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急性骨髄性白血病治療薬の価格、償還、アクセス

Acute Myeloid Leukemia Pricing, Reimbursement, and Access

出版日: | 発行: Datamonitor Healthcare | ページ情報: 英文 132 Pages | 納期: 即日から翌営業日

価格
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本日の銀行送金レート: 1USD=109.37円
急性骨髄性白血病治療薬の価格、償還、アクセス
出版日: 2017年12月15日
発行: Datamonitor Healthcare
ページ情報: 英文 132 Pages
納期: 即日から翌営業日
  • 全表示
  • 概要
  • 目次
概要

急性骨髄性白血病治療薬市場は、固形腫瘍と比較して患者数が比較的少なく、重症度が高く、ブランド薬が不足していることから、注目度はこれまで低いものでした。しかし最近になり薬剤が認可され、後期パイプラインが充実してきたことから、多くの参入企業が将来的な予算投入に関心を示しています。

当レポートでは、急性骨髄性白血病治療薬市場について調査し、市場の現状と価格動向、法規制動向、製品概要とパイプライン動向、国別動向、今後の見通しなどをまとめています。

エグゼクティブサマリー

法規制ラベル

  • 米国、日本、欧州主要5カ国における製品
  • 関連情報

典型的支払者

  • 動向と戦略的提言
  • 現在の予算規模は小さいが更なる認可に向け増加する見込み
  • 現在は比較的受動的アプローチをとっている
  • Vidaza、Dacogenなどの上市済み薬剤に対する規制レベルは緩やか
  • 治療は個別化の方向になっている
  • 将来的治療法は併用療法
  • アンメットニーズの高さから投与ルートが有効性の決め手となる
  • 最近認可された治療薬およびパイプライン薬は複合的ラベル拡大を求めており、結果的に価格低下につながる
  • 関連情報

最近上市された製品、パイプライン製品へのアクセス

  • Rydapt
  • Gilteritinib、quizartinib
  • Vyxeos
  • Guadecitabine、Venclexta
  • Rydaptは好ましい価格、償還の提言を得られる可能性があるがEUは年齢により規制される可能性がある
  • 第二世代のFLT3阻害剤は臨床アウトカムを改善する見込み
  • Vyxeosは様々な資金調達により影響を受ける見込み
  • Vyxeosはより好ましい注入スケジュールにより恩恵を受ける見込み
  • ジェネリックのVidazaは一定のパイプライン治療薬について欧州でベンチマークとなる
  • 関連情報

治験デザインと証拠用件

  • 動向と戦略的提言
  • OSが最も重要なエンドポイントだがPFSで十分な場合もある
  • OS改善の閾値は治療ラインにより変化する
  • HRQoLデータは重要だがほとんど収集されない
  • 年齢が治療可能性の最善の予測因子
  • 治験責任医師の選定が認められたが各市場でSoCを反映しなければならない
  • 幹細胞移植の可能性を示すには再発率の改善が重要
  • 関連情報

米国の価格

米国の償還

日本

欧州5カ国の価格

  • フランス
  • ドイツ
  • イタリア
  • 英国
目次
Product Code: DMKC0185628

The level of concern surrounding the budget impact of acute myeloid leukemia (AML) has traditionally been low due to the relatively small size of the population in comparison to solid tumors, the high severity of the disease, and a lack of branded treatment options. As a result, few access controls are utilized for AML therapies. However, with several AML medications recently gaining approval, and many more in the late-stage pipeline, many interviewed payers express concern about the potentially growing budget impact of the indication in the future.

A considerable proportion of the recently approved and pipeline AML treatments are mutationally targeted, and despite their smaller target patient populations, are anticipated to contribute heavily to the increasing cost of therapy. Payers and key opinion leaders also highlight the likelihood of treatment with combinations of targeted therapies in the future, further increasing the level of payer concern. Due to the rising expenditure, payer acceptance will become more critical for the commercial success of new AML therapies. This analysis examines payer views on recently approved and pipeline AML agents, the tools used to moderate budget impact, and changing evidentiary requirements.

