DOCSLIB.ORG

Federal Register/Vol. 82, No. 214/Tuesday

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Federal Register/Vol. 82, No. 214/Tuesday Federal Register / Vol. 82, No. 214 / Tuesday, November 7, 2017 / Rules and Regulations 51567 range of age and educational levels and lead to prophylactic screening, SUPPLEMENTARY INFORMATION: have no prior experience with the test confirmatory procedures, or treatments I. Statutory Background or its manufacturer. These factors shall that may incur morbidity or mortality to be well defined in the inclusion and the patient; Section 510(k) of the Federal Food, exclusion criteria. (iii) Assessing the presence of genetic Drug, and Cosmetic Act (the FD&C Act) (ii) All sources of bias must be variants that impact the metabolism, (21 U.S.C. 360(k)) and the implementing predefined and accounted for in the exposure, response, risk of adverse regulations, 21 CFR part 807 subpart E, study results with regard to both events, dosing, or mechanisms of require persons who intend to market a responders and non-responders. prescription or over-the-counter device to submit and obtain FDA (iii) The testing must follow a format medications; or clearance of a premarket notification where users have limited time to (iv) Assessing the presence of (510(k)) containing information that complete the studies (such as an onsite deterministic autosomal dominant allows FDA to determine whether the survey format and a one-time visit with variants. new device is ‘‘substantially equivalent’’ a cap on the maximum amount of time within the meaning of section 513(i) of Dated: November 1, 2017. that a participant has to complete the the FD&C Act (21 U.S.C. 360c(i)) to a tests). Lauren Silvis, legally marketed device that does not (iv) Users must be randomly assigned Chief of Staff. require premarket approval. to study arms. Test reports in the user [FR Doc. 2017–24159 Filed 11–6–17; 8:45 am] On December 13, 2016, the 21st comprehension study given to users BILLING CODE 4164–01–P Century Cures Act (Pub. L. 114–255) must define the target condition being (Cures Act) was signed into law. Section tested and related symptoms, explain 3054 of the Cures Act amended section the intended use and limitations of the DEPARTMENT OF HEALTH AND 510(m) of the FD&C Act. As amended, test, explain the relevant ethnicities in HUMAN SERVICES section 510(m)(2) provides that, 1 regard to the variant tested, explain calendar day after the date of Food and Drug Administration genetic health risks and relevance to the publication of the final list under user’s ethnicity, and assess participants’ paragraph (1)(B), FDA may exempt a 21 CFR Part 866 ability to understand the following class II device from the requirement to comprehension concepts: The test’s [Docket No. FDA–2015–N–3455] submit a report under section 510(k) of limitations, purpose, appropriate action, the FD&C Act, upon its own initiative or test results, and other factors that may Medical Devices; Exemption From a petition of an interested person, if have an impact on the test results. Premarket Notification; Class II FDA determines that a 510(k) is not (v) Study participants must be Devices; Autosomal Recessive Carrier necessary to provide reasonable untrained, be naı¨ve to the test subject of Screening Gene Mutation Detection assurance of the safety and effectiveness the study, and be provided the labeling System of the device. This section requires FDA prior to the start of the user to publish in the Federal Register a AGENCY: Food and Drug Administration, comprehension study. HHS. notice of intent to exempt a device, or (vi) The user comprehension study of the petition, and to provide a 60- must meet the predefined primary ACTION: Final order. calendar-day comment period. Within endpoint criteria, including a minimum SUMMARY: The Food and Drug 120 days of publication of such notice, of a 90 percent or greater overall Administration (FDA or Agency) is FDA must publish an order in the comprehension rate (i.e., selection of the publishing an order to exempt Federal Register that sets forth its final correct answer) for each comprehension autosomal recessive carrier screening determination regarding the exemption concept. Other acceptance criteria may gene mutation detection systems from of the device that was the subject of the be acceptable depending on the concept the premarket notification requirements, notice. If FDA fails to respond to a being tested. Meeting or exceeding this subject to certain limitations. This petition under this section within 180 overall comprehension rate exemption from 510(k), subject to days of receiving it, the petition shall be demonstrates that the materials certain limitations, is immediately in deemed granted. presented to the user are adequate for effect for autosomal recessive carrier II. Criteria for Exemption over-the-counter use. screening gene mutation detection There are a number of factors FDA (vii) The analysis of the user systems. This exemption will decrease may consider to determine whether a comprehension results must include regulatory burdens on the medical 510(k) is necessary to provide results regarding reports