,,ii #aagth ,,congress #ast ,session ,s4 #fgj ,,in ,,the ,,senate ,,of ,,the ,,united ,,states ,march #aj1 #bjba,ms4 ,klobuchar 7for herself and ,mr4 ,wicker7 introduced the following bill2 which was read twice and referred to the ,committee on ,health1 ,education1 ,labor1 and ,pensions ,a ,,bill ,to amend the ,federal ,food1 ,drug1 and ,cosmetic ,act to accelerate development of therapies across the spectrum of rare diseases and conditions and facilitate patient access to such therapies1 and for other purposes4 #a4 ,short title ,this ,act may be cited as the ,speeding ,therapy ,access ,today ,act of #bjba4 #b4 ,table of contents ,the table of contents of this ,act is as follows3 ,sec4 #a4 ,short title4,sec4 #b4 ,table of contents4,sec4 #c4 ,intercenter ,institute on ,rare ,diseases and ,conditions4,sec4 #d4 ,rare ,disease and ,condition ,drug ,advisory ,committee4,sec4 #e4 ,grants and contracts for development of drugs for rare diseases and conditions4 #c4 ,intercenter ,institute on ,rare ,diseases and ,conditions 7a7 ,establishment required ,the first sentence of section #ajad7;a7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cii;g7a77 is amended by inserting 1 at least one of which shall be focused on rare diseases and conditions before the period at the end of the sentence4 7b7 ,timing of establishment ,subsection 7c7 of section #ajad of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4 #cii;g7 is amended to read as follows3 7c7 ,timing ,not later than the date that is #a year after the date of enactment of the ,speeding ,therapy ,access ,today ,act of #bjba1 the ,secretary shall establish1 in accordance with this section and section #ebi;,b1 an ,institute under subsection 7a7 focused on rare diseases and conditions1 to be known as the ,intercenter ,institute on ,rare ,diseases and ,conditions4 4 7c7 ,responsibilities ,subchapter ,b of chapter ,v of the ,federal ,food1 ,drug1 and ,cosmetic ,act 7relating to drugs for rare diseases or conditions7 is amended by inserting after section #ebi;,a of such ,act 7#ba ,u4,s4,c4 #cfj;ff-#a7 the following new section3 #ebi;,b4 ,intercenter ,institute on ,rare ,diseases and ,conditions 7a7 ,responsibilities ,in addition to carrying out activities listed in section #ajad7;a71 the ,intercenter ,institute on ,rare ,diseases and ,conditions shall-- 7#a7 serve as the ,food and ,drug ,administration's coordinating office for engagement with rare disease and condition stakeholders1 complementing but not supplanting engagement activities between stakeholders and the review divisions2 7#b7 build1 within the ,food and ,drug ,administration1 knowledge and understanding associated with the review of medical products to treat rare diseases and conditions1 including advancements in trial design1 statistical analysis1 regulatory science1 product manufacturing1 and other topics as determined by the ,secretary2 7#c7 implement cross-center rare disease and condition-focused meetings and policy development2 7#d7 coordinate rare disease and condition-specific regulatory science initiatives2 7#e7 facilitate stakeholder engagement to the external community and international regulatory agencies on rare disease and condition product development2 7#f7 establish and implement the ,accelerating ,lifesavings ,therapies in ,treating ,ultra-rare ,disease ,entities ,program under subsection 7b72 and 7#g7 establish and carry out the rare disease and condition third-party payor program under subsection 7d74 7b7 ,,altitude ,program 7#a7 ,in general ,the ,intercenter ,institute shall establish and implement a program1 to be known as the ,accelerating ,lifesavings ,therapies in ,treating ,ultra-rare ,disease ,entities ,program1 to identify and make recommendations to address current and emerging regulatory science and public policy challenges associated with developing medical products to treat rare diseases or conditions in an individual or very small populations4 7#b7 ,issues ,the program under paragraph 7#a7 shall focus on issues including-- 7,a7 manufacturing standards for therapies described in such paragraph1 including in non-industry settings2 7,b7 trial designs and metrics2 7,c7 regulatory flexibilities for abbreviated toxicology studies1 overlapping animal studies1 and patient dosing2 7,d7 regulatory science1 chemistry1 manufacturing1 and other needs associated with developing such therapies2 and 7,e7 other issues as determined by the ,secretary4 7c7 ,proposals for amending labels 7#a7 ,stakeholder group ,not later than #ahj days after the date of enactment of this section1 the ,intercenter ,institute shall convene a