TABLE OF CONTENTS

EXECUTIVE SUMMARY

REGULATORY LABELS

  • AML products in the US, Japan, and five major EU markets
  • Bibliography

PAYER ARCHETYPES

  • Insights and strategic recommendations
  • The budget impact of AML is currently small, but is likely to increase following further approvals
  • Payers currently have a relatively passive approach to AML
  • Moderate levels of restriction are imposed on marketed AML medicines Vidaza and Dacogen
  • AML treatment is becoming increasingly personalized
  • Combinations are likely to be the future of AML
  • Efficacy trumps route of administration in AML due to high unmet need
  • Recently approved and pipeline AML therapies are likely to seek multiple label expansions, resulting in subsequent price decreases
  • Bibliography

ACCESS TO RECENTLY LAUNCHED AND PIPELINE PRODUCTS

  • Rydapt
  • Gilteritinib and quizartinib
  • Vyxeos
  • Guadecitabine and Venclexta
  • Rydapt has the potential to gain positive pricing and reimbursement recommendations, but EU payers might restrict by age
  • Second-generation FLT3 inhibitors could improve clinical outcomes; if not, price will be an important factor
  • Vyxeos likely to be impacted by different funding mechanisms
  • Vyxeos likely to benefit from more favorable infusion schedule
  • Generic Vidaza will set the price benchmark in Europe for certain pipeline therapies
  • Bibliography

CLINICAL TRIAL DESIGN AND EVIDENTIARY REQUIREMENTS

  • Insights and strategic recommendations
  • OS is considered the most important endpoint, but PFS is sometimes sufficient
  • Threshold for OS improvement varies with line of therapy
  • HRQoL data is important, yet rarely collected
  • Age may not be the best predictor of treatment eligibility
  • "Investigator choice" is accepted but needs to reflect SoC in each market
  • Improvements in complete remission rate are important as they indicate an increased eligibility for stem cell transplants
  • Bibliography

US PRICING

US REIMBURSEMENT

  • Insights and strategic recommendations
  • There are minimal controls for AML therapies
  • Uptake will largely be dictated by specialist physicians
  • New oral AML therapies found in tier 3 of most commercial formularies
  • Prior authorization generally follows FDA labels for recently approved AML therapies
  • Bibliography

JAPAN

  • Price premiums are awarded for added benefit or innovation
  • Pricing of launched AML treatments
  • Bibliography

PRICING IN THE FIVE MAJOR EU MARKETS

FRANCE

  • Insights and strategic recommendations
  • ASMR rating has an impact on pricing
  • Dacogen is not included on the "liste-en-sus" and is unlikely to be routinely reimbursed in the hospital setting
  • Vidaza reimbursed only for those with 20-30% blasts
  • Exclusion from the liste-en-sus will likely be a cost-containment measure for new hospital-administered drugs
  • Oral AML therapies will be fully reimbursed irrespective of ASMR rating
  • Relapsed and refractory AML patients are considered to have the highest unmet need
  • Mutationally targeted therapies may be able to achieve higher prices in negotiations with the CEPS
  • Vyxeos could gain an ASMR III in France, and be used alongside chemotherapy add-ons that are expected to gain approval
  • Bibliography

GERMANY

  • Insights and strategic recommendations
  • Positive assessment from the G-BA will impact price negotiations
  • Recently approved and pipeline orphan therapies will be given an automatic additional benefit rating from the G-BA
  • Without mature Phase III data, orphan drugs are likely to receive "no additional benefit" at the second G-BA assessment
  • Dacogen received a minor additional benefit rating due to unclear OS data
  • Price negotiations after the benefit assessment are the main levers for sickness funds to control expenditure on AML drugs
  • Cost-containment tools for AML may be implemented as the market gets more crowded
  • Label expansions to wider patient populations will result in multiple G-BA assessments and pricing negotiations
  • Unmet need is not considered in an added benefit assessment by the G-BA
  • Mutationally targeted therapies will not be viewed more favorably by the G-BA, and reimbursement of genetic tests may be an issue
  • Expensive AML medications will require an NUB to gain funding through the hospital system
  • Vyxeos is unlikely to be assessed by the G-BA, and access will depend heavily on price
  • Safety issues are likely to limit IDHIFA's uptake in the German market
  • Bibliography

