The promise of futility trials in neurological diseases.
Identifieur interne : 000517 ( PubMed/Corpus ); précédent : 000516; suivant : 000518The promise of futility trials in neurological diseases.
Auteurs : Marcus W. Koch ; Lawrence Korngut ; David G. Patry ; Yahya Agha-Khani ; Christopher White ; Justyna R. Sarna ; Michael Yeung ; V Wee Yong ; Daniel Y C. Heng ; Gary Cutter ; Luanne MetzSource :
- Nature reviews. Neurology [ 1759-4766 ] ; 2015.
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Abstract
Double-blinded randomized controlled trials (RCTs) have contributed much important evidence to guide treatment decisions in neurology. RCTs are relatively straightforward to conduct, provided that they investigate common diseases, have clearly defined outcome measures, and are of short duration. In neurology, however, many diseases are uncommon, have no consensus outcome measures, and develop over decades. Basic research into neurological diseases continues to identify candidate therapies faster than they can be tested for their clinical utility, leading to a 'translational gap'. Futility trials were initially developed in oncology to efficiently test candidate therapies in phase II trials. As single-arm unblinded studies, futility trials are relatively easy to conduct, and they generally require smaller sample sizes than RCTs. In this article, we discuss futility models, highlighting their advantages as well as challenges to their application in several neurological diseases, including Parkinson disease, stroke and multiple sclerosis.
DOI: 10.1038/nrneurol.2015.34
PubMed: 25776824
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[2] Department of Community Health Sciences, TRW Building, University of Calgary, 3280 Hospital Drive NW, Calgary, AB T2N 4Z6, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Korngut, Lawrence" sort="Korngut, Lawrence" uniqKey="Korngut L" first="Lawrence" last="Korngut">Lawrence Korngut</name><affiliation><nlm:affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Patry, David G" sort="Patry, David G" uniqKey="Patry D" first="David G" last="Patry">David G. 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Heng</name><affiliation><nlm:affiliation>Department of Oncology, Tom Baker Cancer Centre, 1331 29th Street NW, Calgary, AB T2N 4N2, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Cutter, Gary" sort="Cutter, Gary" uniqKey="Cutter G" first="Gary" last="Cutter">Gary Cutter</name><affiliation><nlm:affiliation>Department of Biostatistics, School of Public Health, University of Alabama at Birmingham, 1665 University Boulevard, Birmingham, AL 35294-0022, USA.</nlm:affiliation></affiliation></author><author><name sortKey="Metz, Luanne" sort="Metz, Luanne" uniqKey="Metz L" first="Luanne" last="Metz">Luanne Metz</name><affiliation><nlm:affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</nlm:affiliation></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2015">2015</date><idno type="RBID">pubmed:25776824</idno><idno type="pmid">25776824</idno><idno type="doi">10.1038/nrneurol.2015.34</idno><idno type="wicri:Area/PubMed/Corpus">000517</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Corpus" wicri:corpus="PubMed">000517</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en">The promise of futility trials in neurological diseases.</title><author><name sortKey="Koch, Marcus W" sort="Koch, Marcus W" uniqKey="Koch M" first="Marcus W" last="Koch">Marcus W. Koch</name><affiliation><nlm:affiliation>1] Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada. [2] Department of Community Health Sciences, TRW Building, University of Calgary, 3280 Hospital Drive NW, Calgary, AB T2N 4Z6, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Korngut, Lawrence" sort="Korngut, Lawrence" uniqKey="Korngut L" first="Lawrence" last="Korngut">Lawrence Korngut</name><affiliation><nlm:affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Patry, David G" sort="Patry, David G" uniqKey="Patry D" first="David G" last="Patry">David G. Patry</name><affiliation><nlm:affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Agha Khani, Yahya" sort="Agha Khani, Yahya" uniqKey="Agha Khani Y" first="Yahya" last="Agha-Khani">Yahya