The document summarizes a comparative analysis of access to orphan medicinal products (OMPs) in the UK, France, Germany, Italy, and Spain. It finds that OMPs have the widest availability in Germany and Italy, where Germany automatically reimburses all authorized OMPs and around 60% are reimbursed in Italy. Germany and France also provide the broadest access overall. The UK mechanisms provide access to less than 50% of authorized OMPs, while Germany provides the quickest access. On average, it takes around 24 months for countries to grant access, but times vary between countries.

1. Access to Orphan Drugs in the UK and
other European Countries
B. Zamora, M. Garau, F. Maignen, P. O’Neill, J. Mestre-Ferrandiz
XIV HTAi 2017 Annual Meeting
Rome, Italy
17th-21st June 2017

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Outline
• Background and objectives
• Methods
• Results:
 Orphan designations and marketing
authorisations
 Comparison of access among countries
• Limitations
• Summary

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Objectives
• To perform a comparative analysis of access to
OMPs in the UK (including the devolved
nations) and in four other EU countries (France,
Germany, Italy, Spain)
• To assess the speed of access of OMPs to
patients in these countries

4. Methods

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Methods
• Data from the European Medicines Agency’s (EMA) and
DG Health and Food Safety’s websites on medicinal
products with an orphan designation and marketing
authorisation
• Time period covered from 2000 to June 2016, i.e. from
inception of Regulation (EC) No 141/2000 on orphan
drugs, to date
• Orphan indications with marketing authorisation were
matched with an HTA decision, a “commissioning”
decision, and/or a reimbursement decision
• We developed a protocol to ensure a systematic and
consistent approach to data extraction for the UK HTA
systems and the other four EU countries

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Methods: Working definitions
• Availability:
• Access:
Early access before the marketing authorisation
Launch
− Positive HTA (recommended or optimised),
commissioning, or reimbursement
− Decision leading to use within national NHS

7. Results

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Orphan Designations and Marketing
Authorisations

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Availability and access to OMPs in
the UK
(*) For these 143 OMPs, 145 different indications were appraised by NICE in England (split
indications for sunitinib and nilotinib).
Reported percentages for England were calculated over 145 indications.

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Availability and access to OMPs used
in oncology in the UK
England Scotland Wales
Number of Oncology OMPs centrally authorised 56(*) 56 56
#Oncology OMPs with a decision on use 49 45 42
% of Oncology OMPs centrally authorised 84.50% 80.35% 75%
#Oncology OMPs with HTA Appraisals 39 45 42
% of Oncology OMPs centrally authorised 67.20% 80.35% 75%
#Oncology OMPs Reimbursed, of which 33 26 21
# with HTA Decision Recommended 9 19 9
# with HTA Decision Optimised 9 7 12
# with NHS England Commissioning 2
# included in Cancer Drugs Fund 13
% of Oncology OMPs centrally authorised 56.90% 46.40% 37.50%
(*) For these 56 OMPs, 58 different indications were appraised by NICE in England (split
indications for sunitinib and nilotinib).
Reported percentage for England were calculated over 58 indications.

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Availability and access to other (non-
oncology) OMPs in the UK
England Scotland Wales
Number of non-oncology OMPs centrally authorised 87 87 87
#Non-oncology OMPs with a decision on use 71 51 42
% of non-oncology OMPs centrally authorised 81.60% 58.60% 48.30%
#Non-oncology OMPs with HTA Appraisals 14 51 42
% of non-oncology OMPs centrally authorised 16.10% 58.60% 48.30%
#Non-oncology OMPs Reimbursed, of which 35 29 26
# with HTA Decision Recommended 2 15 19
# with HTA Decision Optimised 3 14 7
# with NHS England Commissioning 30
% of OMPs centrally authorised 40.20% 33.30% 29.90%

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Time access to OMPs and non-OMPs
in the UK
• Time used to grant access to OMPs in the UK
• Time to NICE decision for orphan and non-
orphan drugs

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France
• Availability and access to OMPs in France
• Time used to grant access to OMPs in France

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Germany
• Availability and access to OMPs in Germany
• Time used to grant access to OMPs in Germany

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Italy
• Availability and access to OMPs in Italy
• Time used to grant access to OMPs in Italy

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Spain
• Availability and access to OMPs in Spain
• Time used to grant access to OMPs in Spain

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Comparison among countries

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Comparison among countries

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Comparison among countries

20. Limitations

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Limitations of the analysis
• There are a number of mechanisms other than HTA approvals by which
patients can access market authorised OMPs. These include clinical trials or
compassionate programmes funded by the sponsors/marketing authorisation
holders, individual patient funding requests (e.g. in England, Scotland, and Wales)
• Our data do not capture those alternative routes and so might underestimate
the number of OMPs available to patients
• On the other hand, HTA positive recommendations might not necessarily
lead to a fast(er) use of a new intervention in practice (e.g. in England). In this
sense our analysis might overestimate the number of OMPs actually available
• The analysis is based on publicly available information. In some instances
(such as Spain), managed entry agreements are negotiated at regional level.
Little public information is available on these agreements.
• Time elapsed between EU marketing authorisation, marketing
authorisation issued by each of the countries included in our analysis
and company launch in individual countries were not captured
 There was variation in the way data was captured due to different regulation
and sources

22. Summary

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Summary of comparative findings
• Among the countries included in the study, OMPs are most widely available in
Germany and Italy
 Although in Italy 60% of centrally authorised OMPs are reimbursed
• Germany and France provide the broadest access to OMPs
 Germany automatically reimburses all authorised OMPs
 In France OMPs are generally fully reimbursed via the ALD mechanism. Therefore, the actual
proportion of products reimbursement of OMPs could be close to 100%
• In England mechanisms providing access are diverse and cover less than 50%
of centrally authorised OMPs
 Only one-third of OMPs routinely funded by the NHS received positive decision by NICE
• Germany is the country providing the quickest access (by automatically
reimbursing centrally authorised OMPs)
• Italy and France provide access (reimbursement) in time intervals below our
countries average (which is almost 24.4 months excluding Germany)
• England and Wales are above average (27.6 and 29.3 respectively), while
Scotland is below (22.6 months)

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References and data sources
• European Medicines Agency and DG Health and Food Safety (community registers
of orphan medicinal products for human use and medicinal products for human
use – accessed on 20 September 2016)
• United Kingdom: NICE (National Institute for Health and Care Excellence), NHS
England, SMC (Scottish Medicines Consortium), AWMSG (All Wales Medicines
Strategy Group) websites
• France: Base de données Publique des Médicaments et base de données Vidal des
médicaments, Haute Autorité de la Santé (HAS), Official Journal and Agence
Nationale de Securité des Médicaments (ANSM). Information accessed between
16-20 September 2016.
• Germany: Institute for Quality and Efficiency in Health care (IQWiG), Federal Joint
Committee (G-BA), and Lauer-Taxe database: https://www.lauer-
fischer.de/LF/Seiten/Verwaltung/Kundencenter/1.aspx
• Italy: Agenzia Italiana del Farmaco (AIFA)
• Spain: Consejo General de Colegios Oficiales de Farmacéuticos
(https://botplusweb.portalfarma.com/)

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This study was commissioned and funded by Shire, and managed by
Hanover Communications on behalf of Shire.
To enquire about additional information and analyses, please contact
Bernarda Zamora at bzamora@ohe.org
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