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Revision of the EU legislation on medicines for children and rare diseases

Public health

EU legislation on medicines for children and rare diseases was introduced in the early 2000’s, to address the lack of medicines in this area. This initiative will explore the need for adequate measures to address a number of shortcomings in the functioning of the existing framework detected during a recent evaluation.

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Public consultation

ID: 12767

Lobbying Activity

50 responses

Consultation response

American Chamber of Commerce to the European Union, 6 Jan 2021

Representing American companies operating in the EU, AmCham EU is well aware of the differences and respective strengths of innovation ecosystems on both sides of the Atlantic. The options laid out in the inception impact assessment (IIA) risk undermining an EU framework that has proven largely…

Consultation response

European Federation of Pharmaceutical Industries and Associations, 6 Jan 2021

EFPIA members are committed to ensuring that unmet needs are addressed and that available treatments reach all European patients. Children and rare disease (RD) patients have greatly benefited from the progress achieved through the Paediatric and Orphan Regulations. The Paediatric Regulation is…

Medicines for Europe seeks limits on orphan drug monopolies

MEDICINES FOR EUROPE, 21 Dec 2020

The organization requests preventing companies from combining orphan and paediatric incentives to delay competition. They want explicit exclusion of generics and biosimilars from orphan 'similar product' definitions after ten years. They also seek pull incentives for off-patent paediatric medicines and oppose transferrable exclusivity vouchers.
This would allow their generic and biosimilar products to enter markets faster after exclusivity periods expire.
Originator pharmaceutical companies lose extended monopoly periods and additional exclusivity protections for orphan drugs.

Consultation response

Novartis International AG, 5 Jan 2021

At Novartis, our mission is to reimagine medicine to improve and extend people’s lives, including transformative medicines for rare disease patients and children in Europe and around the world. Novartis appreciates the Commission (EC)’s recognition of the importance of orphan medicinal products…

Consultation response

EuropaBio, 6 Jan 2021

Developing medicines for children and rare diseases is a lengthy and complex undertaking. Much less is known about these illnesses when compared to more common conditions, meaning extensive research is required before scientists and researchers can seek to transform their ideas into potential…

Consultation response

SANOFI, 21 Dec 2020

The OMP Regulation has been a significant success. It has contributed to bringing around 180 medicines on the EU market and has benefitted more than 6 million patients across the EU. Sanofi welcomes the European Commission’s (EC) efforts to explore the creation of additional incentives to…

Consultation response

Johnson Johnson, 6 Jan 2021

The Orphan and Paediatric Regulations have spearheaded a wave of innovations in underserved areas. They have undeniably delivered on their objectives considering the marginal economic case for developing most of these medicines; but more must be done. We welcome the opportunity to discuss how to…

Consultation response

Merck Sharp Dohme Europe Belgium SRL, 6 Jan 2021

MSD SUBMISSION TO THE OMP AND PAEDIATRIC ROADMAP As shown by the Commission's studies, the EU incentives framework has enabled research-based companies such as MSD to develop medicines for children. With over 260 new medicines for children, the EU’s Paediatric Regulation has proven its value.…

Consultation response

AbbVie, 6 Jan 2021

As an innovative biopharmaceutical company with over 11.000 employees in the EU, AbbVie supports the Commission’s aims to develop more medicines for unmet needs, speed access to new therapies for all EU citizens, and ensure the maximum benefits from scientific and technological advances. AbbVie…

Patient alliance urges seamless EU rare disease therapy pathway

EUROPEAN ORGANISATION FOR RARE DISEASES, 6 Jan 2021

EURORDIS calls for early multi-stakeholder dialogue on unmet needs, graduated incentives rewarding earliest engagement, strengthened regulatory committee mandates, and a common European negotiation table for timely equitable access across member states.
This would accelerate development and approval of treatments for their 30 million constituents.

