HTA decisions in view of timely access

Anke Hövels, PhDNov 15th 2017

My presentation

• Evidence vs politics in decision making

• What impacts HTA?

• HTA-regulatory interactions

• Possible solutions for early access

Components of a Medical Decision

Assessmentcomponent Appraisalcomponent

The funnel of Dunning

Essential care

Efficacy

Cost effectiveness

Own account and

responsibility

Basicpackage

Broad solidarityBroad solidarity

Limited access Limited access

1thcriterium

2ndcriterium

3thcriterium

4th

criterium

After drug development

RegulatoryMarket Authorisation

Pricing and Reimbursem

ent

EfficacySafetyQuality

Unmet medical need,

Relative effectiveness

Cost-effectivenessBudget impact

(national)

Bridging the “Value Gap”

Short-term DataSurrogate endpointsLimited ComparatorsLow Response Rate

Broad population

What R&D Often Provided

Evidence of meaningful benefit on

survival/qol overrelevant comparators

in a select well-definedpopulation at a

justifiableprice

What Customers Want

The Value Gap

Bridge: the Reimbursable File

Strong evidence package needed

Different countries, different outcomes

England(NICE)

France(HAS)

Germany(IQWiG)

Netherlands(ZIN)

Poland(AOTMiT

)

Scotland(SMC)

52

27

214

9 40

11

1830

523

9

34

27

26

3339

28

24

50

56=100%

98=100%

50=100%

93=100%

78=100%

95=100%

Jurisdiction

HTAoutcomes- All

79

12

9

38 4

9

13

2228

503

0

224

8

37

42 4

8

10

37

26

27=100%

37=100%

19=100%

32=100%

31=100%

34=100%

HTAoutcomes-Cancer

HTAoutcomes:allvs.cancervs.non-cancerdrugs98drugswithHTAsavailablefor≥4jurisdictions*,EMAapprovalyears2007– 2013,6jurisdictions

36

36

285

6 34

10

1631

534

8

45

719

2832

401

665

29=100%

61=100%

31=100%

61=100%

47=100%

61=100%

HTAoutcomes– non-Cancer

England/Wales:Seemsmorerestrictiveinevaluatingcancerthananti-cancerdrugs.ThisisduetothefactthatmanycancerdrugsareavailablethroughtheCancerDrugFund.

Sources:EMA,NICE,HAS,IQWiG,ZIN,AOTMiT,SMC,author’sanalysis

*https://www.england.nhs.uk/ourwork/cancer/cdf/

Scotland:SMC’sapproachisrestrictiveas79%ofcancerdrugshavereceivednegativerecommendations.Somecancerdrugsmaybemadeavailabletopatientsthrough„IndividualPatientTreatmentRequests”.

GermanyandFrance:Moregenerousinrecommendingcancerthannon-cancertherapies.

Regulatory-HTA

• No difference betweenstandard marketing approval andconditional marketing approval.

• Is there any overlap?• How is evidence

considered?

Where is time spent?

Where is time spent? Agency specific?

Solution? Standardisation?

• EuNetHTA– European wide assessment– Interaction with EMA

• Good predictive modelling

• Appropriate use of real world data

EURegulatoryProcess WP4HTAProcess Stakeholderinvolvement

100

-180

-90

0

35

85

75

EMAProcess

CHMPopinion

PositivedecisionofEC

EPAR

Co-productionof1stversionofREA

2ndversionofREAIncludingeditorialreview

ExpressionofinterestfrompMAH

PreparationofdraftsubmissionfilefrompMAH

Developmentdraftprojectplan

ScopingmeetingwithpMAH

Finalizationofprojectplan

Receivefinalsubmissionfile

Consultation

FinalversionofREA

LocalREA’s(e.g.national,regional)

Timeline(days)

Reviewprojectplanbyclinicalexperts

involvementofpatients

Identificationofclinicalexpertsandpatients

MAHprovidesevidencefile

ReviewbyexternalexpertsandfactcheckbyMAH

Thank you!