H.F. 287 Section 1. FINDINGS. 1 1. A rare disease, sometimes called an orphan disease, 2 is defined as a disease that affects fewer than two hundred 3 thousand people in the United States. 4 2. There are seven thousand known rare diseases affecting 5 approximately twenty-five to thirty million adults and children 6 in the United States. 7 3. While the exact cause for many rare diseases remains 8 unknown, many rare diseases are genetic in origin and can be 9 linked to mutations in a single gene or in multiple genes which 10 can be passed down from generation to generation. 11 4. People with rare diseases face many challenges, 12 including delays in obtaining a diagnosis or misdiagnosis, 13 shortages of medical specialists who can provide treatment, and 14 lack of affordable access to therapies and medication used to 15 treat rare diseases. 16 5. A state rare disease advisory council composed of 17 qualified professionals and persons living with rare diseases 18 could educate medical professionals, government agencies, 19 legislators, and the public about rare diseases as an important 20 public health issue and encourage and secure funding for 21 research for the development of new treatments for rare 22 diseases. 23 Sec. 2. NEW SECTION . 135.133 Rare disease advisory council 24 —— membership —— duties. 25 1. A rare disease advisory council is created within the 26 department for the purpose of advising the department and 27 other state agencies, the general assembly, and the public on 28 research, diagnosis, and treatment efforts related to rare 29 diseases in this state. 30 2. The council shall consist of the following members: 31 a. The director of health and human services, or the 32 director’s designee. 33 b. The Medicaid director, or the director’s designee. 34 c. The commissioner of insurance, or the commissioner’s 35 -1- LSB 1927YH (3) 90 pf/rh 1/ 6

H.F. 287 designee. 1 d. The chairperson of the congenital and inherited disorders 2 advisory committee, or the chairperson’s designee. 3 e. All of the following appointed by the director: 4 (1) A representative from an academic research institution 5 in the state that receives grant funding for rare disease 6 research. 7 (2) A geneticist licensed and practicing in the state. 8 (3) A registered nurse or an advanced registered nurse 9 practitioner licensed and practicing in the state with 10 experience in treating rare diseases. 11 (4) A physician practicing in the state with experience in 12 treating rare diseases. 13 (5) A hospital administrator, or the administrator’s 14 designee, from a hospital in the state that provides care to 15 persons diagnosed with a rare disease. 16 (6) At least two persons diagnosed with a rare disease. 17 (7) At least two caregivers of persons diagnosed with a rare 18 disease. 19 (8) A representative of a rare disease patient organization 20 that operates in the state. 21 (9) A pharmacist with experience dispensing drugs used to 22 treat rare diseases. 23 (10) A representative of the biopharmaceutical industry. 24 (11) A representative of an insurance company. 25 (12) A member of the scientific community who is engaged 26 in rare disease research including but not limited to a 27 medical researcher with experience conducting research in rare 28 diseases. 29 3. a. The members selected by the director shall serve 30 two-year terms. A vacancy on the council shall be filled in 31 the same manner as the original appointment. The council shall 32 elect a chairperson every two years. A majority of the members 33 of the council constitute a quorum and a quorum is required for 34 official action of the council. 35 -2- LSB 1927YH (3) 90 pf/rh 2/ 6

H.F. 287 b. The council shall meet on a monthly basis for the 1 first six months following creation of the council, and on a 2 quarterly basis thereafter. 3 c. The council members appointed by the director shall be 4 reimbursed for their actual and necessary expenses incurred 5 while engaged in the performance of official duties. Members 6 may also be eligible for compensation as provided in section 7 7E.6. 8 4. The council shall do all of the following: 9 a. Convene public hearings, make inquiries, and solicit 10 comments from the general public to assist the council in 11 surveying the needs of rare disease patients, caregivers, and 12 providers in the state. 13 b. Consult with experts on rare diseases to develop policy 14 recommendations to improve patient access to and the quality of 15 rare disease specialists, affordable and comprehensive health 16 care coverage, relevant diagnostics, timely treatment, and 17 other services. 18 c. Research and make recommendations to state agencies and 19 insurers that provide services and coverage to persons with a 20 rare disease on the impact of coverage, cost-sharing, tiering, 21 or other utilization management procedures on the provision of 22 treatment and services. 23 d. Research and identify priorities related to treatments 24 and services provided to persons with a rare disease in 25 the state and develop policy recommendations that include 26 safeguards against discrimination for these populations, 27 including in disaster and public health emergency-related 28 planning. 29 e. Evaluate and make recommendations to improve the newborn 30 screening program. 31 f. Evaluate and make recommendations to improve Medicaid 32 coverage of treatment and medications for persons with a rare 33 disease. 34 g. Publish a list of existing, publicly accessible resources 35 -3- LSB 1927YH (3) 90 pf/rh 3/ 6