that are device industry and will eliminate reasonable assurance of the safety and provided for each gene/variant/ethnicity private costs and expenditures required effectiveness of a class II device. These tested, statistical methods used to to comply with certain Federal factors are discussed in the January 21, analyze all data sets, and completion regulations. FDA is also amending the 1998, Federal Register notice (63 FR rate, non-responder rate, and reasons for codified language for the autosomal 3142) and subsequently in the guidance nonresponse/data exclusion. A recessive carrier screening gene the Agency issued on February 19, 1998, summary table of comprehension rates mutation detection system devices entitled ‘‘Procedures for Class II Device regarding comprehension concepts (e.g., classification regulation to reflect this Exemptions from Premarket purpose of test, test results, test final determination. limitations, ethnicity relevance for the Notification, Guidance for Industry and test results, etc.) for each study report DATES: This order is effective November CDRH Staff’’ (referred to herein as the must be included. 7, 2017. Class II 510(k) Exemption Guidance) (4) The intended use of the device FOR FURTHER INFORMATION CONTACT: (Ref. 1). must not include the following Steven Tjoe, Center for Devices and III. Device Description indications for use: Radiological Health, Food and Drug (i) Prenatal testing; Administration, 10903 New Hampshire On February 19, 2015, FDA (ii) Determining predisposition for Ave., Bldg. 66, Rm. 4550, Silver Spring, completed its review of a De Novo cancer where the result of the test may MD 20993–0002, 301–796–5866. request for classification of the 23andMe VerDate Sep<11>2014 15:04 Nov 06, 2017 Jkt 244001 PO 00000 Frm 00019 Fmt 4700 Sfmt 4700 E:\FR\FM\07NOR1.SGM 07NOR1 nshattuck on DSK9F9SC42PROD with RULES 51568 Federal Register / Vol. 82, No. 214 / Tuesday, November 7, 2017 / Rules and Regulations Personal Genome Service (PGS) Carrier IV. Comments on the Proposed based upon scientific and clinical Screening Test for Bloom syndrome. Exemption and FDA Response literature as to the autosomal recessive FDA classified the 23andMe PGS Carrier In response to the October 2015 notice nature of a particular disease or Screening Test for Bloom syndrome, announcing FDA’s intent to exempt condition, we are not providing a and substantially equivalent devices of autosomal recessive carrier screening revised list in this final order. (Comment 2) One commenter this generic type, into class II (special gene mutation detection system devices requested clarification that § 866.5940 controls) under the generic name from premarket notification applies to OTC carrier detection devices ‘‘Autosomal recessive carrier screening requirements, FDA received for the determination of carrier status by gene mutation detection system.’’ This submissions from three commenters—a detection of clinically relevant gene type of device is a qualitative in vitro device industry manufacturer, a mutations associated with cystic molecular diagnostic system used for professional organization, and a health fibrosis. genotyping of clinically relevant care organization—supporting an variants in genomic DNA isolated from (Response) In the October 2015 exemption from premarket notification notice, FDA stated ‘‘[a] gene mutation human specimens intended for for this type of device. prescription use or over-the-counter detection system indicated for the To make it easier to identify determination of carrier status by (OTC) use. The device is intended for comments and our responses, the word autosomal recessive disease carrier detection of clinically relevant gene ‘‘Comment’’ and a comment number mutations associated with Cystic screening in adults of reproductive age. appear in parentheses before each The device is not intended for copy Fibrosis is not 510(k)-exempt since it is comment’s description, and the word a class II device subject to premarket number variation, cytogenetic, or ‘‘Response’’ in parentheses precedes biochemical testing. notification and special controls under each response. Similar comments are 21 CFR 866.5900—Cystic fibrosis FDA believes that De Novo grouped together under the same transmembrane conductance regulator classification will enhance patients’ number. Specific issues raised by the (CFTR) gene mutation detection access to beneficial innovation, in part comments and the Agency’s responses system.’’ Similarly, in the final order by reducing regulatory burdens. When follow. announcing the classification of an FDA classifies a device into class I or II (Comment 1) Two commenters autosomal recessive carrier screening via the De Novo process, the device can requested that FDA clarify that the list gene mutation detection system into serve as a predicate for determining of autosomal recessive carrier diseases class II (80 FR 65626, October 27, 2015), substantial equivalence for future included in the October 2015 notice is FDA stated ‘‘A gene mutation detection devices within that type (see 21 U.S.C. not exhaustive or expand the list of system indicated for the determination 360c(f)(2)(B)(i)).