Consultation response

European Public Health Alliance, 18 Dec 2020

Small biotechs, university spin-offs and other research institutions are now at the heart of pharmaceutical R&D conducting the early-stage research, financed by venture capitalists, hedge funds and private equity. Large pharmaceutical companies perform less research in-house while they scan the…

Consultation response

Pfizer Inc., 4 Jan 2021

As an innovative biopharmaceutical company, Pfizer is a global leader in R&D of new treatments for paediatric and rare diseases. In the EU, the framework created by the Orphan and Paediatric Medicines Regulations has allowed investment in areas of unmet need, to the benefit of millions of patients.…

Consultation response

Merck, 5 Jan 2021

Merck, a leading science and technology company with headquarters in Germany, is strongly committed to developing innovative medicines in areas of unmet needs, improving patients clinical outcomes and quality of life. We share the European Commission’s (EC) goal to continue supporting medical…

Consultation response

European Confederation of Pharmaceutical Entrepreneurs, 6 Jan 2021

EUCOPE’s represents 130 small to medium-sized companies playing a key role in the European pharmaceutical environment, many focused on rare diseases. Some of them have unique profiles due to their highly specialised product portfolio, no or limited revenues to date and/or significant risky R&D…

U.S. Chamber warns EU against weakening orphan drug protections

Chamber of Commerce of the United States of America, 6 Jan 2021

The organization urges the EU to maintain current market exclusivity protections for orphan and pediatric medicines. They argue reducing these incentives will not improve research or address identified challenges.
This would protect their members' ability to recoup research investments in rare disease treatments.
European patients may face reduced access as companies shift rare disease research elsewhere.

Consultation response

Boehringer Ingelheim, 5 Jan 2021

Family-owned since it was established in 1885, Boehringer Ingelheim (BI) pursues a long-term vision and stands for continuous investment in medical innovation to improve the health and quality of life of humans and animals. Building on a strong heritage in treating communicable and non-communicable…

Consultation response

Teva Pharmaceuticals Europe BV, 6 Jan 2021

Teva is a strategic partner of European healthcare systems. Teva is committed to researching and developing new therapeutic options for unmet medical needs and improving affordable and equitable access to quality medicines. As a hybrid company and the European leading provider of generic medicines,…

Consultation response

Bristol-Myers Squibb Company, 6 Jan 2021

As a global biopharmaceutical company focused on transforming the lives of patients through science, Bristol-Myers Squibb (BMS) strongly believes in a legislative framework conducive to medical innovation addressing unmet needs (UMN) and available to all patients who need it. However, we believe…

AIM calls for transparency and fair pricing in rare medicines

Association Internationale de la Mutualité, 5 Jan 2021

AIM demands reinforcing investment criteria to ensure medicines have fair compensation models. They seek yearly reporting to stop companies turning treatments into an overprofiteering opportunity. The group also advocates for transparency regarding the underlying costs of drug development.
Fairer pricing models would reduce the financial burden on health insurance funds.
Drug manufacturers would face stricter monitoring and potential limits on their market exclusivity.

Consultation response

European Social Insurance Platform AISBL, 6 Jan 2021

ESIP, representing the statutory social health insurances in the EU, UK and Switzerland, welcomes the initiative of the European Commission to revise the existing legislation concerning orphan medicinal products (OMPs) and paediatric medicines with a view to addressing the shortcomings identified…

Consultation response

European Respiratory Society, 6 Jan 2021

The European respiratory Society (ERS) is an international organisation that brings together physicians, healthcare professionals, scientists and other experts working in respiratory medicine. It is one of the leading medical organisations in the respiratory field, with a growing membership…

Consultation response

European Society for Paediatric Oncology, 6 Jan 2021

SIOPE and CCI-E welcome the proposed revision of EU regulations on medicines for children and rare diseases. All paediatric cancers individually are rare, but jointly are the leading cause of death by disease in children above one year of age in Europe. Most survivors experience long-term…

Consultation response

Association of European Cancer Leagues, 16 Dec 2020

The ECL Access to Medicines Task Force (A2M TF) welcomes the Commission’s initiative to seek a new legal framework to solve issues within current legislation on medicines for children and rare diseases. The A2M TF urges the Commission to prioritise the following problems: There are no approved…

