Recognising Value and Rewarding
Innovation in Rare Diseases
EUCAN Policy Position
Key messages
01 02
As pharmaceutical companies Takeda advocates for
face several challenges in the governments to continue to
development of rare disease provide R&D incentives which
treatments, R&D incentives are are appropriate and
key to continue to promote sustainable, including R&D tax
investment in these areas, credits, to reflect the significant
where treatments are still investment in time and
scarce. resources rare disease treatment
development requires.
What is the issue?
• Pharmaceutical companies face enormous challenges in
the research and development (R&D) of rare disease
treatments, also known as orphan drugs.1 These
challenges include the complex nature of the science,2
high development costs,3, 4 difficulty in recruiting patients
for clinical trials5 and low patient numbers who can
benefit from the drug.6 For detailed information on these
challenges please refer to the EUCAN Policy Position on
Pricing and Rare Diseases.
• These challenges have led governments to introduce
incentives to encourage companies to invest in R&D:
– The EU introduced the Orphan Medicinal Products
(OMP) Regulation which provides the following set of
incentives: 5, 7, 8
– Funding for R&D
– Assistance and reduction of fees in the market
access application process
– Access to a centralised authorisation procedure
in the EU, with a single application, evaluation,
and authorisation across the EU
– Ten years of market exclusivity across EU Member
States
– Inviting Member States to provide tax benefits for
manufacturers, however this is yet to be widely
adopted
What is Takeda’s position on the issue?
• At Takeda, we advocate for the following changes to R&D
incentives for rare diseases:
– Evolving the current incentive framework by
maintaining current orphan designation thresholds
whilst allowing for recalibration of incentives:
increasing incentives in areas that have so far attracted
insufficient investment, and recalibrating incentives in
well-defined areas where this will not have an impact
on the progress of standard of care.13
– Continuing to provide R&D incentives which:10
– Are appropriate and sustainable, to reflect the
significant investment in time and resources
OMP development requires.
What is Takeda already doing about the issue?
03
We support the development
of incentive frameworks that
look holistically at the entire
drug development pathway
and that increase investment
in areas that have attracted
insufficient investment.
– Canada adopted two mechanisms: 9, 10
– Data protection: protection of undisclosed data
relevant to the safety and efficacy of an innovative
drug, with a guaranteed minimum of eight years of
market exclusivity. An additional period of six months
of market exclusivity is granted for paediatric drugs.
– Patent term extension: the Certificate of
Supplementary Protection (CSP) regime provides up to
two additional years of patent-like protection for drugs
protected by an eligible patent.
• In the EU, it is estimated that, since introduced, these
incentives have stimulated an increase in the
development of OMPs in the range of 51% to 94%.11
• While these incentives, together with EU member state
commitment to pay for rare disease drugs, have increased
the expected return on investment for drug development,
95% of rare diseases still lack a licensed treatment.12
• The OMP Regulation, for example, created two challenges: 10
– it does not provide incentives at all stages along the
OMP lifecycle, including in addressing the lack of basic
research that prevents OMP development for some rare
diseases;
– incentives along the drug development path are not fully
aligned and sometimes even work against each other.
– Take a modulated approach, by using indicators
that reflect a strong or weak case for investment,
with a policy that moves away from one-size fits
all.
– Set up a public-private partnership for basic research
funding in underserved areas of rare diseases: as
the lack of basic research is a key barrier to the
development of innovative therapies.10
– Introduce R&D tax credits: as these could allow
OMP developers to save costs invested in intense
R&D activity.
• Centre of Excellence for Innovation and Evidence • Shonan Health Innovation Park: In 2018, Takeda
Generation in Sweden: Takeda is collaborating broadly transformed its Shonan Research Centre in Japan.
with partners across the region and beyond to create Bringing researchers, industry, venture start-ups,
cutting-edge innovations beyond the medicines. government and academia together to form a true
• Innovating for people living with a rare disease: Why
partnerships are key for the European OMP ecosystem
report: Takeda commissioned a report which assesses
co-location ecosystem for collaboration and co-
creation, resulting in the incubation and acceleration
of research and transformation of cutting-edge
science into impactful health solutions for patients
how barriers to innovation in OMP development can be
tackled and provides several recommendations, around the world.
including for better incentive adoption by the European
OMP Regulation.
What do other stakeholders say about the issue?
• Canadian government: has adopted two mechanisms, • European Expert Group on Orphan Drug Incentives:
data protection and patent term extension, aimed at authored a report outlining 14 policy proposals that aim
incentivising manufacturers to continue their investment to improve the rare disease incentive framework.
