2020 Alnylam Rare Disease Trend Report

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2020 AlnylamRare Disease Trend ReportPERSPECTIVES FROM HEALTHCARE PAYERSINAUGURAL EDITIONalnylam.com

ForewordIn the past decade, rare disease managementhas become an increased focus for payers.While unmet need and clinical burden of rarediseases have long been acknowledged, thelack of available treatment options has leftpatients living with these diseases despondent.Rare diseases, however, affect nearly 10%of the US population and transformativechanges to research and development, aswell as modernized regulatory pathways,have accelerated the entry of orphan drugsto the market. This uptick has necessitatedtransparent discussions on how best to managethese conditions to ensure the right therapy isprovided to the right patient at the right time.From the perspective of US payers, this inauguralRare Disease Trend Report is a first step increating an open and transparent dialogue onthe challenges faced by insurers in a resourceconstrained healthcare environment. Byhighlighting the key issues and opportunities,we hope that payers, providers, patients, andbiopharmaceutical manufacturers will bridgethe access gaps and ensure patients can receivemedically necessary therapies to ultimatelyimprove patient outcomes.2

PERSPECTIVES FROM HEALTHCARE PAYERS 2020 Alnylam Rare Disease Trend ReportAuthorsEDITORIAL BOARD:GUIDEHOUSE:Jay McKnight PharmD, BCPSVice President, Pharmacy Clinical &Specialty Strategies at HumanaDoug Martin, MDClinical AdvisorKent Rogers, MBASVP, Chief Industry Relations Officer at Optum RxChance Scott, MSManaging Editor & Director of ResearchSneha Peck, MPHEditorial & Project ManagementKathryne Kirk, MBAEditorial & Research3

2020 Alnylam Rare Disease Trend Report PERSPECTIVES FROM HEALTHCARE PAYERS4

PERSPECTIVES FROM HEALTHCARE PAYERS 2020 Alnylam Rare Disease Trend ReportContentsForeword2Authors3Introduction7Key Findings9What factors drive payers to implement active utilization managementstrategies?. 31Does benefit category assignment impact utilization managementtechnique?. 33Scope and Structure.9What impact does setting of care have on benefit category assignment andutilization management?. 36Annual Updates and Other Follow-Up Publications.9How prevalent are rare disease health plan carve-outs?. 3710Distribution: What distribution channels are preferred by payers?. 38Survey Development . 10Distribution of Rare Disease Products. 38Recruitment and Fielding . 10What is the role of the specialty pharmacy?. 40MethodologyParticipant Selection and Demographics . 10Follow-Up Interviews . 11Innovative Payment Models: How are innovative payment models used todayand what is the potential for future use?. 41Data Analysis, Reporting and Analysis . 11Innovative Reimbursement Models for Rare Diseases. 41Payer Priorities: What factors are considered when identifying prioritytherapeutic areas and products?. 12How widely are innovative payment models leveraged in rare diseases?. 43Payer Definitions for Rare Diseases. 12How do payers assess rare disease management?. 15What resources and evidence primarily inform rare disease policydevelopment?. 18What proportion of payer spend is attributable to rare diseases?. 23What challenges do payers face when developing rare disease coveragepolicies?. 26Utilization Management: How do benefit category assignment and payer activemanagement strategies impact patient access?. 27What factors support payer engagement and participation in innovativecontracting?. 45What barriers exist to implementation of innovative reimbursement models?. 49Patient Cost Impact: How do payers consider patient cost burden associatedwith rare disease management?. 49How can payer action impact patient cost burden?. 52Conclusions & Feedback54References54How are benefits within the rare disease space assigned?. 28Availability of Ancillary Benefits . 305

2020 Alnylam Rare Disease Trend Report PERSPECTIVES FROM HEALTHCARE PAYERSThis inaugural issue of theAlnylam Rare Disease TrendReport aims to offer readersa view of the latest trends inrare disease / orphan drugmarket access, summarizingcurrent payer perspectives,and offering insights andpotential implications of thesedata on rare disease andorphan drug management.The report is designed to assistcommercial payers in the U.S.in understanding key trendsand benchmarking rare disease/ orphan drug managementagainst industry peers.6

