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

The right to access to medicines requires the implementation of complex mechanisms with the intervention of various actors, among which public institutions prevail as guarantors of collective health. The role of the administrations around access to medicines cannot be limited to a passive position and that far from being exhausted with the material delivery of the medicine to those who need it, it imports a series…

Horizon Therapeutics plc (Horizon) appreciates this opportunity to comment on the European Commission’s (EC) initiative to revise the Orphan and Paediatric Regulations. Horizon is a biopharmaceutical company focused on researching, developing and commercialising medicines that address critical needs for people impacted by rare and rheumatic diseases.

The U.S. Chamber of Commerce (“Chamber”) Global Innovation Policy Center (GIPC) appreciates the opportunity to respond to this European Commission (“Commission”) Inception Impact Assessment on intellectual property (IP) incentives, which complements the Chamber’s ongoing support for the Commission’s work to remove trade barriers and to protect IP in the European Union (EU) and internationally . The U.S.

EPTRI, the European Paediatric Translational Research Infrastructure, is an initiative aimed to accelerate and expand the paediatric drug discovery and development processes by focusing on basic, preclinical, and translational research methods, services and technologies, curried on in collaboration between 112 research Institutions which have signed the EPTRI Memorandum of Understanding by October 2020.

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 representing over 160 countries worldwide.

We welcome the opportunity to provide comments on the European Commission’s initial impact assessment on Regulations for medicines for children and rare diseases (RD). Amgen discovers, develops, manufactures, and delivers innovative human therapeutics. A biotechnology pioneer since 1980, Amgen was one of the first companies to realize the new science’s promise.

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.

EURORDIS Rare Diseases Europe welcomes the opportunity to provide feedback to the European Commission Inception Impact Assessment (IIA) on Paediatric Medicines and Orphan Medicinal Products. EURORDIS is of the opinion that moving forward, any future improvements to the Regulation on Orphan Medicinal Products and Paediatric Medicines are to reinforce the EU processes in a structured and seamless way for all rare…

After discussing the European Commission’s Inception Impact Assessment, we support the implementation of joint solutions to harmonise and optimise procedures, incentives and rewards addressed to paediatric and orphan medicinal products.

In Forum, the Slovenian innovative pharmaceutical association, which represents 22 global research companies, we are aware, that to date, the Paediatric and Orphan Regulations have provided immense benefits for children and patients with rare diseases (RD) in Slovenia. We would welcome Commission’s effort to maintain the legislative framework which would ensure possibilities to treat DS also in the future.

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 successful, rather than preserving the EU’s strengths and incorporating learnings from other regions.

Insufficient development in areas of greatest unmet medical needs for patients: the development of medicines is not covering all major therapeutic areas. In the area of medicines for rare diseases, the evaluation found that 95% of rare diseases still have no treatment option. Unmet Medical Need (UMN) definition and criteria need to be well established and monitored.

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 with the Pharma strategy, should make the legislation future-proof and consider also future treatment options and…

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 treatments.

HollandBIO, the Dutch biotech industry association, appreciates the opportunity to provide feedback on the inception impact assessment on the revision of the EU legislation on medicines for children and rare diseases. We endorse the four objectives for the revision of the two Regulations. We acknowledge there is room to improve and strengthen the effectiveness of the legislation.

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.

The Association of Innovative Pharmaceutical Industry (AIFP) shares the EFPIA´s view expressed in its input into this Inception Impact Assessment. Further to this we would like to stress some of our main concerns and observations.

Alexion is on a mission to transform the lives of people with rare disease and devastating conditions through the research and development of innovative medicines. Thanks to our commitment and sustained investments patients suffering from rare and ultra-rare conditions such as Paroxysmal Nocturnal Hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS), generalized Myasthenia Gravis (gMG), Neuromyelitis…

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 improve them so they better address unmet needs, unintended effects and are more future proof.

On behalf of the Ministry of Public Health, Welfare and Sport, please find attached a file containing the following: (1) Joint feedback from the Netherlands and Belgium on the inception impact assessment for the revision of the legislation for orphan and paediatric medicinal products; (2) Feedback from the Netherlands on the inception impact assessment for the revision of the legislation for orphan and paediatric…

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 specific patients achieve their goals.

PhRMA member companies share the goal of ensuring that patients with unmet medical needs in Europe have timely access to the latest cutting-edge treatments and cures. It’s been proven that innovative medicines help extend lives, lower hospital and overall health care costs and improve the financial sustainability of health systems.