Agha-Khani</name><affiliation><nlm:affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="White, Christopher" sort="White, Christopher" uniqKey="White C" first="Christopher" last="White">Christopher White</name><affiliation><nlm:affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Sarna, Justyna R" sort="Sarna, Justyna R" uniqKey="Sarna J" first="Justyna R" last="Sarna">Justyna R. Sarna</name><affiliation><nlm:affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Yeung, Michael" sort="Yeung, Michael" uniqKey="Yeung M" first="Michael" last="Yeung">Michael Yeung</name><affiliation><nlm:affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Yong, V Wee" sort="Yong, V Wee" uniqKey="Yong V" first="V Wee" last="Yong">V Wee Yong</name><affiliation><nlm:affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Heng, Daniel Y C" sort="Heng, Daniel Y C" uniqKey="Heng D" first="Daniel Y C" last="Heng">Daniel Y C. Heng</name><affiliation><nlm:affiliation>Department of Oncology, Tom Baker Cancer Centre, 1331 29th Street NW, Calgary, AB T2N 4N2, Canada.</nlm:affiliation></affiliation></author><author><name sortKey="Cutter, Gary" sort="Cutter, Gary" uniqKey="Cutter G" first="Gary" last="Cutter">Gary Cutter</name><affiliation><nlm:affiliation>Department of Biostatistics, School of Public Health, University of Alabama at Birmingham, 1665 University Boulevard, Birmingham, AL 35294-0022, USA.</nlm:affiliation></affiliation></author><author><name sortKey="Metz, Luanne" sort="Metz, Luanne" uniqKey="Metz L" first="Luanne" last="Metz">Luanne Metz</name><affiliation><nlm:affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</nlm:affiliation></affiliation></author></analytic><series><title level="j">Nature reviews. Neurology</title><idno type="eISSN">1759-4766</idno><imprint><date when="2015" type="published">2015</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Clinical Trials, Phase II as Topic</term><term>Cognition Disorders (therapy)</term><term>Humans</term><term>Medical Futility</term><term>Models, Biological</term><term>Nervous System Diseases (therapy)</term><term>Randomized Controlled Trials as Topic</term><term>Sample Size</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Cognition Disorders</term><term>Nervous System Diseases</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Clinical Trials, Phase II as Topic</term><term>Humans</term><term>Medical Futility</term><term>Models, Biological</term><term>Randomized Controlled Trials as Topic</term><term>Sample Size</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Double-blinded randomized controlled trials (RCTs) have contributed much important evidence to guide treatment decisions in neurology. RCTs are relatively straightforward to conduct, provided that they investigate common diseases, have clearly defined outcome measures, and are of short duration. In neurology, however, many diseases are uncommon, have no consensus outcome measures, and develop over decades. Basic research into neurological diseases continues to identify candidate therapies faster than they can be tested for their clinical utility, leading to a 'translational gap'. Futility trials were initially developed in oncology to efficiently test candidate therapies in phase II trials. As single-arm unblinded studies, futility trials are relatively easy to conduct, and they generally require smaller sample sizes than RCTs. In this article, we discuss futility models, highlighting their advantages as well as challenges to their application in several neurological diseases, including Parkinson disease, stroke and multiple sclerosis.</div></front></TEI><pubmed><MedlineCitation Status="MEDLINE" Owner="NLM"><PMID Version="1">25776824</PMID><DateCreated><Year>2015</Year><Month>05</Month><Day>07</Day></DateCreated><DateCompleted><Year>2016</Year><Month>03</Month><Day>21</Day></DateCompleted><DateRevised><Year>2015</Year><Month>05</Month><Day>07</Day></DateRevised><Article PubModel="Print-Electronic"><Journal><ISSN IssnType="Electronic">1759-4766</ISSN><JournalIssue CitedMedium="Internet"><Volume>11</Volume><Issue>5</Issue><PubDate><Year>2015</Year><Month>May</Month></PubDate></JournalIssue><Title>Nature reviews. Neurology</Title><ISOAbbreviation>Nat Rev Neurol</ISOAbbreviation></Journal><ArticleTitle>The promise of futility trials in neurological diseases.