H.F. 287 on research, diagnosis, treatment, and education relating to 1 rare diseases in the state on the department’s internet site. 2 h. Identify areas of unmet needs for research and to inform 3 the work of the council. 4 i. Establish resources for academic institutions, state 5 agencies, health care professionals, and other entities to 6 provide a basis for training on rare diseases in the state. 7 j. Identify and distribute educational resources for health 8 care providers to foster recognition and optimize treatment of 9 rare diseases in the state. 10 k. Research and identify best practices to reduce health 11 disparities and achieve health equity in research, diagnosis, 12 and treatment of rare diseases in the state. 13 l. Establish best practices and protocols to use during a 14 state of emergency to aid persons with a rare disease. 15 m. Coordinate with rare disease and related entities, 16 community-based organizations, and other public and private 17 organizations in performing its duties to ensure greater 18 cooperation between this state, other states, and the federal 19 government regarding the research, diagnosis, and treatment of 20 rare diseases. 21 5. The council may solicit funds and accept donations, 22 gifts, and bequests approved by the council in accordance with 23 the duties of the council. 24 6. The council shall submit an annual report to the governor 25 and the general assembly, and publish the annual report on the 26 department’s internet site, that includes all of the following: 27 a. A summary of the activities and progress of the council 28 in carrying out the council’s duties in the previous year. 29 b. An update on the status of funding sought and received, 30 the use of the funds, and any remaining balances. 31 c. Provide recommendations to address the needs of people 32 living with a rare disease in the state. 33 EXPLANATION 34 The inclusion of this explanation does not constitute agreement with 35 -4- LSB 1927YH (3) 90 pf/rh 4/ 6

H.F. 287 the explanation’s substance by the members of the general assembly. 1 This bill creates a rare disease advisory council within the 2 department of health and human services (HHS) for the purpose 3 of advising HHS and other state agencies, the general assembly, 4 and the public on research, diagnosis, and treatment efforts 5 related to rare diseases in the state. The bill specifies the 6 members of the council and the procedures for the functioning 7 of the council. 8 The bill specifies the duties of the council including: 9 convening public hearings, making inquiries, and soliciting 10 comments from the general public to assist the council in 11 surveying the needs of persons with a rare disease, caregivers, 12 and providers in the state; consulting with experts on rare 13 diseases to develop policy recommendations to improve patient 14 access to and the quality of rare disease specialists, 15 affordable and comprehensive health care coverage, relevant 16 diagnostics, timely treatment, and other services; researching 17 and making recommendations to state agencies and insurers that 18 provide services and coverage to persons with a rare disease 19 on the impact of coverage, cost-sharing, tiering, or other 20 utilization management procedures on the provision of treatment 21 and services; researching and identifying priorities related to 22 treatments and services provided to persons with a rare disease 23 in the state and develop policy recommendations that include 24 safeguards against discrimination for these populations, 25 including in disaster and public health emergency-related 26 planning; evaluating and making recommendations to improve 27 the newborn screening program; evaluating and making 28 recommendations to improve Medicaid coverage of treatment and 29 medications for persons with a rare disease; publishing a 30 list of existing, publicly accessible resources on research, 31 diagnosis, treatment, and education relating to the rare 32 diseases in the state on the HHS internet site; identifying 33 areas of unmet needs for research and to inform the work of 34 the council; establishing resources for academic institutions, 35 -5- LSB 1927YH (3) 90 pf/rh 5/ 6

H.F. 287 state agencies, health care professionals, and other entities 1 to provide a basis for training on rare diseases in the state; 2 identifying and distributing educational resources for health 3 care providers to foster recognition and optimize treatment 4 of rare diseases in the state; researching and identifying 5 best practices to reduce health disparities and achieve health 6 equity in research, diagnosis, and treatment of rare diseases 7 in the state; establishing best practices and protocols to use 8 during a state of emergency to aid persons with a rare disease; 9 and coordinating with rare disease and related entities, 10 community-based organizations, and other public and private 11 organizations in performing its duties to ensure greater 12 cooperation between this state, other states, and the federal 13 government regarding the research, diagnosis, and treatment of 14 rare diseases. 15 The council may solicit funds and accept donations, gifts, 16 and bequests approved by the council in accordance with the 17 duties of the council. 18 The council shall submit an annual report to the governor and 19 the general assembly, and publish the annual report on the HHS 20 internet site, summarizing its activities for the prior year, 21 updating the status of funding, and providing recommendations 22 to address the needs of persons living with a rare disease in 23 the state. 24 -6- LSB 1927YH (3) 90 pf/rh 6/ 6