ITALY

  • Insights and strategic recommendations
  • All marketed AML treatments are reimbursed in Italy
  • AML drugs assessed and reimbursed by AIFA are found in regional formularies investigated by Datamonitor Healthcare
  • Some Italian regions impose further restrictions
  • Gaining a therapeutic innovation rating will be highly beneficial for new AML medications
  • Rydapt has the potential to receive "innovative" or "conditionally innovative" drug status
  • Bibliography

SPAIN

  • Insights and strategic recommendations
  • National reimbursement decision is not a major access barrier in Spain
  • Regional and local bodies give more stringent reimbursement recommendations
  • Restrictions from local authorities often have the greatest impact
  • Dacogen received a broad recommendation in its IPT
  • Recommendations for Dacogen and Vidaza differ regionally and locally
  • Drugs targeting the relapsed/refractory setting may gain easier access
  • Label expansions will result in price decreases in Spain
  • Mutationally targeted therapies are likely to be assessed more favorably in Spain
  • Early access can be granted in Spain for one year, based on provisional data
  • Vyxeos is expected to be recommended at a national level, but pricing and local recommendations will be the most important
  • Bibliography

UK

  • Insights and strategic recommendations
  • NICE and SMC determinations drive reimbursement decisions
  • NICE and SMC recommend Vidaza for patients with 20-30% blasts only
  • Vidaza required PAS for acceptance by NICE and SMC
  • Dacogen is not available on the UK or Scottish healthcare system
  • NICE committee fails to recommend Rydapt for reimbursement
  • Cancer Drugs Fund redesigned for goal of early access to novel drugs
  • Payers are likely to be more "reactive" rather than "proactive"
  • Treatment algorithms will likely have an important impact on the uptake of AML therapies in the future
  • Bibliography

APPENDIX

  • Primary research
  • Price assumptions
  • Exchange rates
  • Bibliography

LIST OF FIGURES

  • Figure 1: Price sources and calculations, by country

LIST OF TABLES

  • Table 1: Marketed products and approved indications for acute myeloid leukemia in the US, Japan, and five major EU markets
  • Table 2: Factors influencing the pricing and reimbursement of Vidaza and Dacogen in the US and five major EU markets
  • Table 3: Pricing and reimbursement of mutationally targeted therapies in the US and five major EU markets
  • Table 4: Investigated populations for recently approved and pipeline AML therapies
  • Table 5: The impact of in-patient funding mechanisms on Vyxeos's use
  • Table 6: US pricing of key intravenous AML drugs
  • Table 7: US pricing of key oral AML drugs
  • Table 8: Formulary placement of marketed AML drugs in selected commercial formularies
  • Table 9: Formulary placement of marketed AML drugs in select Medicare Part D formularies
  • Table 10: Prior authorization criteria for marketed AML drugs in six major health plans
  • Table 11: Pricing premiums given to medicines that can demonstrate benefit over comparators
  • Table 12: Pricing of key marketed intravenous AML drugs in Japan
  • Table 13: Pricing of key AML drugs in the five major EU markets
  • Table 14: Transparency Committee's ASMR ratings and pricing implications
  • Table 15: Transparency Committee's SMR ratings and pricing implications
  • Table 16: Transparency Commission's assessment of AML treatments
  • Table 17: G-BA assessments of key AML drugs
  • Table 18: Reimbursement conditions for AML treatments in Italy
  • Table 19: Italian regional formulary decisions for AML drugs
  • Table 20: AML drug assessments by the Emilia-Romagna region
  • Table 21: AIFA Technical Scientific Committee innovation algorithm
  • Table 22: National and regional reimbursement decisions in Spain
  • Table 23: NICE assessments of key AML therapies
  • Table 24: SMC decisions on key AML therapies
  • Table 25: Exchange rates used for calculating branded drug prices
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