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PTC Therapeutics

Our mission at PTC Therapeutics has always been to build an integrated biopharmaceutical company based on our expertise in RNA biology.

Lobbying Activity

Meeting with Olivér Várhelyi (Commissioner) and

2 Oct 2025 · All pressing portfolio topics

Meeting with Vincenzo Matano (Cabinet of Executive Vice-President Raffaele Fitto) and Novartis International AG and Unknown Organization

17 Sept 2025 · Introductory Meeting on Coalizione VITA Activities

Meeting with Adam Jarubas (Member of the European Parliament, Committee chair)

21 Mar 2025 · Choroby rzadkie

Meeting with Eszter Lakos (Member of the European Parliament)

19 Mar 2025 · Biotechnology policy

Meeting with Nicolás González Casares (Member of the European Parliament) and Novavax

19 Mar 2025 · Health

Meeting with Ondřej Knotek (Member of the European Parliament)

9 Jan 2024 · Pharmaceutical package

Meeting with Pernille Weiss-Ehler (Member of the European Parliament, Rapporteur) and Novartis International AG and

18 Jul 2023 · Directive on Medicinal products for human use

Response to Evaluation and revision of the general pharmaceutical legislation

26 Jun 2023

PTC therapeutics agrees with the overall objectives of the proposed Pharmaceutical Package (proposed Regulation and Directive): As a company we are striving to develop therapies for people living with a rare disease and as such we want to contribute to creating an innovative and competitive EU healthcare ecosystem with standards that can compete at a global level. We also support increasing equal access to and availability of innovative medicines to patients across the EU. However, there needs to be a better balance between stimulating innovation and providing patient access. Also as regards patient access, we believe this is primarily a Member State issue, which cannot be effectively addressed through EU legislation. As part of the ongoing review, it is essential that the revised regulatory system builds on the well-established and well-functioning elements of the past 20 years and does not disregard them. PTC appreciates measures that aim to accelerate, streamline and adapt regulatory processes to more efficiently bring much needed innovative therapies to patients with high unmet need. We believe that e.g., the restructuring of EMA, the shortening of the approval timelines, the use of real-world data and the regulatory sandbox proposals are a step in the right direction but will not be sufficient to significantly impact the current regulatory process, and second, that we are concerned that certain other provisions will undermine R&D, innovation, and EU competitiveness, therefore delaying access to novel therapies. We are concerned about several elements: The overall reduction of RDP and market protection periods in the revised Regulation and Directive, will not stimulate innovation nor will it speed up access for patients to new treatments. Instead, it might signal to global companies that the EU is less interested in stimulating innovation within its region. The market launch obligations are a faulty measure for several reasons: Access of innovative therapies to patients depends not only on companies but also on HTA and national payer bodies. It is our experience that often robust discussions cannot be concluded within a 2-year window. Secondly, the number of people living with a certain rare disease can be very small, or even extremely small to the point that some countries may not have any patients. Commencing pricing and reimbursement discussions in all EU countries often makes no sense. Finally certain, technologies such as gene therapies, need to be administered in specialised treatment centres, not present in all Member States. For these therapies it would make much more sense to improve the EUs cross border healthcare system. Finally, whilst we recognise the public authorities wish to have innovation taking place where the highest unmet medical needs exist, we feel that the proposed descriptions of unmet medical need and high unmet medical need are poorly crafted and leave room for too much interpretation and risk a ranking of patients needs which we believe is unfair and unjustifiable. We stand ready to be part of any more elaborative, multistakeholder exercise to refine the approach to UMN. These proposals would be detrimental not only for small and mid-sized innovative biopharmaceutical companies, but for patients living in the EU as well. The innovative pharmaceutical industry adopts significant risks in discovering and developing new medicines, through which they seek to offset some of this risk with conservative forecasting post-authorisation. A competitive environment will increase investments in R&D in the EU, therefore bringing more innovative products to patients, that will therefore benefit from more treatment options. The 2016 Council Conclusions recognised the potential need for a revised pharmaceutical framework, but stressed that any revision, included in the incentive framework, should not discourage the development of medicinal products needed for the treatment of rare diseases.
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Response to Evaluation and revision of the general pharmaceutical legislation

