医疗补助计划中加速批准药物使用者的人口统计特征

MILLIMANWHITE PAPER Demographic Characteristics ofAcceleratedApprovalDrugUtilizersin Medicaid Commissioned by PhRMA Katie Holcomb, FSA, MAAABriana Botros, FSA, MAAAKristin Niakan,MPHBenton Whaley require long study periods. Following accelerated approval,manufacturers are required to conductpost-approval studiestoverifytheanticipatedclinical benefit.Upon verification ofa clinicalbenefit,thedrugcan be converted totraditional approval.However, the acceleratedapproval can be withdrawn ifpost-approval studies fail to verifythe expectedclinical benefit. SUMMARY TheUSFood and Drug Administration’s(FDA)acceleratedapproval pathway enables expedited access todrugs thataddress an unmet need for serious and life-threatening diseasesand conditions.Milliman analyzedthe demographiccharacteristics of Medicaidbeneficiarieswho utilizeacceleratedapprovaldrugsusing2018-2019 national data from theTransformed Medicaid Statistical Information System (T-MSIS).Overall, we found: The accelerated approval pathway was formally established bythe FDA in 1992 in recognition of theurgent unmet medical needfortreatments with lifesaving potential during the HIV / AIDSepidemic.3Between the program’s initiation in 1992 and 2010,accelerated approval was predominantly utilized fordrugs to treatHIV, cancer, and rare conditions. From 2010 to 2020, 85% ofdrugs approved via accelerated approvalwereoncology drugs.4Figure1below illustratesthe therapeutic classes that have haddrugswith accelerated approval in recent years. ▪On average,accelerated approvaldrugutilizers aremorelikely to beolderandaremore commonlyeligibleforMedicaid based on their disability statuscompared toMedicaid beneficiaries usingnon-accelerated approvaldrugs. This is likely related to the types of conditionsaccelerated approvaldrugstreat.It is important to note,thatsome of thesedrugsmay only beindicated for adults, whileMedicaid covers a large number of children. ▪We observed a higher proportion of Black and Asianbeneficiaries using accelerated approval drugsthan for allother drug utilizers,based onrace and ethnicitydatareported in T-MSIS.Demographic characteristics ofaccelerated approval drug utilizers are directly influenced bythe demographic characteristics of populationsdisproportionately impacted by certain diseases. BACKGROUND Accelerated approvalallows for the approval ofdrugsthat treatseriousor life-threatening diseasesconditionsand that fill anunmet medical need baseduponmeasure of asurrogateendpoint1. A surrogate endpoint isamarker, such asalaboratoryresult or imaging, that isreasonably likelyto predict a clinicalbenefit, but is not a measure of how the patient feels, functions orsurvives.2Surrogate endpointsfacilitate earlier access than atraditional approval because theycan be measured earlier thanadirect measure of clinicalbenefit,such as overall survival.This isbeneficialespecially whendirect measures ofclinicalbenefit Drugs approved under accelerated approval made up28.0% ofall FDA drug approvals in 2021 and22.6%of all FDA drug approvals in 2020.6,7This is up from2019 (18.8%), 2018 (6.8%)and 2017(13.0%).8,9,10The overall number of drug approvalsdropped in 2020 due to the COVID-19 pandemic, but it is difficultto say whetheracceleratedapprovalswere affectedsimilarly astraditionalapprovals. RESULTS The graphs below summarize the 2018 and 2019 demographicsofnationwideMedicaid beneficiaries who use acceleratedapproval drugs compared to the demographics of Medicaidbeneficiaries using anyotherprescription drug, includingdrugsprovidedunder the medical benefit.Our analysis included67milliondrugutilizers, of which 455,000were acceleratedapprovaldrugutilizers.The demographics summarized includeage,sex, income as a percent of the Federal PovertyLevel(FPL),race/ethnicity,anddisability status(defined byenrollmentin Medicaid on the basis of disability). These results includeutilization across all indications of drugsever receivingaccelerated approval. Oncology drugs are some of the most utilized acceleratedapproval drugs in Medicaid. As such, the demographiccharacteristics of accelerated approval utilizers is likely reflectiveof the demographic characteristics of individuals with cancer,which is supported by our results. WHITE PAPER There is a higher proportion of females usingdrugs for both thepopulation utilizing accelerated approvaldrugsand thepopulation utilizing otherdrugs.This is expected, as theproportion of womenenrolledin Medicaidis higher in general. While thesexdistribution of members who utilized acceleratedapprovaldrugsis very similar to those whodidnot useaccelerated approvaldrugs, the age distribution is quite different.Accelerated approvaldrugutilizersskew toward higher agebuckets on averageandaremore likely to beolderthanmembers who have not utilized accelerated approvaldrugs. The income distribution ofpatients takingaccelerated approvaldrugsissimilar to those who have nottakenacceleratedapprovaldrugs, although approximately50% of the sample w