Takeda Pharmaceutical Co. Ltd. · TSE:4502
Takeda Pharmaceutical — Nordic Rare Diseases Summit 2021
39 slides from Takeda Pharmaceutical's conference presentation, Apr 13, 2021
Slide 1: European Recommendations for Improving Overall Access to Medicines for Patients with Rare Diseases
EUROPEAN RECOMMENDATIONS FOR IMPROVING OVERALL ACCESS TO MEDICINES FOR PATIENTS WITH RARE DISEASES ACROSS THE EU Nordic Rare Diseases Summit 2021, April 13th Yann Le Cam, Chief Executive Officer, EURORDIS C-ANPROM/DK/TAKH/0014
Slide 2: Unmet needs as perceived by people living with a rare disease
Unmet needs as perceived by people living with a rare disease? 31% have never experienced 69% have already experienced a any treatment treatment Curative Preventative treatment 5% treatment 3% Slow down treatment 31% Symptomatic treatment 61% 2
Slide 3: Challenges
Challenges Regulatory not optimum; HTA Product Availability High Unmet Medical and Patient Access is Eco-system to fragmented and Needs; development slow, unequal, poor. translate science and diverging; P&R non clustered on some technology into adapted to low Affordability and diseases; very rare he
Slide 4: Rare 2030 Recommendation for Available, Accessible and Affordable Treatments
Rare 2030 Recommendation for Available, Accessible and Affordable Treatments Establish streamlined regulatory, pricing and reimbursement policies. These policies should encourage a continuum of evidence generation along the full life cycle of a product or technology as well as the patient journey fr
Slide 5: Specifically recommends…
Specifically recommends… a threshold of eligibility that includes early-stage multi-stakeholder a graduated system of incentives, incidence in addition to prevalence of identification of unmet needs and rewarding earliest dialogue and areas 5/10000 individuals and avoids artificial subsequent priori
Slide 6: With measurable goals
With measurable goals More and better quality curative, stabilising, palliative, assistive, rehabilitative and preventive technologies and therapies available, accessible and affordable A European competitive ecosystem in the development of RD therapies and a more robust pharma and biotech manufactu
Slide 7: EURORDIS suggested approach (2018)
EURORDIS suggested approach (2018) The ambition of EURORDIS is to have 3 to 5 times more new rare disease therapies approved per year, 3 to 5 times cheaper than today by 2025 A structured approach to market access in Europe Structured voluntary cooperation between healthcare systems in the European
Slide 8: The (policy) opportunity
The (policy) opportunity A strategic, coordinated approach to development & access to treatment to rare diseases in Europe reconciling unmet needs and innovation Source: European Commission 8
Slide 9: A word about COVID
EURORDIS EURORDIS.ORG
Slide 10: Some learning lessons
Some learning lessons Massive use of data Regulatory flexibility Collaboration before Maturing of genome and computing to (namely, rolling data competition sequencing system identify suitable review) candidate Mature scientific Growing acceptance Procurement to knowledge Investments (public, of othe
Slide 11: Thank you
gh * ow ee We EvRORDIS RARE DISEASES EUROPE THANK YOU EURORDIS.ORG
Slide 12: How the Nordic countries align with the European ORPH-VAL principles
How the Nordic countries align with the European ORPH-VAL principles for assessing medicines for rare diseases Lieven Annemans Ghent University, Brussels University (VUB) Copenhagen (virtually) April 13 2021 Lieven.annemans@ugent.be https://www.linkedin.com/in/lieven-annemans-3376b46/ C-ANPROM/DK/TA
Slide 13: Content
Content • Values, Value and Value for Money in Healthcare • How valuable is the value assessment in the highly valued Nordics? • Takeaways
Slide 14: No-one should be left behind
14 No-one should be left behind ✓ “Ensuring universal access to quality care demands greater efforts to improve the effectiveness, accessibility and resilience of health systems in all EU countries.” ✓ “More should be done to improve the health of populations in EU countries and, in particular, to r
Slide 15: The aim of healthcare policy is to optimize the health of the population
“The aim of healthcare policy is to optimize the health of the population within the limits of the available resources, and within an ethical framework built on equity and solidarity principles.” EU Council of Ministers of Health Dec 2010, based 15 on the text of the Belgian Presidency
Slide 16: It's about adding value
It’s about adding value OXFORD DICTIONARY
Slide 17: What is the value of an orphan medicine?
What is the value of an orphan medicine?
Slide 18: Guide to core elements of value
Guide to core elements of value Value of an orphan medicine Impact of DISEASE on Impact of TREATMENT on Survival/life expectancy; Morbidity Patient experience and quality of life (QALYs) Patient level Patient economic burden Healthcare system Healthcare system resources and budget level Healthcare s
Slide 19: Value for Money
Value for Money Cost NOT C-Eff New Current C-Eff New care New Dominant Health effect (QALYs) Annemans L. Health Economics for non-Economists. Pelckmans Pro 2017
Slide 20: Single Threshold? No way!