Load more

Recommended publications
  • EFFECTIVE NEBRASKA DEPARTMENT of 01/01/2017 HEALTH and HUMAN SERVICES 173 NAC 1 I TITLE 173 COMMUNICABLE DISEASES CHAPTER 1
    EFFECTIVE NEBRASKA DEPARTMENT OF 01/01/2017 HEALTH AND HUMAN SERVICES 173 NAC 1 TITLE 173 COMMUNICABLE DISEASES CHAPTER 1 REPORTING AND CONTROL OF COMMUNICABLE DISEASES TABLE OF CONTENTS SECTION SUBJECT PAGE 1-001 SCOPE AND AUTHORITY 1 1-002 DEFINITIONS 1 1-003 WHO MUST REPORT 2 1-003.01 Healthcare Providers (Physicians and Hospitals) 2 1-003.01A Reporting by PA’s and APRN’s 2 1-003.01B Reporting by Laboratories in lieu of Physicians 3 1-003.01C Reporting by Healthcare Facilities in lieu of Physicians for 3 Healthcare Associated Infections (HAIs) 1-003.02 Laboratories 3 1-003.02A Electronic Ordering of Laboratory Tests 3 1-004 REPORTABLE DISEASES, POISONINGS, AND ORGANISMS: 3 LISTS AND FREQUENCY OF REPORTS 1-004.01 Immediate Reports 4 1-004.01A List of Diseases, Poisonings, and Organisms 4 1-004.01B Clusters, Outbreaks, or Unusual Events, Including Possible 5 Bioterroristic Attacks 1-004.02 Reports Within Seven Days – List of Reportable Diseases, 5 Poisonings, and Organisms 1-004.03 Reporting of Antimicrobial Susceptibility 8 1-004.04 New or Emerging Diseases and Other Syndromes and Exposures – 8 Reporting and Submissions 1-004.04A Criteria 8 1-004.04B Surveillance Mechanism 8 1-004.05 Sexually Transmitted Diseases 9 1-004.06 Healthcare Associated Infections 9 1-005 METHODS OF REPORTING 9 1-005.01 Health Care Providers 9 1-005.01A Immediate Reports of Diseases, Poisonings, and Organisms 9 1-005.01B Immediate Reports of Clusters, Outbreaks, or Unusual Events, 9 Including Possible Bioterroristic Attacks i EFFECTIVE NEBRASKA DEPARTMENT OF
    [Show full text]
  • C Lif I I I I C Lif I I I I California Immunization Requirements
    ClifCaliforn ia IiiImmunization Requirements for School & Child Care Entry Jennifer Sterling San Diego Immunization Partnership www.sdiz.org CtCounty of San Diego, HlthHealth and Human SiServices Agency Immunization Partnership Training Objectives At the end of this training, you will be able to: • List the immunization requirements for child care entry. • Identify acceptable forms of personal immunization records. • Correctly complete the California School Immunization Record (CSIR)/Blue Card. • Create a management system for conditional entrants and exemptions. Why Do We Need Immunizations? • Protect children and families • Prevent illnesses which can cause death and disability • Protect the health of others in the community • Good Public Health Practice Spread of Disease read of Disease –– a a cough, cough, sneezesneeze, or handshake away, or handshake away Vaccine Preventable Diseases in San Diego County Pertussis (Whooping Cough) >604 cases so far in 2010! The worst epidemic in 55 years. • Adults spreading sickness to babies. Nine babies under 3 months of age have died in California this year. • To preven t the sprea d o f Per tuss is, ge t the TDAP vacci ne! Chickenpox • 2009: 69 cases in 10 school districts • 2010: 10 cases in 2 school districts Measles • 2010: Three measles cases in Carlsbad, nine children quarantined • Measles outbreak in San Diego during January and February 2008‐first measles outbreak since 1991! ‐12 cases in 2008 ‐71 peopl e quarantine d Polio is still epidemic in 4 countries! Pertussis – Nearly 50% of the time, infants are infected by their parents Prevention of Disease – SimpleSimple!! Immunizations help prevent the spread of communicable diseases. Cover mouth (w/arm) when coughing or sneezing.