Consultation response

Bundesverband der Pharmazeutischen Industrie e.V., 22 Dec 2020

The German Pharmaceutical Industry Association (BPI e.V.), representing more than 270 members, comprises the whole spectrum of the pharmaceutical industry, ranging from multinational corporations to SMEs, Mid-Caps as well as Start-ups. This also includes companies with a special focus on the…

Takeda urges broader definitions and flexible incentives for rare diseases

Takeda Pharmaceuticals International AG, 6 Jan 2021

Takeda recommends redefining unmet needs to include factors like disease severity and quality of life. They urge for flexibility in paediatric study requirements and the integration of real-world evidence. The evaluation should cover the entire value chain from research to patient access.
Broader definitions and flexible requirements would reduce Takeda's regulatory hurdles and protect its research investments.
National health systems could face increased budget pressure if more treatments qualify for high-value incentives.

Consultation response

Lääketeollisuus ry, 22 Dec 2020

The Pharma Industry Finland (PIF) welcomes the Commission efforts to deliver more health and wellbeing to people. PIF members are committed to ensuring that unmet medical needs are addressed and that available treatments reach patients. Children and rare disease patients have benefitted from the…

Consultation response

The European Association of Hospital Pharmacists, 6 Jan 2021

Attached please find the feedback of the European Association of Hospital Pharmacists (EAHP) on the “Inception Impact Assessment for the Revision of the EU legislation on medicines for children and rare diseases”.

Amgen warns that reducing incentives stifles rare disease innovation

Amgen Inc, 6 Jan 2021

Amgen urges the EU to maintain current market exclusivity rewards and avoid narrowing the definition of unmet medical needs. They oppose tying incentives to mandatory market placement across all member states.
This would protect the company's financial returns and secure its future investments in high-risk research.
Patients with conditions falling outside stricter criteria could lose access to new research.

Consultation response

F. Hoffmann-La Roche Ltd, 6 Jan 2021

Roche is committed to support the improvement of the regulatory environment in Europe and welcomes the opportunity to contribute to the revision of the pediatric and rare diseases legislation. We believe that the revision should not only address current bottlenecks but more importantly, and in line…

Consultation response

PHARMIG - Verband der pharmazeutischen Industrie Österreichs, 23 Dec 2020

The creation of a system of incentives within these Regulations have been identified as the essential and target-oriented measures to increase research and development for rare diseases and children in the EU. And indeed, the evaluation of the Regulations found that both Regulations fostered the…

Consultation response

Plasma Protein Therapeutics Association Europe, international Association without lucrative purpose, 6 Jan 2021

The Plasma Protein Therapeutics Association (PPTA) promotes the availability of, and access to, safe and effective plasma-derived medicinal products (PDMPs) which treat a variety of rare, chronic, and potentially life-threatening conditions. 300,000 European patients rely on these essential…

Consultation response

Stichting Health Action International, 21 Dec 2020

Health Action International (HAI) welcomes the opportunity to contribute to the ‘Revision of the EU legislation on medicines for children and rare diseases’ and commends the European Commission for the thorough and comprehensive consultation process that is shaping the reform of legislation on this…

Consultation response

Dachverband der österreichischen Sozialversicherung, 6 Jan 2021

In view of the steady increase in pharmaceutical spending and the relatively high prices of orphan medicinal products (OMP), the Austrian Social Insurance welcomes the revision of the EU legislation on OMPs and medicines for children. As mentioned in this document, the system of incentives can…

Consultation response

PTC Therapeutics, 7 Dec 2020

The OMP Regulation was introduced to stimulate research in rare diseases. PTC Therapeutics believes the Regulation has been a successful European regulatory instrument as it not only led to an increase in the number of orphan drugs but also put a spotlight on the high unmet medical needs of rare…

Consultation response

Ipsen Pharma, 6 Jan 2021

As a global specialty-driven biopharmaceutical company committed to discovering new solutions for debilitating rare and ultra-rare diseases and improving quality of life for patients suffering from these diseases, Ipsen welcomes the opportunity to comment on the inception impact assessment. The…

Consultation response

Associazione delle Imprese del farmaco, 6 Jan 2021

Farmindustria welcomes the Commission’s commitment to make the pharmaceutical legislation pace with scientific and technological advances in order to ensure a faster patient access to therapies. Innovation in life science is advancing quickly, the regulatory procedures have to keep up as well,…

Consultation response

Hellenic Association of Pharmaceutical Companies, 5 Jan 2021

Hellenic Association of Pharmaceutical Companies (SFEE) members are committed to ensuring that unmet needs are addressed and that available treatments reach all European patients. Children and rare disease (RD) patients have greatly benefited from the progress achieved through the Paediatric &…

Consultation response

Association Mieux Prescrire, 5 Jan 2021

See the Prescrire's response to the public consultation attached.

