Acute Myeloid Leukemia Pricing, Reimbursement, and Access

  • ID: 4462201
  • Report
  • 132 pages
  • Datamonitor Healthcare
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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.
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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
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

List of Figures
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
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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