    [Show full text]
  • European Conference on Rare Diseases
    EUROPEAN CONFERENCE ON RARE DISEASES Luxembourg 21-22 June 2005 EUROPEAN CONFERENCE ON RARE DISEASES Copyright 2005 © Eurordis For more information: www.eurordis.org Webcast of the conference and abstracts: www.rare-luxembourg2005.org TABLE OF CONTENT_3 ------------------------------------------------- ACKNOWLEDGEMENTS AND CREDITS A specialised clinic for Rare Diseases : the RD TABLE OF CONTENTS Outpatient’s Clinic (RDOC) in Italy …………… 48 ------------------------------------------------- ------------------------------------------------- 4 / RARE, BUT EXISTING The organisers particularly wish to thank ACKNOWLEDGEMENTS AND CREDITS 4.1 No code, no name, no existence …………… 49 ------------------------------------------------- the following persons/organisations/companies 4.2 Why do we need to code rare diseases? … 50 PROGRAMME COMMITTEE for their role : ------------------------------------------------- Members of the Programme Committee ……… 6 5 / RESEARCH AND CARE Conference Programme …………………………… 7 …… HER ROYAL HIGHNESS THE GRAND DUCHESS OF LUXEMBOURG Key features of the conference …………………… 12 5.1 Research for Rare Diseases in the EU 54 • Participants ……………………………………… 12 5.2 Fighting the fragmentation of research …… 55 A multi-disciplinary approach ………………… 55 THE EUROPEAN COMMISSION Funding of the conference ……………………… 14 Transfer of academic research towards • ------------------------------------------------- industrial development ………………………… 60 THE GOVERNEMENT OF LUXEMBOURG Speakers ……………………………………………… 16 Strengthening cooperation between academia
    [Show full text]
  • Diseases Transmitted Through the Food Supply
    DEPARTMENT OF HEALTH AND HUMAN SERVICES Centers for Disease Control and Prevention Diseases Transmitted through the Food Supply AGENCY: Centers for Disease Control and Prevention (CDC), Department of Health and Human ­ Services (HHS). ­ ACTION: Notice of annual update of list of infectious and communicable diseases that are ­ transmitted through handling the food supply and the methods by which such diseases are ­ transmitted. ­ SUMMARY: Section 103 (d) of the Americans with Disabilities Act of 1990, Public Law 101–336, ­ requires the Secretary to publish a list of infectious and communicable diseases that are ­ transmitted through handling the food supply and to review and update the list annually. The ­ Centers for Disease Control and Prevention (CDC) published a final list on August 16, 1991 (56 ­ FR40897) and updates on September 8, 1992 (57 FR 40917); January 13, 1994 (59 FR 1949); ­ August 15, 1996 (61 FR 42426); September 22, 1997 (62 FR 49518–9); September 15, 1998 (63 ­ FR 49359); September 21, 1999 (64 FR 51127); September 27, 2000 (65 FR 58088); September ­ 10, 2001 (66 FR 47030); September 27, 2002 (67 FR 61109); September 26, 2006 (71 FR 56152); ­ November 17, 2008 (73 FR 67871); and November 29, 2009 (74 FR 61151). The final list has ­ been reviewed in light of new information and has been revised as set forth below. ­ DATES: Effective Date: January 31, 2014. ­ FOR FURTHER INFORMATION CONTACT: ­ Dr. Art Liang, Division of Foodborne Waterborne and Environment Diseases, National Center for ­ Emerging and Zoonotic Infectious Diseases, Centers for Disease Control and Prevention (CDC), ­ 1600 Clifton Road, NE., Mailstop G–24, Atlanta, Georgia 30333.