PERSPECTIVES FROM HEALTHCARE PAYERS 2020 Alnylam Rare Disease Trend ReportIntroductionRare diseases are defined by the U.S. Food &Drug Administration (FDA) as those affectingunder 200,000 patients in the U.S., in accordancewith the Orphan Drug Act of 1983.1 Whileindividual rare diseases affect relatively smallpopulations of patients, the category of raredisease therapeutics on the market is growing,garnering the attention of manufacturers,payers, providers, and patients. Estimatessuggest that rare diseases affect around 30million people ( 10% of the population) livingin the United States (U.S.)—over half of whomare children.2 Many of these diseases negativelyimpact life expectancy and quality of life, andmost remain untreatable. As many rare diseasesare also genetic in nature, they may also lead tofamilial or regional pockets of prevalence. Dueto the absence of therapeutic options for theserare conditions, these new medicines came to beknown as orphan drugs. The significant unmetneed to treat patients with rare diseases ledCongress to pass legislation providing financialincentives to stimulate the development of newtreatments for rare diseases. This legislation,the Orphan Drug Act, went into effect in 1983when only 38 orphan drugs had been approvedin the U.S.3Today the growing pace of innovation madepossible by emerging scientific breakthroughsand new understanding of diseases has ledto the development of novel therapies. Theseinclude modalities that are directed towardgenes and gene expression, allowing providersto address what were previously considereduntreatable diseases. While 2018 saw the highestnumber of annual orphan drug approvals,2020 is expected to surpass this record.2 Still,despite the rise in the number of orphan drugapprovals in recent years, only 5% of rarediseases are treated with commercially availabletherapeutics.2The global orphan drugs market is forecastedto reach 242 billion by 2024, growing at acompound annual growth rate (CAGR) of 12.3%from 2019 to 2024.3 Improved detection andincreased rates of diagnosis of rare diseasesare likely to contribute to rising costs. Whilethe humanitarian benefits of rare diseasetherapies are indisputable, concerns regardinghigh treatment costs play a meaningful role inhow payers administer and manage healthcarebenefits that ultimately dictate patient access toorphan drugs.This report was sponsored and developed by Alnylam Pharmaceuticals, Inc. Alnylam is a biopharmaceutical company leading the translation of RNA interference (RNAi) intoa new class of innovative medicines with the potential to transform the lives of patients who have limited or inadequate treatment options.7

2020 Alnylam Rare Disease Trend Report PERSPECTIVES FROM HEALTHCARE PAYERSAs our healthcare system faces the challenge ofhaving to allocate limited financial resourcesto optimize both patient access and clinicaloutcomes, rapidly rising spending on drugshave encouraged discussion about potentialnovel approaches to manage spending. Severalinitiatives have been proposed or launchedto address the rising costs of U.S. healthcare,such as the Most Favored Nation Pricing model,which uses foreign reference prices as a basis forMedicare drug price negotiations for high cost,high spend drugs. Along with implementingnew utilization management techniques,payers are considering methods such as shiftingmanagement of a drug class from the medicalto pharmacy benefit or mandating the use ofspecialty pharmacies to more effectively managenew market entrants. Another approach thatvarious stakeholders have shown continuedinterest in is the potential to develop cost- orrisk-sharing agreements between the payer anddrug manufacturer (or some other counterparty).The goal of such arrangements is to distributerisk so that no one party is wholly responsiblefor the entire cost of care. Value-Based8Contracting (VBC) agreements have emergedas opportunities to tie clinical outcomes topayment and to adjust for the financial risks thatpayers assume; these are of particular interest inrare disease, where orphan therapies have beenstudied in limited populations and often entailhigh therapy costs.The confidential nature by which each payermakes decisions (e.g., in policy development)bars widespread understanding of whichtactics effectively manage rare diseases. Assuch, the intention of this report is to increasetransparency across the payer community ontrends in the management of rare diseases,with an in-depth assessment on the role ofinnovative reimbursement models within thisspace. Offering key insights and perspectives oncurrent and future management considerationsof rare disease products, as well as some of theperceived challenges that may unfold, will allowfor benchmarking and could potentially lead tomore innovative approaches and opportunitiesfor rare disease and orphan drug management.

