Member Spotlight / News - February 5, 2024

EUCOPE Member Spotlight: Q&A with Chiesi GRD

Every month, EUCOPE spotlights a member company and the great work they’re doing to advance the life sciences industry and drive innovation to serve patients better. In February, we spoke with Enrico Piccinini, Head of Europe & International Rare Diseases, Chiesi Global Rare Diseases

Enrico has been the Head of Europe & International Rare Diseases, at the Chiesi Group since November 2019, leading a team fully dedicated to make available value-adding treatments for rare and ultra-rare diseases. He previously had a long tenure in Genzyme and SanofiGenzyme covering different sub-regional and regional/EMEA/EU roles in both functional areas and business ones. During the last seven years as General Manager of SanofiGenzyme Italy & Malta, he led his team through different launches and sustained business growth within Rare and other Specialty Care diseases. He is member of various Biotech/RD and Orphan Drugs groups and associations, and since 2022, he leads the Famindustria (Italy) RD national working group.

Tell us about your organisation and its mission and how you drive innovation internally?

Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver transformative therapies and solutions for people living with rare diseases. It reflects Chiesi Group’s decades of experience in research and drug development and our commitment to putting patients’ needs at the forefront of everything we do. As a family business, we relentlessly pursue progress in our research and sustainable growth in our business by developing close partnerships with the patients’ community, caregivers, healthcare professionals, academic and industry partners. We apply a deliberate and careful approach to the production and commercialization of all our unique products and services. We started our journey in 2020 and today we can count on a portfolio of 10 commercialized products worldwide and an increasing presence in different therapeutic areas, such as Lysosomal Storage Disorders, Immunology, Haematology, Dermatology, Ophthalmology and endo-metabolic diseases.

From research to production, we are committed to making a difference and reducing the impact of rare diseases on patients and families.

How do your organisation’s activities help patients now and into the future?

Chiesi Global Rare Diseases is focused on making available treatments to serve the unmet medical needs of people living with a rare disease. We bring therapeutic solutions or services in those areas where there’s no other option available or where there’s still a high unmet medical need.

Our collaborative approach with the patients’ communities is very unique. We let the patient voice shape who we are. We internalize patient stories highlighting the patient journey, unmet needs and psychosocial-emotional aspects of living with a rare disease. We provide compliant support to patient organizations and connect them with resources for community benefit. We involve community in co-creation of patient friendly information, data, tools and inclusion opportunities. We also partner with a range of international and regional rare disease advocacy groups to learn and co-develop programs to empower patients and caregivers.

February is a special time as it’s Rare Disease Month and culminates on Rare Disease Day on 29 February. Chiesi is happy to continue its #PlayTheRareGame campaign in 2024 to pass the knowledge and raise awareness of rare diseases. This year, we revamped the campaign leveraging our current partnerships with F.I.R. (Italian Rugby Federation), patient advocacy groups, and Make-A-Wish International to shift the focus from the diseases to people, focusing on their courage and capacity of enjoying the best from their lives. Thanks to our rugby partnership, we’re bringing the game to be played during two games at the Six Nations tournament, which will help us amplify the message and raise rare disease awareness to an international audience.

What do you see as the biggest challenge facing the life sciences industry today?

High inflation and the increase in public debt, are serious challenges to the life science industry. We are already witnessing a gradual disinvestment in public healthcare budgets and the access to new rare disease therapies will be a struggle in many countries. Furthermore, the evidence requirements for rare disease therapies are already becoming more stringent and this will impact the accessibility and availability of new treatments across European countries.

Overall, rare diseases present a societal concern owing to difficulties and delays in diagnosis, a lack of treatment availability, investment risk in developing treatments and the need for reliable regulatory and access conditions. Delayed diagnosis often complicates therapeutic options because rare diseases are progressive, leading to loss of function, and the systemic impact may be permanent in the absence of early intervention.

