Evidence-Based Pharmacovigilance: Clinical and Quantitative Aspects PDF
Preview Evidence-Based Pharmacovigilance: Clinical and Quantitative Aspects
Methods in Pharmacology and Toxicology Andrew Bate Editor Evidence-Based Pharmacovigilance Clinical and Quantitative Aspects M P ET HODS IN HARMACOLOGY A ND T OXICOLOGY SeriesEditor Y.James Kang Department ofPharmacology &Toxicology University ofLouisville, Louisville Kentucky,USA Forfurther volumes: http://www.springer.com/series/7653 Evidence-Based Pharmacovigilance Clinical and Quantitative Aspects Edited by Andrew Bate Epidemiology Group Lead, Analytics, Worldwide Safety and Regulatory, Tadworth, Surrey, UK Division of Translational Medicine, Department of Medicine, New York University, New York, USA Editor AndrewBate EpidemiologyGroupLead,Analytics WorldwideSafetyandRegulatory Tadworth,Surrey,UK DivisionofTranslationalMedicine DepartmentofMedicine NewYorkUniversity NewYork,USA ISSN1557-2153 ISSN1940-6053 (electronic) MethodsinPharmacologyandToxicology ISBN978-1-4939-8816-7 ISBN978-1-4939-8818-1 (eBook) https://doi.org/10.1007/978-1-4939-8818-1 LibraryofCongressControlNumber:2018957661 ©SpringerScience+BusinessMedia,LLC,partofSpringerNature2018 Thisworkissubjecttocopyright.AllrightsarereservedbythePublisher,whetherthewholeorpartofthematerialis concerned,specificallytherightsoftranslation,reprinting,reuseofillustrations,recitation,broadcasting,reproduction onmicrofilmsorinanyotherphysicalway,andtransmissionorinformationstorageandretrieval,electronicadaptation, computersoftware,orbysimilarordissimilarmethodologynowknownorhereafterdeveloped. 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Dedication ToJenny,Agnes,andMary Tomymum Tomydad:towhomIsowishIcouldhavehandedoveracopyofthisbook. v Foreword Noeffectivemedicineiswithouthazard.Furthermore,notallhazardscanbeknownbefore a medicine is marketed. Safety monitoring of medicines in clinical use is therefore a vital activitytoprotectpeoplefromharm. Such a simple summary belies the challenges and complexities of pharmacovigilance, a scientific discipline whose goal is to ensure that for all marketed medicines the benefits outweigh the risks. Pharmacovigilance is a dynamic and constantly evolving field, which is focusedontheoptimalutilizationofdata,tools,andmethodologiestorapidlyidentifyand managerisksandtomonitor theeffectivenessofriskminimization. Importantly, pharmacovigilancehas emerged fromthe early days of crisis management andsuddendrugwithdrawalsasaspecialtywhosegrowingimportanceiswidelyrecognized initsownright.Notificationsystemstoreportsuspectedadversereactionswereputinplace after thethalidomidetragedyofthe1960sinmostcountriesandestablishedthevalueofa direct link between the healthcare professional and the authorities. But maintaining public confidence has demanded more than this: it has demanded a shift in culture from reactive firefighting to proactive, planned characterization of safety, always taking into account a medicine’stherapeuticrole. Technological advances in information transmission, management, and analysis have enabled groundbreaking developments in pharmacovigilance, particularly in detecting sig- nalsofemergingharm.Newdatasourcesandwaystointegratedifferentkindsofdatahave been key, for example methodologies for linking spontaneous case reports with electronic health records to contextualize signals. There is no doubt that careful evaluation and wise useofnewdatatools and methodologieswill continueto bethespringboardfor improve- mentinpharmacovigilance. Pharmacovigilance must also keep pace with increasingly complex medicinal products. Robust pharmacovigilance is increasingly seen as an enabler of innovation rather than a barrier. Novelmedicines which aredeveloped for small target populations require specially designed approaches to best characterize their safety profile and reduce uncertainties. Evaluating the safety of advanced therapies may extend to the process of product adminis- tration itself. Follow-up of patients may need to be lengthy, even lifelong. Unusual or previouslyunknownadverseeffectsmaybeanticipated. Increasing societal expectations have posed special challenges and dilemmas for phar- macovigilance: operating best protection of individual privacy while optimizing data shar- ing; promoting greater transparency of signal