Consultation response

KickCancer, 6 Jan 2021

KickCancer is a Belgian foundation with the mission to improve the survival chances of children with cancer, to reduce the toxicity of treatments and thereby restoring them to their full health after treatment. KickCancer was founded after the difficult experience of its founders, the parents, the…

Consultation response

Anticancer Fund, 5 Jan 2021

The Anticancer fund applauds the awareness of the commission that in oncology common cancers are split into many subsets based on biomarkers, leading to unnecessary multiplication of rare diseases. Therefore, we support the idea that it will no longer be possible to obtain orphan designation for…

Consultation response

Verband Forschender Arzneimittelhersteller e.V., 5 Jan 2021

The German Association of research-based pharmaceutical companies (vfa) welcomes that the European Commission (EC) is focusing on unmet medical needs (UMN). The current range of therapies for the treatment of patients with rare diseases (RD) and of children is encouraging and has been boosted by…

Consultation response

UCB, 6 Jan 2021

UCB is dedicated to finding unique solutions for diseases with high degree of patient need. Regardless of the population size, it’s patients’ needs that direct our efforts. Our ambition for patients relies on our ability to innovate & bring differentiated solutions with unique outcomes that help…

Consultation response

European Haemophilia Consortium, 6 Jan 2021

The European Haemophilia Consortium (EHC) welcomes the revision of paediatric and orphan medicinal products' regulations. In haemophilia, the OMPR has brought much innovation in the past decade; however, we are disappointed to see that the uptake from the Member States has been variable. Although…

Consultation response

Children's Tumor Foundation Europe, 18 Dec 2020

The Children’s Tumor Foundation (CTF) Europe welcomes the European Commission’s efforts to address shortcomings in the development of pediatric and orphan medicines through the evaluation of the EU legislative framework. CTF Europe is concerned with neurofibromatosis (NF), three rare genetic tumor…

Consultation response

KWF Kankerbestrijding, 15 Dec 2020

European Fair Pricing Network - EFPN The European Fair Pricing Network (EFPN) welcomes the Commission’s initiative to seek a new legal framework to solve issues within current legislation on medicines for children and rare diseases. The EFPN urges the Commission to prioritise the following…

Consultation response

Protection sociale française, 6 Jan 2021

The French representation of social security institutions in Brussels (REIF) welcomes the launch of the revision process of the regulations on medicines for children and rare diseases by the European Commission. REIF especially acknowledges the consideration given to a potential major overhaul of…

Consultation response

Biogen Inc., 21 Dec 2020

Biogen discovers, develops, and delivers innovative therapies for people living with serious neurological and neurodegenerative diseases, including rare conditions – an area to which we are strongly committed to. Biogen developed the first approved treatment for spinal muscular atrophy (SMA), which…

Consultation response

European Children's Hospitals Organisation, 6 Jan 2021

Please see the attached feedback from the European Children's Hospitals Organisation (ECHO).

Consultation response

Swedish Orphan Biovitrum, 5 Jan 2021

Sobi is a Swedish based international biopharmaceutical company focused on rare diseases (RDs), mostly within Haematology and Immunology. We are dedicated to researching and providing sustainable access to innovative treatments that transform the lives of people with RDs. Sobi has carefully…

Consultation response

Initiative Arzneimittel für Kinder, 23 Dec 2020

Medicines for children - PUMA concept The PUMA concept was doomed, because the idea that national health care systems should cover the investments for voluntary paediatric drug development by reimbursing those new products failed. The idea to stipulate voluntary paediatric research for products, no…