investigational drugs for the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis4 ,in the case of an applicant seeking such a grant1 an expanded access request must be 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;bbb7 and part #cab of title #ba1 ,code of ,federal ,regulations 7or any successor regulations71 before the application for such grant is submitted4 7b7 ,application 7#a7 ,in general ,a participating 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 7#b7 ,use of data ,an application submitted under paragraph 7#a7 shall include a description of how data generated through an expanded access request under section #efa of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb7 with respect to the investigational drug involved may be used by the ,secretary to support research or development related to the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis or other rare neurodegenerative diseases4 7c7 ,selection ,not later than #abj 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-- 7#a7 whether awarding such grant will support a research objective relating to expanding access to investigational drugs 7as described in subsection 7a772 and 7#b7 whether awarding such a grant may have the effect of diminishing eligibility for1 or impeding enrollment of1 ongoing clinical investigations4 7d7 ,use of funds ,a participating 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 participating 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;a77 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 public-private partnership for rare neurodegenerative diseases 7a7 ,establishment ,not later than one year after the date of enactment of this ,act1 the ,secretary of ,health and ,human ,services 7referred to in this section as the ,secretary7 shall establish and implement a ,public-,private ,partnership for ,neurodegenerative ,diseases between the ,national ,institutes of ,health1 the ,food and ,drug ,administration1 and one or more eligible entities 7to be known and referred to in this section as the ,partnership7 through cooperative agreements1 contracts1 or other appropriate instruments with such eligible entities1 for the purpose of developing treatments for amytrophic lateral sclerosis and other rare neurodegenerative diseases4 ,the ,partnership shall-- 7#a7 establish partnerships1 consortia1 and collaborations with other public and private entities and individuals with expertise in amyotrophic lateral sclerosis and other rare neurodegenerative diseases for the purposes described in this subsection2 7#b7 focus on advancing regulatory science and scientific research that will support and accelerate the development and review of drugs for patients with amyotrophic lateral sclerosis and other rare neurodegenerative diseases2 and 7#c7 foster the development of effective drugs that improve the lives of people that suffer from amyotrophic lateral sclerosis and other rare neurodegenerative diseases4 7b7 ,eligible entity ,in this section1 the term eligible entity means an entity that-- 7#a7 is-- 7,a7 an institution of higher education 7as such term is defined in section #ajja of the ,higher ,education ,act of #aife 7#bj ,u4,s4,c4 #ajja77 or a consortium of such institutions2 or 7,b7 an organization described in section #eja7;c77#c7 of the ,internal ,revenue ,code of #aihf and exempt from tax under subsection 7a7 of such section2 7#b7 has experienced personnel and demonstrated connection to the patient population2 7#c7 demonstrates to the ,secretary's satisfaction that the entity is capable of identifying and establishing collaborations between public and private entities and individuals with expertise in neurodegenerative diseases1 including patients1 in order to facilitate-- 7,a7 development and critical evaluation of tools1 methods1 and processes-- 7i7 to characterize neurodegenerative diseases and their natural history2 7ii7 to identify drug targets for neurodegenerative diseases2 and 7iii7 to increase efficiency1 predictability1 and productivity of clinical development of therapies1 including advancement of rational therapeutic development and establishment of clinical trial networks2 and 7,b7 securing funding for the ,partnership from ,federal and non-,federal governmental sources1 foundations1 and private individuals2 and 7#d7 provides an assurance that the entity will not accept funding for a ,partnership project from any organization that manufactures or distributes products regulated by the ,food and ,drug ,administration unless the entity provides assurances in its agreement with the ,secretary that the results of the project will not be influenced by any source of funding4 7c7 ,gifts 7#a7 ,in general ,the ,partnership 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 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 ,partnership may use1 without further appropriation1 any funds derived from a gift1 grant1 or other donation accepted pursuant to paragraph 7#a74 #d4 ,,als and other rare neurodegenerative disease action plan 7a7 ,in general ,not later than #f months after the date of enactment of this ,act1 the ,secretary of ,health and ,human ,services shall publish on the website of the ,department of ,health and ,human ,services an action plan describing actions the ,food and ,drug ,administration intends 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 or extend1 or both1 the lives of people living with amyotrophic lateral sclerosis and other rare neurodegenerative diseases as quickly as possible2 and 7#b7 facilitate access to investigational drugs for amyotrophic lateral sclerosis and other rare neurodegenerative diseases4 7b7 ,contents ,the initial action plan published under subsection 7a7 shall-- 7#a7 identify appropriate representation from within the ,food and ,drug ,administration to be responsible for implementation of such action plan2 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 the external biomedical research 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 enhancement of collaboration and engagement by staff of the relevant centers of the ,food and ,drug ,administration and other relevant offices of the ,food and ,drug ,administration with other operating divisions within the ,department of ,health and ,human ,services1 the ,partnership1 and the broader neurodegenerative disease community2 and 7#c7 be subject to revision1 as determined appropriate by the ,secretary of ,health and ,human ,services4 #e4 ,,fda rare neurodegenerative disease grant program ,the ,secretary of ,health and ,human ,services shall use funds made available under section #f to award grants and contracts to public and private entities to cover the costs of research on1 and development of interventions intended to prevent1 diagnose1 mitigate1 treat1 or cure1 amyotrophic lateral sclerosis and other rare life-threatening or severely debilitating neurodegenerative diseases in adults and children1 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 new or leverage existing 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