October 2023
EFPIA Japan

EFPIA Day 2023 Event: EFPIA discusses the valuation of pharmaceutical products with multistakeholders

On 5th October 2023, EFPIA (European Federation of Pharmaceutical Industries andAssociations, headquartered in Brussels, Belgium) and EFPIA Japan hosted a live webinar on
the role of pharmaceutical value assessments and its role in the Pharmaceutical Innovation
Ecosystem (PIE) in Tokyo. A multi-dimensional panel of industry representatives, healtheconomists, patient organisations, parliamentarians, and local and global experts came
together to discuss their visions and challenges of assessing the value of pharmaceuticals topropel them to the next stage of innovation.

In her opening remarks, EFPIA Japan Access Committee Chair Emiko Yoshikawa beganby commending the universal health coverage in Japan, which enables timely reimbursement
and patient access to medicines after regulatory approval, while highlighting that designs ofpricing and its complemental cost-effectiveness assessment systems are crucial in maintaining
the Pharmaceutical Innovation Ecosystem and to avoid drug lag/loss.

Part 1: Presentations
Representatives from industry, academia and government gave presentations fromtheir respective perspectives.

Social Welfare and Healthcare Systems around the World and in Japan
Dr. Isao Kamae, Project Professor, University of Tokyo

Dr. Kamae outlined a global healthcare
snapshot, its implications for Japan, and subsequent
recommendations to create better value forpatients.
With growing worldwide pharmaceuticalexpenditure and the risk of unforeseen resource
conflicts, the importance of assessment quality andpatient-reported outcomes inclusion is becoming
increasingly apparent. This is especially vital in Japan, where the life expectancy-healthexpenditure correlation is close to the threshold where increased investment will cease toabout meaningful effect increases in an ageing and shrinking population. A new system with astronger focus on scientific evidence and better transparency and consistency is required toensure optimal cost-effectiveness, while the Ministry of Health, Labour and Welfare should
expand from optimising assessments to value creation and build compelling logic for a patientcentric cost-effectiveness assessment system.
Sustainable Pharmaceutical Innovation Ecosystem – Pricing and HTA to support sustainable
innovation for patients
Lars Fruergaard Jørgensen, President of EFPIA

President Jørgensen highlighted how theindustry can contribute to a patient-centric approachfor Japan by drawing global examples of thechallenges and impact of cost-effectivenessassessments and best practices.
Active contribution from key stakeholders(especially patients) enables good decision-making
processes that are pivotal for innovation, asexemplified by the high number of assessment criteria in leading markets that include thepatient perspective component. Current sub-optimal stakeholder involvement in Japantranslates to less well-defined processes and potentially lower market attractiveness. To shapefuture assessments and decision-making, Japan should build on current strengths, including theshort duration of reimbursement and increase market attractiveness by involving patient
stakeholders.

How Japan evaluates the value of medicine
Gaku Hashimoto, Member of House Representative
MP Hashimoto highlighted the importance ofpromoting innovation while ensuring thesustainability of universal health coverage, thecurrent challenges with drug lag and loss along withrecommendations for resolution, and the need forpricing reform to increase the Japanese market
attractiveness.
A sustainable healthcare system is vital in
Japan with its sizeable pharmaceutical industry andageing population. While recent regulatory changes have largely alleviated the drug lag problem,
drug loss remains a major challenge, along with an increasingly grave stable supply issue withgenerics.
To tackle this problem and increase market attractiveness, the pricing and HTA systemneeds to be more predictable and considerate of the patient’s perspective to reward innovation
better.

Scientific relevance and improvements in the evaluation system
Dr. Ataru Igarashi, Associate Professor, Yokohama City UniversityDr. Igarashi provided an introduction to using
scientific evidence to strengthen pricing and HTA
systems and drive the Pharmaceutical Innovation
Ecosystem, juxtaposing the current practices ofEurope and Japan.
The current cost-effectiveness assessment inJapan is limited to the price and drug effectiveness
relationship, and variations among different clinicalsubgroups and other qualitative parameters are not considered in drug pricing. Instead oflimiting the assessment to quantifiable considerations, additional factors such as unmet needs
and caregivers’ QOL should be considered to reflect the true value of treatments.

Part 2: Panel discussion
Moderated by the EFPIA Japan Chair Takahiko Iwaya, a panel session followed to discuss
how the value of medicines should be evaluated in Japan with the patient representative,
Specified NPO PanCan Japan Chair Yoshiyuki Majima.

Japan, as a society, should reflect treatment values from multiple perspectives
The patient representative and Specified NPO PanCan Japan Chair Yoshiyuki Majima
started with an illustration of his own journey as a pancreatic cancer patient to point out thesignificant problem of drug loss where access to drugs that are not yet approved or not being
developed in Japan is the issue. This is especially pronounced for progressive and rare cancers,
as the predetermined national healthcare budget does not account for the underrepresentationof rare diseases and patient subgroups.
Majima suggested that a broad perspective on the value assessment of medicines isimportant to bridge this gap, and to achieve this, the participation of patient organizations in
multi-stakeholder dialogues should be increased. The practice of patient involvement in Europe
can serve as a reference for Japan, and the expectation for EFPIA Japan is to provide a platform
for patients and other stakeholders to exchange ideas.
EFPIA Japan Chair Takahiko Iwaya added that evaluations should be a reflection of thevalue of treatment from multiple perspectives, including the QOL of caregivers. The participants
noted that the designation of advanced medical care as an example of a solution to provide
patient access to treatments not yet approved in Japan, and suggested that regulations should
be developed where patients can quickly access groundbreaking innovations, using private
insurance in addition to public insurance.

Inquiries:Riku Ota

Emiko Yoshikawa

EFPIA Japan Access Committee HTA Taskforce
Novo Nordisk Pharma Ltd.
Email: RQXO@novonordisk.com

Chair of EFPIA Japan Access Committee
Novartis Pharma K.K.
Email: emiko.yoshikawa@novartis.com