evaluation while maintaining commercial confidentiality;increasingspeedofregulatorydecision-makingwhileensuringrobustanaly- sis of all available data. The full impact of involving patients and the public in pharmacov- igilance systems is yet to be realized. These tensions, if well managed, will stimulate productivechange. Thefutureopportunitiesforpharmacovigilanceareexciting.Theadvantagesofharnes- sing mobile technologies and social media, the use of artificial intelligence and machine learning, the potential of pharmacogenetics, and new scientific disciplines such as imple- mentation science—these and others will all influence and shape the future of the field. vii viii Foreword With such opportunities will come responsibilities—social as well as scientific. The same standardsofsafetyprotectionshouldsurelyapplywhereverintheworldamedicineistaken. Withthisbookasacompass,aroadmaptoachievingimprovedpublichealthoutcomesfor allcanbecomeareality. Director of Vigilance and Risk Management of Medicines JuneRaine Medicines and Healthcare Products Regulatory Agency London, UK Preface Background and Introduction Pharmacovigilance is defined by the World Health Organization as “the science and activ- ities relating to the detection, assessment, understanding and prevention of adverse effects or any other drug-related problem” [1]. In the 1960s, in the wake of the thalidomide disaster,itwaspainfullyapparentthattherewasanessentialneedforasystematicapproach to the monitoring of marketed medicinal products, and it was out of this drive for change thatthefieldofpharmacovigilancewasborn. Interest in pharmacovigilance has never been greater than it is now: awareness and attention in pharmacovigilance have spread from being the sole preserve of the safety scientist to a subject of interest to the wider community of healthcare professionals and patientsthemselvesaroundtheworld.Therearemanyissuesanddevelopmentsthatseemto be driving this heightened interest. The use of medicinal products has made such a clear contributiontoincreasedlifeexpectancyandgeneralwell-beingthatbeingsuchacorepart of modern living, there is more discussion and debate about them and their benefits and risks in the lay and social media. This debate has been fueled by several high-profile medicinal product withdrawals and extensive media discussion about them [2]. In a risk- averse society with much focus on healthy living, the perception of preventable harm generates much dialogue. Also, as data associated with, or of relevance to, healthcare is moreextensively collectedand morereadilyaccessible,thishastoincreased researchpossi- bilitieswithinpharmacovigilanceandalsotoomuchresearchactivitytoprogressthescience and discussions about appropriate use of such data and ensuring appropriate privacy of individualdata. Asayoungscienceinthetwentiethcentury,thecorescientificprinciplesthatunderpin thefieldweredeveloped.Initiallyassmallspecializedfield,butwithaverybroadremitand plenty of opportunity for scientific development, much methodological work occurred in silos, for example, statisticians advancing quantitative thinking in pharmacovigilance; computational scientists developing better and more sophisticated approaches for collect- ing, storing, retrieving, and sharing data; and clinicians developing approaches for the diagnosis and prevention of adverse drug reactions. Over time, as other scientific advances (such as the development of the Internet, electronic data capture systems, diagnoses tools, and increasedcomputational power)haveoccurred,theseadvanceshavebeenleveragedto looktoimprovepharmacovigilance.Thefieldhassubsequentlygrownincomplexityasithas matured. Pharmacovigilance is now widely accepted as a broad discipline requiring input from many different fields of work, and it has become increasingly clear that a multidisci- plinaryapproachisessentialandthatscientistscannotfocussolelyononeaspectofpharma- covigilance without a strong grasp of other elements of the field, specifically awareness of both quantitative and clinical aspects. Successful pharmacovigilance requires marrying of bothperspectivesintoanoverallholisticstrategyandthatacceptingsomeamountofnatural tension between these two very different perspectives is a necessity and something to be embracedrather thanresisted. ix x Preface