26 Jun 2023

PTC therapeutics agrees with the overall objectives of the proposed Pharmaceutical Package (proposed Regulation and Directive): As a company we are striving to develop therapies for people living with a rare disease and as such we want to contribute to creating an innovative and competitive EU healthcare ecosystem with standards that can compete at a global level. We also support increasing equal access to and availability of innovative medicines to patients across the EU. However, there needs to be a better balance between stimulating innovation and providing patient access. Also as regards patient access, we believe this is primarily a Member State issue, which cannot be effectively addressed through EU legislation. As part of the ongoing review, it is essential that the revised regulatory system builds on the well-established and well-functioning elements of the past 20 years and does not disregard them. PTC appreciates measures that aim to accelerate, streamline and adapt regulatory processes to more efficiently bring much needed innovative therapies to patients with high unmet need. We believe that e.g., the restructuring of EMA, the shortening of the approval timelines, the use of real-world data and the regulatory sandbox proposals are a step in the right direction but will not be sufficient to significantly impact the current regulatory process, and second, that we are concerned that certain other provisions will undermine R&D, innovation, and EU competitiveness, therefore delaying access to novel therapies. We are concerned about several elements: The overall reduction of RDP and market protection periods in the revised Regulation and Directive, will not stimulate innovation nor will it speed up access for patients to new treatments. Instead, it might signal to global companies that the EU is less interested in stimulating innovation within its region. The market launch obligations are a faulty measure for several reasons: Access of innovative therapies to patients depends not only on companies but also on HTA and national payer bodies. It is our experience that often robust discussions cannot be concluded within a 2-year window. Secondly, the number of people living with a certain rare disease can be very small, or even extremely small to the point that some countries may not have any patients. Commencing pricing and reimbursement discussions in all EU countries often makes no sense. Finally certain, technologies such as gene therapies, need to be administered in specialised treatment centres, not present in all Member States. For these therapies it would make much more sense to improve the EUs cross border healthcare system. Finally, whilst we recognise the public authorities wish to have innovation taking place where the highest unmet medical needs exist, we feel that the proposed descriptions of unmet medical need and high unmet medical need are poorly crafted and leave room for too much interpretation and risk a ranking of patients needs which we believe is unfair and unjustifiable. We stand ready to be part of any more elaborative, multistakeholder exercise to refine the approach to UMN. These proposals would be detrimental not only for small and mid-sized innovative biopharmaceutical companies, but for patients living in the EU as well. The innovative pharmaceutical industry adopts significant risks in discovering and developing new medicines, through which they seek to offset some of this risk with conservative forecasting post-authorisation. A competitive environment will increase investments in R&D in the EU, therefore bringing more innovative products to patients, that will therefore benefit from more treatment options. The 2016 Council Conclusions recognised the potential need for a revised pharmaceutical framework, but stressed that any revision, included in the incentive framework, should not discourage the development of medicinal products needed for the treatment of rare diseases.
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Response to Evaluation of patient rights in cross-border healthcare

