,,ii #aagth ,,congress #ast ,session ,h4 ,r4 #cecg ,,in ,,the ,,senate ,,of ,,the ,,united ,,states ,december #i1 #bjba,received ,,an ,,act ,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 scientific research utilizing data from expanded access to investigational drugs for individuals who are not otherwise eligible for clinical trials for the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis4 ,in the case of a participating entity 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 will be used to support research or development related to the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis4 7#c7 ,noninterference with clinical trials ,an application submitted under paragraph 7#a7 shall include a description of how the proposed expanded access program will be designed so as not to interfere with patient enrollment in ongoing clinical trials for investigational therapies for the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis4 7c7 ,selection ,consistent with sections #djf and #dib of the ,public ,health ,service ,act 7#db ,u4,s4,c4 #bhd;a1 #bhi;a71 the ,secretary shall1 in determining whether to award a grant under this section1 confirm that-- 7#a7 such grant will be used to support a scientific research objective relating to the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis 7as described in subsection 7a772 7#b7 such grant shall not have the effect of diminishing eligibility for1 or impeding enrollment of1 ongoing clinical trials for the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis by determining that individuals who receive expanded access to investigational drugs through such a grant are not eligible for enrollment in-- 7,a7 ongoing clinical trials that are registered on ,clinical,trials4gov 7or successor website71 with respect to a drug for the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis2 or 7,b7 clinical trials for the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis for which an exemption under section #eje7;i7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cee7;i77 has been granted by the ,food and ,drug ,administration and which are expected to begin enrollment within one year2 and 7#c7 the resulting project funded by such grant will allow for equitable access to investigational drugs by minority and underserved populations4 7d7 ,use of funds ,a participating entity shall use funds received through the grant-- 7#a7 to pay the manufacturer or sponsor for the direct costs of the investigational drug1 as authorized under section #cab4h7;d7 of title #ba1 ,code of ,federal ,regulations 7or successor regulations71 to prevent1 diagnose1 mitigate1 treat1 or cure amyotrophic lateral sclerosis that is the subject of an expanded access request described in subsection 7a71 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 such drug4 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;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;i77 to prevent1 diagnose1 mitigate1 treat1 or cure amyotrophic lateral sclerosis4 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 health care facility1 or network of facilities1 at which patients participating in a participating clinical trial receive an investigational drug through such trial4 7f7 ,sunset ,the ,secretary may not award grants under this section on or after ,september #cj1 #bjbf4 #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 mechanisms with such eligible entities1 for the purpose of advancing the understanding of neurodegenerative diseases and fostering the development of treatments for amytrophic lateral sclerosis and other rare neurodegenerative diseases4 ,the ,partnership shall-- 7#a7 establish partnerships and consortia 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 with clinical and other technical expertise in the field of biomedical sciences 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 molecular 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 basic research and research associated with phase #c clinical trials conducted with respect to investigational drugs that are the subjects of expanded access requests 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 ,commissioner of ,food and ,drugs shall publish on the website of the ,food and ,drug ,administration 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 diseases2 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 decisionmaking2 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 of the relevant centers and 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 ,services1 acting through the ,commissioner of ,food and ,drugs1 shall 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 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 through-- 7,a7 the use of master protocols and adaptive and add-on clinical trial designs2 and 7,b7 efforts to establish new or leverage existing clinical trial networks4 #f4 ,,gao report ,not later than #d years after the date of the enactment of this ,act1 the ,comptroller ,general of the ,united ,states shall 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 containing-- 7#a7 with respect to grants awarded under the program established under section #b-- 7,a7 an analysis of what is known about the impact of such grants on research or development related to the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis2 and 7,b7 data concerning such grants1 including-- 7i7 the number of grants awarded2 7ii7 the participating entities to whom grants were awarded2 7iii7 the value of each such grant2 7iv7 a description of the research each such grant was used to further2 7v7 the number of patients who received expanded access to an investigational drug to prevent1 diagnose1 mitigate1 treat1 or cure amyotrophic lateral sclerosis under each grant2 7vi7 whether the investigational drug that was the subject of such a grant was approved by the ,food and ,drug ,administration2 and 7vii7 the average number of days between when a grant application is submitted and when a grant is awarded2 and 7#b7 with respect to grants awarded under the program established under section #e-- 7,a7 an analysis of what is known about the impact of such grants on research or development related to the prevention1 diagnosis1 mitigation1 treatment1 or cure of amyotrophic lateral sclerosis2 7,b7 an analysis of what is known about how such grants increased efficiency and productivity of the clinical development of therapies1 including through the use of clinical trials that operated with common master protocols1 or had adaptive or add-on clinical trial designs2 and 7,c7 data concerning such grants1 including-- 7i7 the number of grants awarded2 7ii7 the participating entities to whom grants were awarded2 7iii7 the value of each such grant2 7iv7 a description of the research each such grant was used to further2 and 7v7 whether the investigational drug that was the subject of such a grant received approval by the ,food and ,drug ,administration4 #g4 ,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 ,passed the ,house of ,representatives ,december #h1 #bjba4;,cheryl ,l4 ,johnson1,clerk4