PERSPECTIVES FROM HEALTHCARE PAYERS 2020 Alnylam Rare Disease Trend ReportKey Findings Rare Disease Spending as a Driver of RisingHealthcare Costs: Rare disease therapies arehaving a greater budget impact as a proportionof total spend on payer budgets and this trend isprojected to grow. There is widespread concernthat the current system may be unsustainablein the long-run given the rate at which overallhealthcare costs are exceeding the rate of inflation. Uncertain Future for Role of Health EconomicEvidence: While the integral role of clinical efficacyand safety in the product evaluation process is notexpected to change, health economic evidenceis expected to play a greater role in the productevaluation process in the future, particularly in therare disease and orphan drug market. Still, somepayers are hesitant to incorporate such evidenceuntil the results are made enforceable. Continued Challenges with InnovativePayment Models: Payers acknowledge severalbarriers in their ability to develop innovativereimbursement contracts that fairly distributecost- or outcomes-related risks across relevantstakeholders. While some potential methods forovercoming these barriers have been proposed,limitations in data sharing and outcomesmeasurement continue to be challenging forpayers, providers, and manufacturers alike.Scope and StructureThis report focuses on rare diseases and orphandrug management from the U.S. commercialpayer perspective. Rare diseases comprisea group of distinct indications that vary byetiology, pathophysiology, and epidemiology.Naturally, the resulting disease characteristicssuch as prognoses, symptoms, and burdensof illness are similarly variable by specificindication. Additionally, there is little clarity onthe distinction between the “rare/orphan” and“ultra-orphan” conditions. While the currentaccepted definition for a rare disease is that itaffects a population of less than 200,000 patients,payer management of this population may differsubstantially when compared with a conditionaffecting less than, for example, 10,000 patients.Drugs treating patients within this type ofpopulation are sometimes referred to as “ultraorphan” therapies. Given the complexity of rarediseases, they are often managed by physicianspecialists, though the exact type of specialistrequired (e.g., rheumatologist, oncologist,etc.) varies according to the specific disease.Considering the broad and diverse set of diseasesencompassed by the label “rare” and the goal ofthis report, the structure is aligned to five priorityquestions and topics of widespread interest forand relevance to rare diseases: What are the priority therapeutic areas for raredisease management? To what extent do distribution modelsimpact payer management decisions of raredisease products? How are innovative contracting structuresconsidered in the management of raredisease products and what can we expect inthe near future? What is the impact of patient out-of-pocketcosts on payer management decisions for raredisease products?Annual Updates and OtherFollow-Up PublicationsThe payer landscape surrounding rare diseasetreatment and management has been changingrapidly in the U.S. and shows no signs of slowing infuture years. As such, this inaugural report will beupdated on an annual basis to capture the evolutionof trends representative of the current environment.The topics included in this report could each,independently, support an entire report.Given the amount of content and interestassociated with the topics presented, there is thepossibility that follow-up reports exploring theconsiderations, challenges, and opportunities ingreater depth will be published. How do payers currently manage rare diseaseproducts and how are management techniqueslikely to evolve in the near future?9