1 Feb 2021

CBHC in Europe presents an opportunity as well as a challenge to ensuring that patients can access orphan medicines and ATMPs. In the case of rare diseases, limited patient populations mean that it would be unreasonable to have a treatment center, with highly specialised knowledge and equipment, in every European country. Furthermore, the complex nature of delivering ATMPs to patients limits the number of physicians and hospitals with the capabilities to provide these treatments and offer the appropriate level of disease management. These limitations make CBHC a critical component of ensuring patients’ access to ATMPs in Europe. The two different routes currently available to patients still present some challenges to accessing ATMPs abroad in a timely manner. Due to the upfront payments imposed by the CBHC Directive, cross-border treatment regulated by the ‘S2 route’ is currently the only practical option for cross-border treatment with ATMPs. It would not be feasible for patients to bear the total costs of their treatment and then subsequently seek reimbursement. Yet, this model also presents some flaws that can impede patients’ access: including, for instance, processing timelines for obtaining prior authorisation using the ‘S2 Form’ can vary greatly depending on which country is processing the application. There may also be difficulties in approving the application if the ATMP is not reimbursed in the home country despite being reimbursed in the treatment country. In some cases, health technology assessment and reimbursement decisions cannot take place if the product is not available in the home country. Additionally, European law does not address national issues in countries with restrictive budgets. In these countries, the cost of the treatment where it is being provided be restrictive for other European countries to cover. Lastly, cross-border is today not compatible with some existing pricing models – such as clawbacks or annual rebates that are accounted for by payers in the treatment country, not considering that costs for cross-border patients are borne by payers in the home country. We believe that all patients should have access to treatments, including orphan medicines and including ATMPs. Given its nature, A gene therapy for a rare disease will not be available immediately in every European country/administered in every country. Treatment will most likely instead be administered in treatment centres in a limited number of countries. Patients will need to travel, using the EU’s cross border mechanisms to access care. It is therefore imperative that a functioning and reliable route is available to patients. Tailored solutions meeting individual patients’ needs must be found through working together with treating physicians, treatment centres, patients, caregivers and social security entities. To achieve this and to address the current challenges with CBHC in Europe, PTC recommends to: • Provide better and above all more practical information to all stakeholders involved (HCPs, patients, companies, social security entities, etc.) on how CBHC can be accessed • Develop European-wide guidelines on what treatments should be approved to ensure harmonised access across countries • Improve HTA coordination to avoid multiple submissions and enable access of CBHC • Ensure mechanisms to allow innovative payment models to function across borders • Establish European-wide patient registries to ease long-term patient follow up
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Response to Revision of the EU legislation on medicines for children and rare diseases

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 disease patients.PTC recognised that after 20 years it is a good moment to take stock of the effectiveness of the Regulation and to assess where improvements should be made. However, we believe that this does not need to be done through legislative changes, given the arsenal of policy instruments the EU has at its disposal. At this stage, we do not comment on the four laid out in its impact assessment document – they all contain options which should be considered but there are many other elements which should be included, to address two critical questions: how to keep Europe attractive for the development and launch of orphan medicines and how to come up with therapies for 95% of rare diseases with no or few treatment options. The rare disease environment of the EU rests essentially on three pillars – the access environment, the regulatory approval, HTA review and P&R modalities of orphan drugs and the incentive schemes of the Regulation. Whilst PTC believes it is important to review the Regulation, we encourage the Commission and Member States, together with all stakeholders to work also on the first two elements to improve the situation for companies and rare disease patients. The Regulation is only one element in a set of measures that aim to improve the life of people with rare diseases. PTC proposes to widen the review of the Orphan Drug consultation to include a discussion to address the following issues: Improve the regulatory review and approval process for OMPs to address the specificities of these medicines such as challenges re choice of biomarkers, clinical data and HTA assessments. This also includes more acceptance of Real-World Evidence (RWE), conditional marketing authorisations, closer European collaboration on HTA and further alignment between regulatory and payers’ requirements. Work with stakeholders and Member States to develop and implement the HTA and P&R approaches which capture the broader benefits the OMPs, including ATMPs, bring to patients, caregivers, health systems and society. This will require a change in approach to value and P&R models, which all too often are geared to traditional, chronic treatments with ‘chronic’ payment intervals. Given the very low numbers of rare disease patients and the fact that they will need specialised treatment (which is particularly true in the case of ATMPs), it will be increasingly necessary to treat patients from one country in another country. Despite the existence of EU cross border health legislation, it is still challenging for patients to obtain treatment abroad. PTC encourages the Commission, in the context of the OMP Regulation review and the EU pharma strategy to include a review of this legislation with the aim to allow patients to receive treatment, throughout the EU, without barriers. An EU OMP policy needs to be supported and coordinated with national pharmaceutical and industrial policies . Such an approach allows for Europe to remain globally competitive re R&D research of innovative therapies for rare diseases patients. The valuation of the OMP Regulation highlights that OMPs are not equally accessible to patients in all EU countries. Access to medicines is primarily a Member State competence. Member States policies plans and strategies for rare diseases vary greatly across the EU. Indeed, only some countries have distinct processes for OMPs in place and delay in patient access to OMP across Member States can vary from few months to years. The review should include an assessment of Member States’ record in terms of formulating a rare disease policy to highlight best practices and to improve approaches.
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Response to Pharmaceutical Strategy - Timely patient access to affordable medicines

