{"id":"https://openalex.org/W7204759317","doi":"https://doi.org/10.1016/j.drudis.2026.104792","title":"Rare disease benchmarks: Investment professionals\u2019 accuracy and beliefs","display_name":"Rare disease benchmarks: Investment professionals\u2019 accuracy and beliefs","publication_year":2026,"publication_date":"2026-09-01","ids":{"openalex":"https://openalex.org/W7204759317","doi":"https://doi.org/10.1016/j.drudis.2026.104792","pmid":"https://pubmed.ncbi.nlm.nih.gov/42674209"},"language":"en","primary_location":{"id":"doi:10.1016/j.drudis.2026.104792","doi":"https://doi.org/10.1016/j.drudis.2026.104792","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.drudis.2026.104792","pdf_url":null,"source":{"id":"https://openalex.org/S152068293","display_name":"Drug Discovery Today","issn_l":"1359-6446","issn":["1359-6446","1878-5832"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-2","ki-jl-1","medline","norway-1","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Drug Discovery Today","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://doi.org/10.1016/j.drudis.2026.104792","any_repository_has_fulltext":false},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5148762793","display_name":"Jonathan Ilicki","orcid":"https://orcid.org/0000-0002-6514-8554","observed_orcids":["https://orcid.org/0000-0002-6514-8554"]},"institutions":[],"countries":[],"is_corresponding":true,"raw_author_name":"Jonathan Ilicki","raw_affiliation_strings":["Industrifonden, Box 1163, 111 91 Stockholm, Sweden. Electronic address: j.ilicki@gmail.com"],"raw_orcid":"https://orcid.org/0000-0002-6514-8554","affiliations":[{"raw_affiliation_string":"Industrifonden, Box 1163, 111 91 Stockholm, Sweden. Electronic address: j.ilicki@gmail.com","institution_ids":[]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5014797356","display_name":"Alexander James Spicer","orcid":"https://orcid.org/0000-0002-7819-1906","observed_orcids":["https://orcid.org/0000-0002-7819-1906"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Alexander J. Spicer","raw_affiliation_strings":["SERB Pharmaceuticals, Unit 1005, Cannon Green, 27 Bush Ln, London EC4R 0AA, UK"],"raw_orcid":"https://orcid.org/0000-0002-7819-1906","affiliations":[{"raw_affiliation_string":"SERB Pharmaceuticals, Unit 1005, Cannon Green, 27 Bush Ln, London EC4R 0AA, UK","institution_ids":[]}]}],"institutions":[],"countries_distinct_count":0,"institutions_distinct_count":0,"corresponding_author_ids":["https://openalex.org/A5148762793"],"corresponding_institution_ids":[],"apc_list":{"value":4720,"currency":"USD","value_usd":4720},"apc_paid":{"value":4720,"currency":"USD","value_usd":4720},"fwci":0.0,"has_fulltext":false,"cited_by_count":0,"citation_normalized_percentile":{"value":0.0,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":null,"biblio":{"volume":"31","issue":"5","first_page":"104792","last_page":"104792"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T11792","display_name":"Pharmaceutical Economics and Policy","score":0.7792999744415283,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},"topics":[{"id":"https://openalex.org/T11792","display_name":"Pharmaceutical Economics and Policy","score":0.7792999744415283,"subfield":{"id":"https://openalex.org/subfields/2002","display_name":"Economics and Econometrics"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},{"id":"https://openalex.org/T11903","display_name":"Private Equity and Venture Capital","score":0.11890000104904175,"subfield":{"id":"https://openalex.org/subfields/2003","display_name":"Finance"},"field":{"id":"https://openalex.org/fields/20","display_name":"Economics, Econometrics and Finance"},"domain":{"id":"https://openalex.org/domains/2","display_name":"Social Sciences"}},{"id":"https://openalex.org/T11649","display_name":"Pharmaceutical industry and healthcare","score":0.015699999406933784,"subfield":{"id":"https://openalex.org/subfields/2910","display_name":"Issues, ethics and legal aspects"},"field":{"id":"https://openalex.org/fields/29","display_name":"Nursing"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/rare-diseases","display_name":"rare diseases","score":0.9580000042915344},{"id":"https://openalex.org/keywords/investment-professionals","display_name":"investment