Single Threshold? No way! Bull World Health Organ 2016;94:925–930 Cost–effectiveness information should be used alongside other considerations in a transparent decision-making process, rather than in isolation based on a single threshold value.
Slide 21: A key challenge for OMPs: Evidence Gaps
A key challenge for OMPs: Evidence Gaps “Give us more evidence that your medicine is PAYER value for money compared to the current SoC” INDUSTRY “Allow us first to the market (reimburse the medicine) and then we will be able to show real life evidence”
Slide 22: Types of Evidence Gaps
Types of Evidence Gaps Treatment related uncertainties - Exact magnitude of treatment effect, variability in effect - Long term effect - Adverse events and safety Disease related uncertainties - Natural course of the disease - Relation between surrogate and hard endpoint - Incidence and prevalence H
Slide 23: The need for adaptive processes
The need for adaptive processes Revise Reimbursement Real world time Reimbursed/ Point of Verification Launch Pay Back
Slide 24: The Need for Iterative Dialogues
The Need for Iterative Dialogues 24 TRUST4RD, Orphanet Journal of rare diseases 2019
Slide 25: Content
Content • Values, Value and Value for Money in Healthcare • How valuable is the value assessment in the highly valued Nordics? • Takeaways
Slide 26: The ORPH-VAL principles
The ORPH-VAL principles 9 ORPH-VAL PRINCIPLES for OMPs 1. The assessment should consider all relevant elements of product value in a multi-dimensional CRITERIA framework 2. Pricing & reimbursement (P&R) decisions should be founded on the assessment of value for money and adjusted to reflect other co
Slide 27: The Dolon report for the Nordics
The Dolon report for the Nordics • Purpose: Dolon was given the assignment to assess alignment of P&R systems in the Nordics applying the ORPH-VAL Principles, providing the possibility to generate a discussion on opportunities to improve access • Content: The assessment highlights where the differen
Slide 28: The ORPH-VAL principles – key areas for improvement
The ORPH-VAL principles – key areas for improvement 9 ORPH-VAL PRINCIPLES for OMPs 1. The assessment should consider all relevant elements of product value in a multi-dimensional framework ✓- ✓✓ 2. Pricing & reimbursement (P&R) decisions should be founded on the assessment of value for money and ✓✓
Slide 29: Key areas for improvements in the Nordic countries
Key areas for improvements in the Nordic countries 1-2 4 5 Va l u e a s s e s s m e n t More consistent RDT assessment processes should disease-specific processes should be consider all RDT expertise should be adaptive and subject to specificities in a incorporated in the need and availability consi
Slide 30: Content
Content • Values, Value and Value for Money in Healthcare • How valuable is the value assessment in the highly valued Nordics? • Takeaways
Slide 31: Takeaways
Takeaways • The importance of investing in health • Value has many faces • Value for Money also needed for OMPs • Evidence Gaps as Key Challenge • Nordics perform generally well on the ORPH-VAL assessment principles • Room for improvement: more formalized, balanced, adaptive and collaborative approa
Slide 32: If it's not ok, it's not the end
“ “ Everything will be ok in the end. If it’s not ok, it’s not the end. John Lennon
Slide 33: Nordic Roadmap for Rare Diseases
Nordic Roadmap for Rare Diseases by Birthe Byskov Holm, President, Rare Diseases Denmark C-ANPROM/DK/TAKH/0015
Slide 34: The Nordic Roadmap for rare diseases
The Nordic Roadmap for rare diseases Patients are facing a diagnostic delay The special challenges There is a need for patient of rare empowerment diseases There is uneven access to innovative and adequate treatment
Slide 35: Diagnostic Delay
Diagnostic Delay Challenges Recommendations • On average it takes 6-8 years before a person with a • Investment in pioneering diagnostic platforms rare disease receives the correct diagnosis • Increase the support to the European Reference • 40% of rare disease patients are initially misdiagnosed Ne
Slide 36: Patient empowerment
Patient empowerment Challenges Recommendations • Empowerment needed due to low prevalence, lack of • Putting empowerment on the agenda to promote patient expertise and poor quality of life empowerment at all levels • People living with a rare disease are facing a lack of • Inclusion of patient repre
Slide 37: Patients' access to innovation
Patients’ access to innovation Challenges Recommendations • Few or no treatment options • Access to treatment should be supported by provision of funding at a national level • Delayed or no access to treatment • Combining immediate access for patients with models that allows for controlled uptake, r
Slide 38: Going forward we promise each other
Going forward we promise each other To continue to collaborate across borders and expert fields Given the rarity and high complexity, we need to recognize that people living with a rare disease must be treated differently to secure The Nordic countries will pave the the same progress as we take for
Slide 39: Roadmap for Rare Diseases
ROADMAP FOR WHAT WE SHOULD STRIVE TO DO: Securing access to innovative treatment As part of addressing the unmet medical needs of people We can overcome the special challenges of rare living with rare diseases, we need to break the access RARE DISEASES diseases by… At the summit leading experts shar
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