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 current mechanisms (Option 4 in particular).

The LEEM and its 260 members are fully mobilised in the light of the objectives identified in the IIA. Any revision of incentives should be designed to maintain a Europe of innovation (one of the challenges of the Commission’s Pharmaceutical Strategy), but the proposed options do not allow this to be achieved.

Filed in French · English published by the European Commission

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 medicines, without which they might not survive or would have a substantially diminished quality of life.

Rare Cancers Europe (RCE), a 39 partner strong multi-stakeholder partnership, welcomes the EU efforts to assessing solutions to improve the legislative environment on medicines for rare diseases, including rare cancers. With more than 20% of all new cancer cases being rare, including all paediatric cancers, rare cancers belong to both cancers and rare diseases.

Please refer to the attachment : Feedback from the Netherlands and Belgium, participants in the Beneluxa initiative on the Inception Impact Assessment of Revision of the EU regulations on medicines for children and rare diseases Joint statement from the Netherlands and Belgium and additional comments for Belgium (FAGG-AFMPS, RIZIV-INAMI, KCE).

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.

Please see our full commentary in the attached file. The German National Alliance of Patient Organisations of People Living with a Rare Disease (ACHSE – Rare Diseases Germany) agrees with the analysis of the Inception Impact Assessment (IIA) of the current problems with availability and accessibility of medicines for rare diseases in Europe.

Pharmaceutical companies are committed to develop solutions for unmet needs and to ensure available treatments reach all patients in Europe. Patients with rare diseases and children have benefited greatly from the progress which has been realised with the OMP Regulations.

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 in the recent joint evaluation of the two Regulations.

As the Incept Impact Assessment notes, 95% of rare diseases currently have no available treatment, while many paediatric needs are unmet. Unfortunately, the patent system discriminates often against commercial R&D into such diseases.

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.

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 meeting best-case expectations from its impact assessment (IA) conducted in 2004.

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 side-effects due to older medicines.

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 investments.

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.

The Proveca team has extensive experience in paediatric medicines, including 13 years with the PUMA process, gaining PUMAs for Sialanar® and Buccolam®. We observe 4 main challenges: 1. Market Protection for PUMA medicines.

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 step-father and one uncle of a 12-year-old boy who relapsed from an aggressive rhabdomyosarcoma subtype.

First we want to make a few general comments on the process and currently available documents and studies: *It is highly relevant to state that the OMP Regulations were able to provide, for the first time in human history, hope for rare disease patients who had no therapies available or in sight, because the advancement of knowledge and tools in biology and biotechnology now allows treatment development for these…

We welcome and thank Commission’s efforts to address shortcomings in the development of paediatric and orphan medicines through the evaluation of the EU legislative framework. Regarding the proposed options of the Commission, we would like to summarize our contribution for consideration in five areas: 1.- Unmet medical needs: We would be in favour of updating the list of “Therapeutic Needs” issued by the PDCO to…

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.

Comments on the EC- Inception Impact Assessment Ref. Ares (2020)7081640 - 25/11/2020 From European CRO Federation (EUCROF), Paediatric Working Group of EUCROF EUCROF is welcoming the opportunity to comment on the Inception Impact Assessment from the EC in view of the Revision of the EU legislation on medicines for children and rare diseases.

ACCELERATE CONTRIBUTION TO THE EU INCEPTION IMPACT ASSESSMENT ON PROPOSED REVISION OF PAEDIATRIC AND ORPHAN REGULATION ACCELERATE, the international multi-stakeholder platform which aims to accelerate innovation in drug development for children and adolescent with cancer, welcomes the initiative of the European Commission to revise both the Paediatric and Orphan Regulations.

The Danish The Danish Association of the Pharmaceutical Industry (Lif) is fully committed to ensuring that solutions to unmet needs are found and that treatments are made available to all European patients. Children and patients with rare diseases have greatly benefited from the developments made through the Paediatric & Orphan Regulations.

The French authorities (FAs) welcome the significant advances that Regulations (EC) No. 141/2000 (orphan medicinal products for rare deseases) and (EC) No. 1901/2006 (medicinal products for paediatric use) have made possible since their entry into force.

Takeda is a global, patient-focused, values-based and R&D-driven biopharmaceutical company committed to bringing better health to people and a brighter future to the world. Takeda believes this review should focus on supporting the sustainable development of innovative therapies addressing unmet medical needs and access pathways for patients to benefit for these therapies in a timely and equitable manner.