professionals","score":0.4580000042915344},{"id":"https://openalex.org/keywords/benchmarking","display_name":"benchmarking","score":0.41999998688697815},{"id":"https://openalex.org/keywords/calibration","display_name":"calibration","score":0.3790000081062317},{"id":"https://openalex.org/keywords/acquisition-timing","display_name":"acquisition timing","score":0.8500000238418579},{"id":"https://openalex.org/keywords/launch-performance","display_name":"launch performance","score":0.8349999785423279},{"id":"https://openalex.org/keywords/venture-capital","display_name":"venture capital","score":0.734000027179718},{"id":"https://openalex.org/keywords/business-development","display_name":"business development","score":0.7110000252723694},{"id":"https://openalex.org/keywords/underestimation","display_name":"underestimation","score":0.5099999904632568}],"concepts":[{"id":"https://openalex.org/C27548731","wikidata":"https://www.wikidata.org/wiki/Q88272","display_name":"Investment (military)","level":3,"score":0.6524999737739563},{"id":"https://openalex.org/C2779701055","wikidata":"https://www.wikidata.org/wiki/Q929833","display_name":"Rare disease","level":3,"score":0.6442000269889832},{"id":"https://openalex.org/C2777317252","wikidata":"https://www.wikidata.org/wiki/Q18393516","display_name":"Rare events","level":2,"score":0.5095000267028809},{"id":"https://openalex.org/C144133560","wikidata":"https://www.wikidata.org/wiki/Q4830453","display_name":"Business","level":0,"score":0.5012999773025513},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.4706999957561493},{"id":"https://openalex.org/C162118730","wikidata":"https://www.wikidata.org/wiki/Q1128453","display_name":"Actuarial science","level":1,"score":0.4410000145435333},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.3801000118255615},{"id":"https://openalex.org/C112930515","wikidata":"https://www.wikidata.org/wiki/Q4389547","display_name":"Risk analysis (engineering)","level":1,"score":0.34549999237060547},{"id":"https://openalex.org/C162324750","wikidata":"https://www.wikidata.org/wiki/Q8134","display_name":"Economics","level":0,"score":0.31929999589920044},{"id":"https://openalex.org/C2779473830","wikidata":"https://www.wikidata.org/wiki/Q1540899","display_name":"MEDLINE","level":2,"score":0.31310001015663147},{"id":"https://openalex.org/C177713679","wikidata":"https://www.wikidata.org/wiki/Q679690","display_name":"Intensive care medicine","level":1,"score":0.31139999628067017},{"id":"https://openalex.org/C2993777677","wikidata":"https://www.wikidata.org/wiki/Q507443","display_name":"Drug industry","level":2,"score":0.2996000051498413},{"id":"https://openalex.org/C100001284","wikidata":"https://www.wikidata.org/wiki/Q2248246","display_name":"Public economics","level":1,"score":0.26579999923706055}],"mesh":[{"descriptor_ui":"D004345","descriptor_name":"Drug Industry","qualifier_ui":"Q000191","qualifier_name":"economics","is_major_topic":true},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007449","descriptor_name":"Investments","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D009965","descriptor_name":"Orphan Drug Production","qualifier_ui":"Q000191","qualifier_name":"economics","is_major_topic":false},{"descriptor_ui":"D011795","descriptor_name":"Surveys and Questionnaires","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D019985","descriptor_name":"Benchmarking","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D035583","descriptor_name":"Rare Diseases","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D035583","descriptor_name":"Rare Diseases","qualifier_ui":"Q000191","qualifier_name":"economics","is_major_topic":true}],"locations_count":2,"locations":[{"id":"doi:10.1016/j.drudis.2026.104792","doi":"https://doi.org/10.1016/j.drudis.2026.104792","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.drudis.2026.104792","pdf_url":null,"source":{"id":"https://openalex.org/S152068293","display_name":"Drug Discovery Today","issn_l":"1359-6446","issn":["1359-6446","1878-5832"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-2","ki-jl-1","medline","norway-1","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Drug Discovery Today","raw_type":"journal-article"},{"id":"pmid:42674209","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/42674209","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Drug discovery