– detailed in the ‘What is the issue?’ section.9, 10 These include:9
• EFPIA: Advocates for continuous collaboration – Forming a rare disease Public Private Partnership fund
between all stakeholders to foster an innovation- for research and early development,
oriented ecosystem for rare diseases. It highlights – Providing guidance and incentives for translational
the need to:14 basic research,
– continue to provide R&D incentives, – Introducing additional financial incentives, such
– not reduce the market exclusivity time, as a transferable voucher or tax credits for drug
– develop additional incentives for certain treatments, development,
– avoid promoting faster generic competition, – Creating a common EU value assessment for rare
– avoid capping the number of patients that can disease medicines.
benefit from a designated orphan drug. • European Joint Programme for Rare Diseases: running
• EMA: Recognises the important role that incentives until 2023, leads the most systematic and coordinated
play in the development of orphan drugs and offers funding efforts for rare diseases basic research in Europe.18
several incentives for medicines that have been • Innovative Medicines Initiative: is an EU-wide Public
granted an orphan designation by the European Private Partnership with the primary objective of speeding
Commission – detailed in the ‘What is the issue?’ up the development of, and the access to, innovative
section.15 medicines – particularly in areas of high unmet need.19
• European Commission: understands the difficulty in • US Orphan Drug Tax Credit: although not in place in the
accessing high quality care and treatments for rare EUCAN region, the ODTC demonstrates how a country
diseases and has dedicated EU-wide funding has decided to promote research spending on OMP
through Horizon 2020 and the previous Seventh development by granting developers a 25% tax credit of
Framework Programme to aid rare disease research clinical trial costs for OMPs.20
in Europe.16, 17
Where can I find out more?
• Copenhagen Economics. Innovating for people living with a rare disease: Why partnerships are key for
the European OMP ecosystem – available here (paper commissioned by Takeda).
• Takeda. The value of immediate access for rare disease treatments – available here
• Takeda. EUCAN Position Narrative – available here
• Takeda. Policy Objections Handler – available here
References
1 EFPIA. Addressing unmet needs in extremely rare and paediatric-onset diseases: how the biopharmaceutical innovation model can help identify current
issues and find potential solutions. 2021. 2 Swinney DC & Xia S. The discovery of medicines for rare diseases. 2014. Future Med Chem. 6(9):987-1002. 3
Wouters OJ. et al. Estimated Research and Development Investment Needed to Bring a New Medicine to Market, 2009-2018. JAMA. 2020. 4 Augustine EF.
et al. Clinical trials in rare disease: challenges and opportunities. J Child Neurol. 2013; 28(9):1142-50. 5 EMA. Centralised procedure. Available here [Last
accessed Aug 2022] 6 Gahl WA. et al. Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment
Access Working Group. 2021. Orphanet Journal for Rare Diseases. 16, 308. 7 Berthelot S. et al. Rising to the Challenges of Developing Rare Disease
Treatments. Global Forum. Available here [Last accessed Aug 2022] 8 Gamba S. et al. R&D and market size: who benefits from orphan drug regulation?.
Journal of Health Economics. 2021. 9 Nicola, KJ. The Pharmaceutical Intellectual Property and Competition Law Review: Canada. 2021. Available here [Last
accessed Aug 2022] 10 McMahon E. et al. Orphan Drugs in Canada: A “One Size Fits All” Regulatory Regime. Food and Law Institute. Available here [Last
accessed Aug 2022] 11 Dolon. Estimated impact of EU Orphan Regulation on incentives for innovation. 2020. 12 European Expert Group for Orphan Drug
Incentives. Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product
Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives. Frontiers in Pharmacology; 12. 2021. 13
Copenhagen Economics. Innovating for people living with a rare disease: Why partnerships are key for the European OMP ecosystem. 2021. 14 EFPIA. 2021.
EFPIA response to the European Commission’s consultation on the revision of EU rules on medicines for children and rare diseases. Available here [Last
accessed Aug 2022] 15 EMA. Orphan Incentives. Available here [Last accessed Aug 2022] 16 European Commission. Expanding research into rare diseases.
Available here [Last accessed Aug 2022] 17 Cordis Europa. Helping the 5 in 10 000: Innovative therapies against rare diseases. Available here [Last
accessed Aug 2022] 18 European Joint Programme for Rare Diseases. Project structure. Available here [Last accessed Aug 2022] 19 IMI. IMI Mission and
objectives. Available here [Last accessed Aug 2022] 20 Investopedia. Orphan Drug Credit. 2021. Available here [Last accessed Aug 2022]