FARMAINDUSTRIA RESPONSE TO THE EC PRELIMINARY IMPACT ASSESSMENT OF THE ORPHAN AND PAEDIATRIC REGULATIONS Farmaindustria, which represents the innovative pharmaceutical industry in Spain, is committed to combating uncovered diseases by promoting research into new drugs and helping to ensure patients’ access to new treatments.

Filed in Spanish · English published by the European Commission

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 that the Commission’s options laid out in the inception impact assessment are unlikely to achieve the desired objectives.

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, considering the achieved scientific progress.

AIM welcomes the steps taken by the European Commission to revise the orphan medicinal products legislation, taking stock of 20 years of operation. While the current legislative framework has brought a number of useful medicinal products to the market, now is a good time to reflect what has worked and not worked in terms of the regulatory framework reaching its objectives.

The platform “Medicine for Society” (Medicijn voor de Maatschappij) is an academic platform in the Netherlands that aims to enhance the availability and accessibility of drugs for rare diseases, funded by the “Vriendenloterij”. We welcome the initiative of the European Commission to address issues within the current legislation on medicines for children and rare diseases.

The European Joint Programme on Rare Diseases (EJP RD) thanks the European Commission (EC) for the opportunity to comment on this inception impact assessment regarding the revision of regulations on medicines for rare and paediatric diseases. EJP RD is a consortium born in the context of the H2020-EU.3.1.3. Programme and co-financed by the EC and participating Member States.

The professionals who are members of the Spanish Association for the Study of Congénitos del Metabolism Errors (AECOM) want to support the European Commission’s efforts to increase the development of orphan medicinal products and improve their access to all potential victims under the best possible conditions.

Filed in Spanish · English published by the European Commission

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 & Orphan Regulations.

As the leading international advocacy organisation dedicated to realizing the promise of advanced therapy medicinal products (ATMPs) and the global voice of the sector, the Alliance for Regenerative Medicine (ARM) welcomes the opportunity to provide feedback on this inception impact assessment.

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.

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 subsets of common diseases.

The Association of Innovative Pharmaceutical Employers INFARMA represents 25 pharmaceutical, R & D and innovative medicines companies. The mission of INFARMA member companies is to ensure that all Polish patients, including those with rare diseases and children, have access to therapies available.

Filed in Polish · English published by the European Commission

The American Chamber of Commerce in Poland (AmCham) is an organisation that brings together more than 340 US companies operating in Poland, including key pharmaceutical companies, representing at the same time one of the largest groups of foreign investors.

Filed in Polish · English published by the European Commission

Lif Sweden’s member companies are developing and producing innovative medicines, including orphan and paediatric medicines, and are committed to patients’ access to innovative treatments and to meet unmet medical needs.

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.

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 innovation in areas of unmet needs and the European pharmaceutical industry as global leader in health innovation.

The Spanish Federation of Rare Diseases (ERDF) would like to welcome the revision of this legislation and the recognition of the problem, giving a very positive assessment of this initiative. In line with the contributions of the European Rare Diseases Alliance (EURORDIS), and based on the experience of our associative fabric, we identify it as a priority for this new European regulation on orphan medicinal products…

Filed in Spanish · English published by the European Commission

About 500.000 patients are suffering from rare diseases (RD) in Belgium. The EU Orphan Regulation has highly incentivized pharmaceutical industries to develop new treatments for those patients. The number of clinical trials in RD in Belgium has increased by 13% over the past 4 years; more than 20% of the clinical trials running today are in RD.

Newesko Ltd is a consultancy company collaborating with several international pharma companies especially in the field of rare diseases. We would like to raise the following concerns regarding the EU Pharmaceutical Strategy. Addressing unmet needs of patients is of vital importance. All the intentions of the Commission to increase health and ensure accessible and affordable drugs for patients are appreciated.

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 (OMPs) and paediatric medicines in its IIA and we welcome the objectives of stimulating more development in areas of unmet…

The Spanish Association of Orphan and Ultraorphan Medicines Laboratories (AELMHU) considers that Regulation 141/2000 and Regulation 1901/2006 have contributed to improving the quality of life of patients with rare diseases.

Filed in Spanish · English published by the European Commission

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 different EU initiatives over the past.

The Irish Pharmaceutical Healthcare Association represents 47 global companies that research, develop and bring to market innovative medicines. We partner for better healthcare and economic development in Ireland and, by extension, in the European Union. We support key goals in the proposed EU Pharmaceutical Strategy.