meeting of stakeholders from the rare disease community1 including patients1 caregivers1 product manufacturers1 third-party payors1 and others1 to consider potential amendments to labels for medical products to treat rare diseases or conditions approved pursuant to a pathway under section #ejf4 7#b7 ,guidance ,not later than #ij days after the date of the meeting under paragraph 7#a71 the ,secretary shall issue guidance to propose changes to how the labels of medical products to treat rare diseases or conditions demonstrate clinical benefits and reflect relevant scientific data including surrogate endpoints4 7d7 ,rare ,disease and ,condition ,third-,party ,payor ,program 7#a7 ,in general ,the ,intercenter ,institute shall establish and carry out a voluntary rare disease and condition early third-party payor feedback program-- 7,a7 to inform coverage policies for rare disease therapies2 and 7,b7 to inform clinical trial design1 patient engagement1 and other data collections4 7#b7 ,program requirements ,the program under paragraph 7#a7 shall-- 7,a7 facilitate voluntary communication between sponsors of medical products to treat rare diseases and conditions and third-party payors2 and 7,b7 require participation of the ,centers for ,medicare ,medicaid ,services with representation from-- 7i7 the ,center for ,medicare2 and 7ii7 the ,center for ,medicaid and ,,chip ,services4 7#c7 ,annual report ,the ,intercenter ,institute shall-- 7,a7 on an annual basis1 submit a report to that ,congress on-- 7i7 the participation within the program under paragraph 7#a72 and 7ii7 the impacts of the program under paragraph 7#a72 and 7,b7 post each such report on the public website of the ,intercenter ,institute4 7#d7 ,bulletin to ,medicaid directors ,following the approval1 clearance1 or authorization by the ,food and ,drug ,administration of a medical product to treat a rare disease or condition1 the ,secretary shall issue a bulletin to ,state ,medicaid directors containing information to help inform coverage decisions on the product by ,state ,medicaid and ,children's ,health ,insurance programs4 7e7 ,definition ,in this section1 the terms ,intercenter ,institute on ,rare ,diseases and ,conditions and ,intercenter ,institute refer to the ,intercenter ,institute on ,rare ,diseases and ,conditions established pursuant to section #ajad4 4 #d4 ,rare ,disease and ,condition ,drug ,advisory ,committee ,subchapter ,b of chapter ,v of the ,federal ,food1 ,drug1 and ,cosmetic ,act is further amended by inserting after section #ebi;,b of such ,act1 as inserted by section #c1 the following new section3 #ebi;,c4 ,rare ,disease and ,condition ,drug ,advisory ,committee 7a7 ,in general ,the ,secretary shall establish and maintain a committee1 to be known as the ,rare ,disease and ,condition ,drug ,advisory ,committee 7in this section referred to as the ,advisory ,committee74 7b7 ,duty of ,committee ,the ,advisory ,committee shall advise the ,secretary on issues associated with development of therapies to treat rare diseases or conditions4 7c7 ,specific issues ,in advising the ,secretary1 the ,advisory ,committee may address issues including-- 7#a7 modified or new regulatory pathways to support review of therapies2 7#b7 clinical trial design needs1 including development of innovative approaches to clinical trials2 7#c7 qualifications of biomarkers or other drug development tools for use in reviews2 7#d7 modified or new standards to support the review of already marketed drugs being evaluated for repurposing to treat a rare disease or condition2 and 7#e7 issues-- 7,a7 that pertain to an application for approval of a therapy to treat a rare disease or condition2 and 7,b7 with respect to which a review division has requested that the ,advisory ,committee provide advice4 7d7 ,membership 7#a7 ,in general ,the ,advisory ,committee shall consist of-- 7,a7 not more than #ae members appointed by the ,secretary in accordance with paragraph 7#b72 and 7,b7 the nonvoting ex officio members under paragraph 7#c74 7#b7 ,appointed members 7,a7 ,special ,government employees ,members of the ,advisory ,committee appointed pursuant to paragraph 7#a77;,a7 shall serve as special ,government employees 7as defined in section #bjb7;a7 of title #ah1 ,united ,states ,code74 7,b7 ,eligibility ,to be eligible for appointment pursuant to paragraph 7#a77;,a71 an individual shall-- 7i7 be eligible to serve as special ,government employee 7as defined in section #bjb7;a7 of title #ah1 ,united ,states ,code72 and 7ii7 have expertise in the fields of public policy1 law1 regulatory policy1 economics1 patient-focused