2 Jul 2020

At PTC, we dedicate ourselves to using ground-breaking science as we research rare diseases. We believe that by shifting our perspective, using the newest technologies available, we can find innovative ways to treat these diseases. We commit ourselves to pushing the envelope of what is possible, whether in a niche oncology platform, gene therapy, or an emerging treatment. However, we also seek to expand our internal treatment pipeline by analysing our scientists’ research and collaborating with other like-minded groups. This aids us in our mission to produce commercial products and medications that can help those with ultra-orphan/very rare disorders. It is important that the Commission develops an EU Pharmaceutical strategy. If the COVID-19 crisis has thought us one lesson, it is that major healthcare challenges can only be tackled if we all rise above our national borders and if all stakeholders work together. In developing such a strategy, the Commission should maintain a careful balance between the three corner stones of any health policy: supporting innovation in healthcare, ensure fast access for patients and sustainable healthcare budgets. It is important to develop an industrial policy for the pharmaceutical sector as the situation in Europe is not entirely positive. Whilst the number of young and very innovative pharmaceutical companies continues to grow, Europe is falling behind. Other countries like the US and China are performing better. There are different causes for these differences - more R&D funding, integrated and unified markets, closer research relations between academia, public and private sectors and not in the least better market conditions for approved therapies. The beauty of Europe lies in its diversity. At the same time, and despite significant efforts and improvements made over the last years, not in the least through European legislation etc, the EU's fragmentation continues and this causes major barriers to innovation abilities and access to treatment for patients: Despite clinical trial legislation revision, organizing clinical trials throughout Europe is still difficult, due to different national approaches and requirements Access to treatment for patients varies widely due to national P&R systems and different believes in concepts like access, affordability, and patient rights The fragmented EU regulatory system leads to inefficiencies, additional costs, and substantial burdens, for smaller companies Countries take different approaches to welcoming cutting-edge innovations, such as cell and gene therapies. For smaller companies, the commercial climate in Europe is challenging. Finally, we would like to offer some areas where further European Commission policy, together with the Member States, could make a difference for the innovative bio-pharmaceutical industry, characterized by many, smaller companies (with less resources), focusing on disease areas with a high unmet medical need (such as rare diseases) and using very innovative approaches (such as cell and gene therapies): Create a framework for the generation and acceptance of RWE to ensure that RWD is fully accepted by both payers and regulators as a robust data source Based on adopted EU legislation, create real, practical measures to allow patients to get treatment in centres of expertise, not necessarily in their home country Develop a dialogue between companies, regulators, and payers to ensure that the concept of conditional marketing authorisations is fully embedded in regulatory and access discussions Create a platform with stakeholders where issues affecting companies focusing on developing treatments for (very) small patient populations can be discussed, such as appropriateness of HTA models, data generation for regulatory and P&R submission, etc. Review the lessons learned from COVID -19 and establish which working methods used during the crisis, could be extended into normal working practices
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Meeting with Annika Nowak (Cabinet of Commissioner Vytenis Andriukaitis)

2 Jun 2016 · HTA