We recently published a report Rare disease burden of care and the economic impact on citizens in Germany, France and Italy as part of our PENDULUM EU project, that opens the door to a more granular way of thinking about rare diseases. The report found that rare diseases incur a significantly higher average cost burden of €107,000 per patient per year (PPPY), approximately 15 times greater than the benchmark for the average burden of high-prevalence diseases. In addition, data showed that indirect costs average 29% of the total burden of the rare diseases when treatment is available, rising to an average of 45% when no treatment is available. With this data, it becomes clearer that accelerating the development and access to treatments for rare diseases has the potential to reduce the overall burden, often carried directly by families and caregivers.

If we want to make a positive impact on the future of the rare disease community, we’ll need a few long-term changes at both the national and EU level. On a national level, payers should consider the full burden of rare diseases and adopt more flexible payment models to allow for timely access to therapies. Future research that supports the value equation should also include the calculation of intangible burdens such as stigmatization and depression. At the EU level, we need an environment which fosters rare disease innovation, through a thoughtfully calibrated incentive design and holistic approach across the entire medicine lifecycle. Reducing existing incentives including market exclusivity will jeopardise long term research & development programmes. It is also important to work with the broader stakeholder community to co-create on initiatives that will better serve the rare disease community. Chiesi’s work within the European Expert Group on Orphan Drug Incentives (OD Expert Group) has helped to develop concrete policy proposals for potential improvements to support the delivery of continued innovation for rare diseases, accelerate regulatory pathways and develop incentives for orphan drugs.

What are the major health policy issues and themes that you are most focused on in 2024?

The European Parliament elections combined with the upcoming implementation of the EU HTA Regulation in 2025 and the revision of the EU Pharmaceutical Legislation and Orphan Medicines Regulation (OMP) make 2024 a transformational year. As a group, we are very focused on joining the industry’s efforts to ensure that the various EU legislative and policy discussions do not harm Europe as a place where innovation can be brought to patients and healthcare is considered a societal investment and not just a pure cost.

While Chiesi will not be immediately impacted by the implementation of the EU HTA Regulation in 2025, we hope to gain some insight and experience from the first companies to be affected for oncological products and ATMPs in 2025.

The general pharmaceutical legislation and the OMP regulation have proven successful in bringing new therapeutic options for patients. From 2000 to 2022, the OMP Regulation has led to more than 200 orphan drugs being developed in Europe. Despite the success, many patients are still lacking treatments and Chiesi supports the idea of modulating incentives to promote investments in areas of unmet medical needs such as rare and ultra-rare disease.

Chiesi shares the overall objectives of increasing the availability and accessibility of medicines for people across Europe and we welcome some regulatory improvements included in the proposals – like reduced timelines for EMA review, and further steps towards acceptance of Real-World Evidence (RWE). However, we are concerned that the proposals in the revision will not help the EU to effectively meet the objectives it has set for itself and risk jeopardizing the competitiveness and innovation capabilities of the EU healthcare ecosystem. This situation could worsen the growing gap with other regions of the world and result, ultimately, in delayed access to medicines for patients.

The EU Pharmaceutical Package must ensure that the EU continues to drive investment and research in chronic and rare diseases and ensure that all patients can benefit from innovation. Innovative medicines development, especially for rare diseases, is a risky endeavor that requires high investment and has a high attrition rate. Developers require a predictable framework that rewards innovation and assure sustainability. Only a courageous policy agenda can bring to the leap needed to address the unmet needs of patients in Europe, today and tomorrow, keeping the region competitive for investments and as a scientific reference point.

What attracted you to join EUCOPE and how can we help you achieve your business goals?

EUCOPE was created to cover the representative gap between large global companies and small biotechs. We have been a member of EUCOPE for a long time and are fully aligned with the vision and objectives of the association. Our Global Head of R&D Diego Ardigó has also been a board member for the past several years.

EUCOPE is an agile organisation, very effective in building trust and constructive dialogue with external stakeholders and PAGs while also providing expert intelligence, enabling us to stay on top of the latest legislative and policy discussions. It is the ideal EU trade organisation for innovative, smaller companies focused on developing therapies for people with rare diseases like Chiesi. EUCOPE excellently ensures that the voices, opinions and expertise of Chiesi and others are heard at the EU and national level.


For more information about EUCOPE membership

Visit our Benefits of Membership Page, or schedule an introductory call with our Business Development team