product development1 or patient advocacy4 7,c7 ,composition ,of the members of the ,advisory ,committee appointed pursuant to paragraph 7#a77;,a7-- 7i7 up to #aj shall be selected from among experts in the disciplines relevant to the activities of the ,intercenter ,institute on ,rare ,diseases and ,conditions1 to include at least one expert in each of-- 7,i7 rare disease product development2 7,,ii7 conducting clinical trials with respect to rare diseases and conditions1 including with respect to very small patient populations2 7,,iii7 rare disease and condition natural history and related studies2 7,,iv7 health economics pertaining to the development of medical products for rare diseases or conditions2 7,v7 manufacturing and related needs associated with medical products for rare diseases or conditions2 and 7,,vi7 patient experience data collection2 and 7ii7 up to #e shall be selected from the public1 to include-- 7,i7 at least #d individuals who are representatives of the rare disease patient community2 7,,ii7 at least one individual who is directly impacted by a rare disease or condition2 and 7,,iii7 at least one person who serves as a family caregiver to a person diagnosed with a rare disease or condition4 7#c7 ,nonvoting ex officio members ,the nonvoting ex officio members of the ,advisory ,committee under paragraph 7#a77;,b7 shall consist of the following3 7,a7 ,the ,secretary 7or the ,secretary's designee74 7,b7 ,the ,director of the ,intercenter ,institute on ,rare ,diseases and ,conditions4 7,c7 ,the ,director of the ,center for ,biologics ,evaluation and ,research 7or the ,director's designee74 7,d7 ,the ,director of the ,center for ,drug ,evaluation and ,research 7or the ,director's designee74 7,e7 ,the ,director of the ,center for ,devices and ,radiological ,health 7or the ,director's designee74 7,f7 ,the ,director of the ,national ,center for the ,advancing ,translational ,sciences of the ,national ,institutes of ,health 7or the ,director's designee74 7,g7 ,the ,administrator of the ,centers for ,medicare ,medicaid ,services 7or the ,administrator's designee74 7,h7 ,any additional officers or employees of the ,department of ,health and ,human ,services as the ,secretary determines necessary for the ,advisory ,committee to effectively carry out its functions4 7#d7 ,chair ,the ,chair of the ,advisory ,committee shall be the ,director of the ,intercenter ,institute for ,rare ,diseases and ,conditions4 7#e7 ,terms 7,a7 ,members 7i7 ,in general ,the term of a member of the ,advisory ,committee appointed pursuant to paragraph 7#a77;,a7 shall be #d years1 except that any member appointed to fill a vacancy in an unexpired term shall be appointed for the remainder of that term4 7ii7 ,continued service ,a member appointed pursuant to paragraph 7#a77;,a7 may continue serving as a member of the ,advisory ,committee for up to #ahj days after the expiration of that member's term if a successor has not been appointed4 7,b7 ,reappointment ,a member of the ,advisory ,committee who has been appointed pursuant to paragraph 7#a77;,a7 for a term of #d years may not be reappointed to serve as a member of the ,advisory ,committee before the date that is #b years after the date of expiration of that member's term4 7e7 ,quorum ,a majority of the appointed members of the ,advisory ,committee shall constitute a quorum for the conduct of business4 4 #e4 ,grants and contracts for development of drugs for rare diseases and conditions 7a7 ,authority of ,secretary ,section #e7;a7 of the ,orphan ,drug ,act 7#ba ,u4,s4,c4 #cfj;ee7a77 is amended-- 7#a7 in paragraph 7#b71 by striking and at the end2 and 7#b7 by inserting before the period at the end 1 and 7#d7 developing practices pertaining to the chemistry1 manufacturing1 regulatory approval of1 and controls of individualized therapies or therapies to treat very small populations4 7b7 ,,altitude ,program ,in supporting grants and contracts under section #e7;a77#d7 of the ,orphan ,drug ,act1 as added by subsection 7a71 the ,secretary of ,health and ,human ,services shall consult with the ,director of the ,intercenter ,institute on ,rare ,diseases and ,conditions regarding the ,accelerating ,lifesavings ,therapies in ,treating ,ultra-rare ,disease ,entities ,program established under section #ebi;,b7b7 of the ,federal ,food1 ,drug1 and ,cosmetic ,act1 as added by section #c7;c7 of this ,act1 to-- 7#a7 identify the regulatory science and related challenges and needs associated with developing individualized therapies or therapies to treat very small patient populations2 and 7#b7 support research to address such challenges4