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.

The proposals put forward by the European Commission address the social need to further develop activities aimed at developing activities on so-called orphan drugs for rare diseases. We are pleased to see that through joint action, Polish patients in Poland will have easier access to medicines, which have so far been used only by a small number of patients.

Filed in Polish · English published by the European Commission

The German social security umbrella organisations in Germany welcome the plan to revise the existing incentive schemes for orphan medicinal products and paediatric medicinal products and agrees with the European Commission that the aim of this revision must be to: (1) focus support on areas where the regulations have not shown the desired success; (2) end support for areas where it is not necessary; (3) link funding…

Filed in German · English published by the European Commission

The German Medicines Manufacturers´ Association (BAH) is the leading trade organisation of the pharmaceutical industry in Germany. It represents the interests of about 400 member companies. Both globally operating medicines manufacturers are involved in the manifold activities of the association as well as the broadly represented medium-sized businesses.

ORPHELIA Pharma is a pharmaceutical company involved in the development and marketing of paediatric medicines. Our first product, Kigabeq®, is the paediatric formulation of the antiepileptic drug vigabatrin, to treat a rare and very serious condition: infantile spasms, also known as West syndrome. Vigabatrin is an essential medicine and was listed twice in the priority list of paediatric medicines of the EMA.

The Regulations that the European Union adopted through the Regulation for Medicines for Rare Diseases in 2001 gave a strong boost to the work of pharmaceutical laboratories in this field, and today more than 160 new drugs are available to treat 90 diseases. But much remains to be done, as 95% of rare diseases need treatment.

Our analysis of the Paediatric Regulation shows that its revision needs to focus on 6 major issues — In cases where the current PIP determination framework using adult development as a starting point does not meet the unmet needs of children, care must be taken to have * a sound scientific case demonstrating the validation of pre-clinical models linked to the pathology concerned and a particular focus on effects on…

Filed in French · English published by the European Commission

Since Hipofam, we welcome the proposed changes and the willingness to improve access to and development of medicines for paediatric patients, and thus how improvements to the system of incentives for medicines for rare diseases. Although inclusion and reimbursement fall within the competence of the States, measures to improve fairness in access across the EU should be considered.

Filed in Spanish · English published by the European Commission

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 longer protected by patents etc.

Only available medication can help patients and it is therefore essential to insist on access. Countries may remain independent in the negotiation of price and reimbursement, but they must establish: 1 deadlines, 2 requiring the participation of painting organisations in the exercise of their empowerment, and 3 transparency and information.

Filed in Spanish · English published by the European Commission

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 development and availability of medicines in these areas.

In 2000, the European Union adopted a Regulation on Orphan Medicinal Products, which was a turning point in providing incentives for research and development of treatments for rare diseases. This stimulated the work of laboratories in this field, leading to the availability of 169 drugs that treat 90 diseases (5 % of all orphan diseases) today.

Filed in Spanish · English published by the European Commission

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 development of medicines for children and rare diseases.

Re: Inception Impact Assessment for Revision on medicines for children and rare disease The Dental and Pharmaceutical Benefits Agency (TLV) is the Swedish pricing and reimbursement agency, answering to the Ministry of Health and Social Affairs. TLV assesses some 50 new substances for the out-patient sector for reimbursement every year.

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 medical progress enabled by the orphan medicinal and paediatric regulations.

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 stimulate innovation and the development of orphan and pediatric medicines in areas of unmet needs.

We welcome the initiative of the European Commission to review the legal frameworks for medicines for children and rare diseases to address their shortcomings. As identified, it is important to stimulate development of medicines for unmet needs and ensure equal access for patients in all EU countries.

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 topic.

Medicines for Europe recognises the importance of incentives for orphan & paediatric medicines and appreciates the opportunity to submit a feedback on the Inception Impact Assessment (IIA). As highlighted in the IIA, while overall existing incentives have generated some success, there is a need to fine-tune elements to ensure incentives achieve the objectives intended by the Regulation whilst avoiding abuses/misuses…

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.

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.

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 horizon for promising products and companies. They subsequently acquire them.

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 treatments for 95% of rare diseases. This must change.

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 problems: First, there are no approved treatments for 95% of rare diseases. This must change.

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 disease patients.PTC recognised that after 20 years it is a good moment to take stock of the effectiveness of the…