    [Show full text]
  • Microtubule Regulation in Cystic Fibrosis Pathophysiology
    MICROTUBULE REGULATION IN CYSTIC FIBROSIS PATHOPHYSIOLOGY By: SHARON MARIE RYMUT Submitted in partial fulfillment of the requirements For the degree of Doctor of Philosophy Dissertation Advisor: Dr. Thomas J Kelley Department of Pharmacology CASE WESTERN RESERVE UNIVERSITY August 2015 CASE WESTERN RESERVE UNIVERSITY SCHOOL OF GRADUATE STUDIES We hereby approve the thesis/ dissertation of Sharon Marie Rymut candidate for the Doctor of Philosophy degree* Dissertation Advisor: Thomas J Kelley Committee Chair: Paul N MacDonald Committee Member: Ruth E Siegel Committee Member: Craig A Hodges Committee Member: Danny Manor Committee Member: Rebecca J Darrah Date of Defense: April 29, 2015 * We also certify that written approval has been obtained for any proprietary material contained therein. ii Dedication There are five chapters in this dissertation. To Mom, Dad, Joe, Marie and Susan, I dedicate one chapter to each of you. You can fight about which chapter you want later. iii Table of Contents Table of Contents................................................................................................................iv List of Tables .................................................................................................................... vii List of Figures .................................................................................................................. viii Acknowledgements ............................................................................................................. x List of Abbreviations .......................................................................................................
    [Show full text]
  • LIST of OCCUPATIONAL DISEASES (Revised 2010)
    LIST OF OCCUPATIONAL DISEASES (revised 2010) Identification and recognition of occupational diseases: Criteria for incorporating diseases in the ILO list of occupational diseases Occupational Safety and Health Series, No. 74 List of occupational diseases (revised 2010) Identification and recognition of occupational diseases: Criteria for incorporating diseases in the ILO list of occupational diseases INTERNATIONAL LABOUR OFFICE • GENEVA Copyright © International Labour Organization 2010 First published 2010 Publications of the International Labour Office enjoy copyright under Protocol 2 of the Universal Copyright Convention. Nevertheless, short excerpts from them may be reproduced without authorization, on condition that the source is indicated. For rights of reproduction or translation, application should be made to ILO Publications (Rights and Permissions), International Labour Office, CH-1211 Geneva 22, Switzerland, or by email: pubdroit@ ilo.org. The International Labour Office welcomes such applications. Libraries, institutions and other users registered with reproduction rights organizations may make copies in accordance with the licences issued to them for this purpose. Visit www.ifrro.org to find the reproduction rights organization in your country. ILO List of occupational diseases (revised 2010). Identification and recognition of occupational diseases: Criteria for incorporating diseases in the ILO list of occupational diseases Geneva, International Labour Office, 2010 (Occupational Safety and Health Series, No. 74) occupational disease / definition. 13.04.3 ISBN 978-92-2-123795-2 ISSN 0078-3129 Also available in French: Liste des maladies professionnelles (révisée en 2010): Identification et reconnaissance des maladies professionnelles: critères pour incorporer des maladies dans la liste des maladies professionnelles de l’OIT (ISBN 978-92-2-223795-1, ISSN 0250-412x), Geneva, 2010, and in Spanish: Lista de enfermedades profesionales (revisada en 2010).
    [Show full text]
  • ICD-10 International Statistical Classification of Diseases and Related Health Problems
    ICD-10 International Statistical Classification of Diseases and Related Health Problems 10th Revision Volume 2 Instruction manual 2010 Edition WHO Library Cataloguing-in-Publication Data International statistical classification of diseases and related health problems. - 10th revision, edition 2010. 3 v. Contents: v. 1. Tabular list – v. 2. Instruction manual – v. 3. Alphabetical index. 1.Diseases - classification. 2.Classification. 3.Manuals. I.World Health Organization. II.ICD-10. ISBN 978 92 4 154834 2 (NLM classification: WB 15) © World Health Organization 2011 All rights reserved. Publications of the World Health Organization are available on the WHO web site (www.who.int) or can be purchased from WHO Press, World Health Organization, 20 Avenue Appia, 1211 Geneva 27, Switzerland (tel.: +41 22 791 3264; fax: +41 22 791 4857; e-mail: [email protected]). Requests for permission to reproduce or translate WHO publications – whether for sale or for noncommercial distribution – should be addressed to WHO Press through the WHO web site (http://www.who.int/about/licensing/copyright_form). The designations employed and the presentation of the material in this publication do not imply the expression of any opinion whatsoever on the part of the World Health Organization concerning the legal status of any country, territory, city or area or of its authorities, or concerning the delimitation of its frontiers or boundaries. Dotted lines on maps represent approximate border lines for which there may not yet be full agreement. The mention of specific companies or of certain manufacturers’ products does not imply that they are endorsed or recommended by the World Health Organization in preference to others of a similar nature that are not mentioned.