Thisbookaimstoprovideinsightsintothelatestthinkingandcoreconceptsinareasof keymethodologicalendeavorinpharmacovigilance(PV).Avastarrayofmethodsunderpins thescienceofPV,aswestrivetoevermoreeffectivelyprotectpatientsfromharmwhilethey have access to the medicines they need. This book aims to give practitioners who wish to contributeorsimplytobetterunderstandthescienceofpharmacovigilanceanawarenessof keyprogressandchallengesinmethodologicaladvances.Thisbookcoversbothclinicaland quantitative aspects and isintended to be accessible for all with a role to play or interest in PV. The book makes no attempt to provide an exhaustive list of all methodological devel- opmentinorassociatedwithpharmacovigilance,butratherprovidesaselectionofsomeof the key areas of methodological development, whether clinical or quantitative, considered eitherparticularlyimportant,controversial,orareaschangingrapidly. Eachbookchaptertendstohaveaclearquantitativeorclinicalslantandaimstoprovide anoverviewofmethodologicalinsightswithinaspecifictopicbutalsoprovideaperspective onhowtheareaisanticipatedtodevelopinthefuture.Quantitativechaptersfocusmoreon statistical and epidemiological strategies and thinking that underpin core developments in pharmacovigilancebutwrittenwithageneralistpharmacovigilancescientistinmind.Clini- cal chapters focus on clinical methods for detecting hypotheses for and determining side effectsof medicinalproducts aswell as misdiagnosis pitfalls:written for boththe medically qualifiedbutalsothosethathavelessclinicalbackground. Examples of areas of importance include signal detection, risk management, and risk benefit.Ultimately,onehopesthatthereadershouldcomeawaywithasenseoftheadvances that have occurred in pharmacovigilance methods and approaches but inspired and moti- vatedtoprogressthefieldofpharmacovigilancewithastrongsensethatthereismuchmore worktobedone:asweasafielddoallthatwecantotrytoensurethesafeuseofmedications bypatients. Tadworth,UK AndrewBate References 1. World Health Organization. The importance of pharmacovigilance. http://apps.who.int/iris/ bitstream/10665/42493/1/a75646.pdf.Accessed19June2016 2. McNaughtonR,HuetG,ShakirS(2014)AninvestigationintodrugproductswithdrawnfromtheEU market between 2002 and 2011 for safety reasons and the evidence used to support the decision- making.BMJOpen4(1):e004221 Contents Dedication.................................................................. v Foreword.................................................................... vii Preface ..................................................................... ix Contributors................................................................. xiii 1 CongenitalMalformations ............................................... 1 Euge`nevanPuijenbroek 2 PharmacovigilanceandtheEye........................................... 17 AnthonyP.GrilloandFrederickW.Fraunfelder 3 PharmacovigilanceofHerbalandTraditionalMedicines ..................... 37 LiZhang 4 PediatricPharmacovigilance:CurrentPracticeandFuturePerspectives ........ 67 LiseAagaard 5 DetectingSafetyIssuesinClinicalTrials ................................... 87 EmmaHeeley 6 DevelopmentsandFutureDirectionsofPrescription-Based ObservationalCohortPharmacovigilance.................................. 105 DeborahLayton 7 ElectronicHealthRecord,TransactionalInsuranceClaims, andDistributedDatabasesinPharmacovigilance............................ 135 KevinHaynes 8 PatientRegistriesforSafetyness........................................... 149 MarcusSchmitt-Egenolf 9 PatientReportedOutcomesinPharmacovigilance .......................... 165 LindaHa€rmark 10 Evidence-BasedPharmacovigilanceforMedicinesUsedinPublic HealthProgramsinAfrica ............................................... 185 HaggarHildaAmpadu,YvonneEsseku,andAlexanderN.O.Dodoo 11 PharmacoepidemiologicalApproachesforPopulation-Based HypothesisTesting...................................................... 201 OlafH.Klungel 12 RiskManagementandMinimization...................................... 217 YolaMoride 13 Benefit-RiskAssessmentinPharmacovigilance.............................. 233 OlaCaster ConcludingThoughts ......................................................... 259 Index ...................................................................... 269 xi
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