Strategies regarding new high cost biological medicines ...€¦ · 1 | TITLE from VIEW and SLIDE...

TITLE from VIEW and SLIDE MASTER | 26 October 2016 1 |

Strategies regarding new high cost biological

medicines when developing formularies across the Interface

Interface Management of Pharmacotherapy

Barcelona – 17th October 2016

NICOLA MAGRINI

ESSENTIAL MEDICINES AND HEALTH PRODUCTS DEPARTMENT

TITLE from VIEW and SLIDE MASTER | 26 October 2016 2 |

Strategies on the selection of high priced medicines

NICOLA MAGRINI

POLICY, ACCESS AND USE ESSENTIAL MEDICINES AND HEALTH PRODUCTS DEPARTMENT

What I’ll speak about

HIS/EMP | Communications Planning HIS | EMP

Contents of the presentation

• WHO EML: a quick history • EML 2015 update and high priced medicines • The WHO strategy on

– Pharmacological class, therapeutic equivalency (EML square box) – Generics and biosimilars – Other options

First EML: 1977 36 pages, 20 references

EML 1977: early evidence-based adopter

No medicines for: • Memory loss and dementia • Hepatoprotectants • Immunostimulants

• No medicines listed subsequently

withdrawn for unexpected risks (e.g., cox-2 inhibitors)

• Important medicines for: – Antibiotics – Neglected diseases – Pain – Mental health – Chronic diseases – Cancer

• Concise and clear • Promoting uptake of best

research findings on medicines into healthcare and national policies

Essential Medicines List: Concept and Procedures 6 |

Essential Medicines

Guiding principle: A limited range of carefully selected medicines leads to better health care, better medicines management, and lower costs

Definition: Essential medicines are those that satisfy the priority health care needs of the population

Selection: Selected with due regard to disease prevalence, evidence on efficacy and safety, and comparative cost-effectiveness


EML criteria (EB 109/8, 2001)

Disease burden and public health need/relevance

Sound and adequate data on the efficacy (on relevant outcomes), safety and comparative cost-effectiveness

– “Absolute cost of the treatment will not constitute a reason to exclude a medicine from the Model List that otherwise meets the stated selected criteria”

– “Affordability changed from a precondition into a consequence of the selection” (Hogerzeil, BMJ, 2004)

WHO responsible management and oversight of CoIs

2008 WHO new Guideline Manual, adopting GRADE

Considering also: regulatory status / availability, WHO guidelines


19th EML & 5th EMLc - 2015

19th EML: 409 medicines – 5th EMLc (children): 294 medicines

586 pages, 1082 references


EML 2015: tough decisions


EML 2015: 77 applications and a few big challenges Cancer drugs: a large comprehensive review led to 16 new

additions (including 2 mabs and imatinib)

New highly effective HCV drugs (all listed)

MDR-TB drugs (4) and 1 for TB prophylaxis

Rejections: New oral anticoagulants (NOACs), polypill(s), ranibizumab


Lancet editorial October 24, 2015




– Pharmacological class, therapeutic equivalency (EML square box) – Generics and biosimilars – Other options


EML and patented drugs

EML 2013: 20 out of 374 (5.4%)

EML 2015: 39 out of 409 (9.5%)

A trend?

Biosimilars: filgrastim, trastuzumab, rituximab

Next EML 2017?

Objectives of phases I, II and III in drug registration : I: safety II: activity III: efficacy/effectiveness or B/R ratio

Drug registration and patent’s life

Patent expiry

Drug’s life and life cycle well beyond patent: the next 20 years

Medicines’ life: longer than the patent

Life after patent expiry


EML cancer medicines 2015 main criterion: magnitude of absolute benefits

Imatinib: vast majority of patients in remission at 7 yrs

Rituximab (large B cell lymphomas): 15% absolute increase in survival rates (from 50-55% to 70%)

Trastuzumab: early stage breast cancer: up to 13% increase in survival in high risk women (from 37% to 50% survival rates at 3-6 yrs)

Bendamustine, ATRA, …

Efficacy estimates applied to all proposed regimens


Imatinib generic prices 2016

Imatinib prices 2013 - 2016

700 USD

EML and pharmacological classes

Listing: one drug per pharmacological class … implications for selection and procurement


EML and therapeutic equivalency 1/3


Example 1


Example 2 : limited square boxing


Example 3: limited square boxing



WHO supports pooled procurement mechanisms that can increase competition among available alternatives/options

Generic substitution and biosimilars role




– Pharmacological class, therapeutic equivalency (EML square box) and tenders

– Generics and biosimilars – Other options

• ¨Conclusions


Biosimilars: regulations still in progress

Recent documents


Biosimilars: no official WHO statement yet

INN, Biological standardisation and EML will be working together to define a WHO position

– Important implications on regulatory pathways, Therapeutic Committees and procurement

– Timeline: early (1st quarter) 2017?






• ¨Conclusions

Public health, innovation and intellectual property 36 |

Policy options

Price controls: ‘Value-based pricing; price/volume agreements; pay for performance; risk-sharing agreement / patient access schemes; reference pricing schemes…

Differential pricing/price negotiations

Voluntary licensing agreements

Local production/import

TRIPS flexibilities, including compulsory licenses

The Un High-level Panel on Access to Medicines

On 14th September 2015

What’s in the UNHLP Report on access to medicines

• The report may spark controversy: • six panel members offered separate commentaries that either

criticized the panel for its timidity or chastised it for focusing too much on IP rights and not being fact driven.

• Some Member States have also voiced concerns about the process before the release of the report.

• Thus, it remains to be seen whether it will be discussed in the UN General Assembly, the WHO or another UN body.

What’s in the UNHLP Report on access to medicines

• The report mentions positively a number of ongoing WHO initiatives – the WHO/DNDI Global Antibiotic R&D Partnership, – the (future) WHO/TDR voluntary pooled funding mechanism for R&D, – the R&D Blueprint.

• Some of the technical recommendations—in particular, the IP-related issues and the establishment of a database on medicine prices—are already within WHO’s mandate

the UNHLP Report on access to medicines What’s new for WHO

• a database on medicine prices • Other recommendations would need endorsement by

member states, such as – the negotiation of international agreements – the SG’s establishment of an interagency task force.






• ¨Conclusions


Conclusions EML, access and affordability

• Growing attention to the issue of access and affordability

• EML can play a role – Strong collaboration with MPP, – Fair pricing forum, – EML discussing thresholds for benefits – Biosimlars WHO GL in 2017

• together with all other stakeholders


Questions? All questions are welcome

all questions can be useful to understand EML role and what I just presented

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