    [Show full text]
  • FAQ REGARDING DISEASE REPORTING in MONTANA | Rev
    Disease Reporting in Montana: Frequently Asked Questions Title 50 Section 1-202 of the Montana Code Annotated (MCA) outlines the general powers and duties of the Montana Department of Public Health & Human Services (DPHHS). The three primary duties that serve as the foundation for disease reporting in Montana state that DPHHS shall: • Study conditions affecting the citizens of the state by making use of birth, death, and sickness records; • Make investigations, disseminate information, and make recommendations for control of diseases and improvement of public health to persons, groups, or the public; and • Adopt and enforce rules regarding the reporting and control of communicable diseases. In order to meet these obligations, DPHHS works closely with local health jurisdictions to collect and analyze disease reports. Although anyone may report a case of communicable disease, such reports are submitted primarily by health care providers and laboratories. The Administrative Rules of Montana (ARM), Title 37, Chapter 114, Communicable Disease Control, outline the rules for communicable disease control, including disease reporting. Communicable disease surveillance is defined as the ongoing collection, analysis, interpretation, and dissemination of disease data. Accurate and timely disease reporting is the foundation of an effective surveillance program, which is key to applying effective public health interventions to mitigate the impact of disease. What diseases are reportable? A list of reportable diseases is maintained in ARM 37.114.203. The list continues to evolve and is consistent with the Council of State and Territorial Epidemiologists (CSTE) list of Nationally Notifiable Diseases maintained by the Centers for Disease Control and Prevention (CDC). In addition to the named conditions on the list, any occurrence of a case/cases of communicable disease in the 20th edition of the Control of Communicable Diseases Manual with a frequency in excess of normal expectancy or any unusual incident of unexplained illness or death in a human or animal should be reported.
    [Show full text]
  • House of Representatives
    House of Representatives General Assembly File No. 45 February Session, 2004 House Bill No. 5201 House of Representatives, March 15, 2004 The Committee on Insurance and Real Estate reported through REP. OREFICE of the 37th Dist., Chairperson of the Committee on the part of the House, that the bill ought to pass. AN ACT CONCERNING NEWBORN SCREENING AND MEDICALLY NECESSARY NUTRITIONAL FORMULA FOR CYSTIC FIBROSIS. Be it enacted by the Senate and House of Representatives in General Assembly convened: 1 Section 1. Section 38a-492c of the general statutes is repealed and the 2 following is substituted in lieu thereof (Effective October 1, 2004): 3 (a) For purposes of this section: 4 (1) "Inherited metabolic disease" means a disease for which 5 newborn screening is required under section 19a-55, as amended by 6 this act. 7 (2) "Low protein modified food product" means a product 8 formulated to have less than one gram of protein per serving and 9 intended for the dietary treatment of an inherited metabolic disease 10 under the direction of a physician. 11 (3) "Amino acid modified preparation" means a product intended HB5201 / File No. 45 1 HB5201 File No. 45 12 for the dietary treatment of an inherited metabolic disease under the 13 direction of a physician. 14 (4) "Specialized formula" means a nutritional formula for children 15 up to age three that is exempt from the general requirements for 16 nutritional labeling under the statutory and regulatory guidelines of 17 the federal Food and Drug Administration and is intended for use 18 solely under medical supervision in the dietary management of 19 specific diseases.