MethodologyThis publication was sponsored and developedby Alnylam Pharmaceuticals, Inc., in partnershipwith Navigant. Alnylam is a biopharmaceuticalcompany focused on the discovery, development,and commercialization of RNA interference (RNAi)therapeutics. Research services were providedby the Commercial Health Group at Navigant,a Guidehouse company, a leading globalconsultancy that specializes in life sciences acrossboth the commercial and public sectors.Survey DevelopmentA survey was developed to capture payersentiment on the quantitative management ofrare diseases. The survey was designed to assesscurrent practices and perspectives as well asto gather information on anticipated changesover the next five years and beyond. Taking2019 as the current health plan year, the surveyspecifically dives into anticipated changes withinthe next plan year (2020), the next 3–5 plan years(2023–2025), and beyond (2026 ).The survey focused on the same payer-resonantthemes used to inform report structure, namely:priority therapeutic areas, benefit assignmentand utilization management, distribution,innovative contracting, and patient costs. Nospecific products were assessed.10The majority (n 31) of the 55 survey questionswere multiple choice questions limited toone response. The remaining questions were:multiple choice questions allowing for multipleanswers to be selected (n 4); forced relativerankings (n 4); forced scale-based ratings(n 4); categorical selections (n 2); respondentdesignated 100% distribution (n 1); or specificindications of anticipated times-to-events (n 9).Potential respondents were screened forparticipation (see ‘Prequalification Criteria’below), and a total of thirty U.S.-based medicaland pharmacy directors meeting the predefinedeligibility criteria were recruited to completethe survey and provided with the online link.Guidehouse partnered with a commercial vendorto recruit participants and translate the surveyto an online format.Recruitment and FieldingPotential respondents were identified by thevendor using predefined screening criteria (see‘Prequalification Criteria’ below). Guidehouseprovided respondents with an assurance thatonly blinded, aggregated data would be madeavailable to the broader public. The authors thenselected participants to ensure a mix of bothmedical and pharmacy directors from a variety ofhealth plan types (i.e., commercial or managedMedicaid affiliate), as well as a mix of stakeholderswith national and regional purviews.The survey was completed by all participantsover the course of four weeks, from August23 to September 17, 2019. While the surveyrespondents may choose to participate in theannual updates to the research, each sampleof respondents will be considered as anindependent sample.Participant Selection andDemographicsResearch participants were required to meetqualifying criteria to ensure integrity ofresponses across the various topics. Specificprequalification criteria for payers included: Current medical or pharmacy director employedby a commercial or managed Medicaid payer, or apharmacy benefit manager Active involvement in policy developmentwithin the organization, including experiencedeveloping policies for rare disease andmanagement

PERSPECTIVES FROM HEALTHCARE PAYERS 2020 Alnylam Rare Disease Trend Report Willingness and ability to discuss decisionmaking focused on rare disease products,such as new product evaluations, pharmacyand therapeutics (P&T) committee processes,innovative reimbursement model compositionand implementation, and distribution networkdeterminationsFollow-Up InterviewsEach respondent participated in a 30-minutefollow-up interview during which the respondentwas probed to provide additional, qualitativeinsight. All interviews were conducted overthe phone by Guidehouse researchers. Theinterviews were conducted in a double-blindedmanner, such that no respondent knew thecompany supporting the research and noAlnylam employee knew which payer individualswere providing input. All interviewees providedconsent for using their responses in thecomposition of this report.Honoraria were paid to respondents who met alleligibility criteria and completed the survey andfollow-up phone interview.Data Analysis, Reportingand LimitationsSurvey and interview responses were collected,analyzed, and reported by Guidehouse. Data wasblinded and aggregated across the entire sampleof respondents.Guidehouse had no way of validating surveyresponses for accuracy regarding payer practicesor internal processes/operations. All statementsand opinions contained within the reportreflect responses received by included payerparticipants and do not necessarily reflect thoseof Alnylam or other reviewers.11

Payer Priorities:How do payers identifypriority therapeutic areasand products?Payer Definitions forRare Diseases12The Orphan Drug Act of 1983 characterizesrare diseases by an absolute prevalence rate,however, the collective set of

Alnylam Rare Disease Trend Report aims to offer readers a view of the latest trends in rare disease / orphan drug market access, summarizing current payer perspectives, and offering insights and potential implications of these data on rare disease and orphan drug management. The r

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