</ArticleTitle><Pagination><MedlinePgn>300-5</MedlinePgn></Pagination><ELocationID EIdType="doi" ValidYN="Y">10.1038/nrneurol.2015.34</ELocationID><Abstract><AbstractText>Double-blinded randomized controlled trials (RCTs) have contributed much important evidence to guide treatment decisions in neurology. RCTs are relatively straightforward to conduct, provided that they investigate common diseases, have clearly defined outcome measures, and are of short duration. In neurology, however, many diseases are uncommon, have no consensus outcome measures, and develop over decades. Basic research into neurological diseases continues to identify candidate therapies faster than they can be tested for their clinical utility, leading to a 'translational gap'. Futility trials were initially developed in oncology to efficiently test candidate therapies in phase II trials. As single-arm unblinded studies, futility trials are relatively easy to conduct, and they generally require smaller sample sizes than RCTs. In this article, we discuss futility models, highlighting their advantages as well as challenges to their application in several neurological diseases, including Parkinson disease, stroke and multiple sclerosis.</AbstractText></Abstract><AuthorList CompleteYN="Y"><Author ValidYN="Y"><LastName>Koch</LastName><ForeName>Marcus W</ForeName><Initials>MW</Initials><AffiliationInfo><Affiliation>1] Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada. [2] Department of Community Health Sciences, TRW Building, University of Calgary, 3280 Hospital Drive NW, Calgary, AB T2N 4Z6, Canada.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Korngut</LastName><ForeName>Lawrence</ForeName><Initials>L</Initials><AffiliationInfo><Affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Patry</LastName><ForeName>David G</ForeName><Initials>DG</Initials><AffiliationInfo><Affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Agha-Khani</LastName><ForeName>Yahya</ForeName><Initials>Y</Initials><AffiliationInfo><Affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>White</LastName><ForeName>Christopher</ForeName><Initials>C</Initials><AffiliationInfo><Affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Sarna</LastName><ForeName>Justyna R</ForeName><Initials>JR</Initials><AffiliationInfo><Affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Yeung</LastName><ForeName>Michael</ForeName><Initials>M</Initials><AffiliationInfo><Affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Yong</LastName><ForeName>V Wee</ForeName><Initials>VW</Initials><AffiliationInfo><Affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Heng</LastName><ForeName>Daniel Y C</ForeName><Initials>DY</Initials><AffiliationInfo><Affiliation>Department of Oncology, Tom Baker Cancer Centre, 1331 29th Street NW, Calgary, AB T2N 4N2, Canada.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Cutter</LastName><ForeName>Gary</ForeName><Initials>G</Initials><AffiliationInfo><Affiliation>Department of Biostatistics, School of Public Health, University of Alabama at Birmingham, 1665 University Boulevard, Birmingham, AL 35294-0022, USA.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Metz</LastName><ForeName>Luanne</ForeName><Initials>L</Initials><AffiliationInfo><Affiliation>Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, AB T2N 2T9, Canada.</Affiliation></AffiliationInfo></Author></AuthorList><Language>eng</Language><PublicationTypeList><PublicationType UI="D016428">Journal Article</PublicationType><PublicationType UI="D016454">Review</PublicationType></PublicationTypeList><ArticleDate DateType="Electronic"><Year>2015</Year><Month>03</Month><Day>17</Day></ArticleDate></Article><MedlineJournalInfo><Country>England</Country><MedlineTA>Nat Rev Neurol</MedlineTA><NlmUniqueID>101500072</NlmUniqueID><ISSNLinking>1759-4758</ISSNLinking></MedlineJournalInfo><CitationSubset>IM</CitationSubset><MeshHeadingList><MeshHeading><DescriptorName UI="D017322" MajorTopicYN="N">Clinical Trials, Phase II as Topic</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D003072" 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