today","raw_type":"Journal Article"}],"best_oa_location":{"id":"doi:10.1016/j.drudis.2026.104792","doi":"https://doi.org/10.1016/j.drudis.2026.104792","is_oa":true,"landing_page_url":"https://doi.org/10.1016/j.drudis.2026.104792","pdf_url":null,"source":{"id":"https://openalex.org/S152068293","display_name":"Drug Discovery Today","issn_l":"1359-6446","issn":["1359-6446","1878-5832"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-2","ki-jl-1","medline","norway-1","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320990","host_organization_name":"Elsevier BV","host_organization_lineage":["https://openalex.org/P4310320990"],"host_organization_lineage_names":["Elsevier BV"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Drug Discovery Today","raw_type":"journal-article"},"sustainable_development_goals":[{"display_name":"Good health and well-being","score":0.4043000042438507,"id":"https://metadata.un.org/sdg/3"}],"study_designs":[{"display_name":"Observational Study","id":"https://openalex.org/study-designs/observational-study"}],"awards":[],"funders":[],"has_content":{"pdf":false,"grobid_xml":false},"content_urls":null,"referenced_works_count":13,"referenced_works":["https://openalex.org/W2118822739","https://openalex.org/W2140543255","https://openalex.org/W2169521630","https://openalex.org/W2545421535","https://openalex.org/W2995245946","https://openalex.org/W4206278453","https://openalex.org/W4233465538","https://openalex.org/W4292157289","https://openalex.org/W4375946897","https://openalex.org/W4394892101","https://openalex.org/W4413042502","https://openalex.org/W4416897712","https://openalex.org/W7160535716"],"related_works":["https://openalex.org/W2801849778","https://openalex.org/W7117488137","https://openalex.org/W1987248838","https://openalex.org/W7222902657","https://openalex.org/W3128481716","https://openalex.org/W7203606099","https://openalex.org/W2802935287","https://openalex.org/W2092445703","https://openalex.org/W4399707345","https://openalex.org/W3006408041"],"abstract_inverted_index":{"Rare-disease":[0],"assets":[1,128,156],"account":[2],"for":[3,19,57,97],"a":[4,142],"substantial":[5],"and":[6,12,14,23,28,48,68,71,119,148],"growing":[7],"share":[8],"of":[9,126,137],"biopharmaceutical":[10],"development":[11],"dealmaking,":[13],"published":[15,77,117,146],"analyses":[16],"provide":[17],"benchmarks":[18,36,56],"their":[20,73],"clinical,":[21],"commercial":[22],"financial":[24],"performance.":[25],"Whether":[26],"investment":[27],"business-development":[29],"practitioners":[30],"hold":[31],"beliefs":[32],"consistent":[33],"with":[34,75,112,135],"these":[35],"is":[37],"largely":[38],"unexamined.":[39],"We":[40],"surveyed":[41],"43":[42],"professionals":[43],"across":[44],"venture":[45],"capital,":[46],"pharmaceutical":[47],"biotechnology":[49],"business":[50],"development,":[51],"asking":[52],"them":[53],"to":[54,65,157],"estimate":[55],"approval":[58],"probability,":[59],"acquisition":[60],"timing,":[61],"exit":[62],"economics,":[63],"time":[64],"peak":[66],"sales":[67],"launch":[69],"performance,":[70],"compared":[72],"responses":[74],"the":[76,94,109],"literature.":[78],"Respondents":[79],"were":[80],"generally":[81],"well":[82],"calibrated":[83],"on":[84,103],"some":[85,154],"structural":[86],"characteristics":[87],"visible":[88],"through":[89],"deal":[90],"flow,":[91],"such":[92],"as":[93],"dominant":[95],"modality":[96],"rare-disease":[98,127,155],"assets,":[99],"but":[100],"systematically":[101],"miscalibrated":[102],"specific":[104],"magnitudes.":[105],"Launch":[106],"performance":[107,164],"was":[108,132],"most":[110,120],"underestimated,":[111],"84%":[113],"placing":[114],"it":[115],"below":[116],"levels,":[118],"respondents":[121],"underestimating":[122],"how":[123],"late":[124],"acquisitions":[125],"occur.":[129],"Estimate":[130],"accuracy":[131],"not":[133],"associated":[134],"years":[136],"experience.":[138],"These":[139],"findings":[140],"suggest":[141],"wide":[143],"gap":[144],"between":[145],"evidence":[147],"practitioner":[149],"belief,":[150],"which":[151],"might":[152],"lead":[153],"be":[158],"assessed":[159],"more":[160],"conservatively":[161],"than":[162],"historical":[163],"would":[165],"justify.":[166]},"counts_by_year":[],"updated_date":"2026-09-26T08:19:03.415552","created_date":"2026-09-01T00:00:00"}