    [Show full text]
  • Estimating the Financial Impact of Gene Therapy*
    medRxiv preprint doi: https://doi.org/10.1101/2020.10.27.20220871; this version posted October 31, 2020. The copyright holder for this preprint (which was not certified by peer review) is the author/funder, who has granted medRxiv a license to display the preprint in perpetuity. It is made available under a CC-BY 4.0 International license . Estimating the Financial Impact of Gene Therapy∗ Chi Heem Wong†, Dexin Li‡, Nina Wang‡, Jonathan Gruber§, Rena Conti¶, Andrew W. Lo‖ This Revision: October 15, 2020 Abstract We assess the potential financial impact of future gene therapies by identifying the 109 late-stage gene therapy clinical trials currently underway, estimating the prevalence and incidence of their corresponding diseases, developing novel mathematical models of the increase in quality-adjusted life years for each approved gene therapy, and sim- ulating the launch prices and the expected spending of these therapies over a 15-year time horizon. The results of our simulation suggest that an expected total of 1.09 million patients will be treated by gene therapy from January 2020 to December 2034. The expected peak annual spending on these therapies is $25.3 billion, and the total spending from January 2020 to December 2034 is $306 billion. We decompose their annual estimated spending by treated age group as a proxy for U.S. insurance type, and consider the tradeoffs of various methods of payment for these therapies to ensure patient access to their expected benefits. Keywords: Gene Therapy; Drug Pricing; Cost Effectiveness; Health Technology Assessment JEL Codes: I11, I13, I18 ∗We thank Sarah Antiles and Nora Yang for assisting with the preparation of data.
    [Show full text]
  • Optional Ashkenazi Panel Brochure
    בס’’ד Fulfilling Our Responsibility To The Next Generation A VITAL PROGRAM THAT SAVES LIVES! The Dor Yeshorim mission: For over 30 years, Dor Yeshorim has successfully prevented the occurrence of genetic diseases in our NOW AVAILABLE: families. Since 1983, Dor Yeshorim with Hashem’s help NEW PANEL OF TESTS has successfully prevented genetic diseases from affecting our Jewish Community by conducting mass screenings in high schools, colleges, universities, FOR SEVEN ADDITIONAL yeshivas and seminaries worldwide- throughout the United States, Israel, Canada, Europe, and countless other orthodox Jewish communities. DEVASTATING AND The Dor Yeshorim genetic screening program was established to provide protection from Jewish genetic diseases, while safeguarding individuals from the potentiallY FatAL psychological stigma associated with knowing their carrier status. DISEASES To date, approximately 375,000 individual genetic screening tests have been performed, and nearly 2,000 potential couples have been spared the List of Diseases: agony of giving birth to a child or children with a 1. Bardet–Biedl Syndrome Type 2 (BBS2) devastating or fatal genetic disease. The birth of a sick child not only impacts the parents of this child 2. Nemaline Myopathy (NM) but also the siblings, grandpare nts and the extended 3. Dihyrolipoamide Dehydrogenase Deficiency (DLDD) family. B’H Dor Yeshorim has been able to spare over 4. Usher Syndrome Type 1 (USH1) 2,000 families the hardships associated with having such a child. 5. Joubert Syndrome (JBTS) DY benefits from
    [Show full text]
  • Summary of Notifiable Diseases — United States, 2010
    Morbidity and Mortality Weekly Report Weekly / Vol. 59 / No. 53 June 1, 2012 Summary of Notifiable Diseases — United States, 2010 U.S. Department of Health and Human Services Centers for Disease Control and Prevention Morbidity and Mortality Weekly Report CONTENTS Preface .......................................................................................................................2 TABLE 5. Reported cases and incidence* of notifiable diseases,† by Background .............................................................................................................2 race — United States, 2010 .......................................................................... 43 Infectious Diseases Designated as Notifiable at the National Level TABLE 6. Reported cases and incidence* of notifiable diseases,† by during 2010* .........................................................................................................3 ethnicity — United States, 2010 ................................................................. 45 Data Sources ...........................................................................................................4 PART 2: Graphs and Maps for Selected Notifiable Diseases Interpreting Data ...................................................................................................4 in the United States, 2010 ............................................................................. 47 Transition in NNDSS Data Collection and Reporting ................................5 PART 3: Historical Summaries
    [Show full text]