Giuliana Miglierini, Autore a NCF International

FDA, a discussion paper on the use of AI

Manufacturing

-

29 June 2023

FDA, a discussion paper on the use of AI

The FDA’s “Discussion Paper: Artificial Intelligence in Drug Manufacturing, Notice; Request for Information and Comments” was open to comments until 1 May 2023. It aims to address areas for consideration and policy development identified by the Center for Drug Evaluation and Research (CDER) scientific and policy experts associated with application of artificial intelligence (AI) to pharmaceutical manufacturing.

The focus is on advanced manufacturing. i.e. innovative pharmaceutical manufacturing technologies and approaches that may improve the reliability and robustness of the manufacturing process and the resilience of the supply chain. Artificial intelligence may play an important role in monitoring and controlling advanced manufacturing processes. The discussion paper addresses the main areas of consideration with respect to the FDA’s existing risk-based regulatory framework, as emerged from a comprehensive analysis of existing regulatory requirements applicable to the approval of drugs manufactured using AI technologies. The document focuses on drug products that would be marketed under a new drug application (NDA), abbreviated new drug application (ANDA), or biologic license application (BLA).

EuropaBio’s recommendations for biomanufacturing in the EU

Manufacturing

Giuliana Miglierini

-

22 June 2023

0

EuropaBio’s recommendations for biomanufacturing in the EU

The FDA’s “Discussion Paper: Artificial Intelligence in Drug Manufacturing, Notice; Request for Information and Comments” was open to comments until 1 May 2023. It aims to address areas for consideration and policy development identified by the Center for Drug Evaluation and Research (CDER) scientific and policy experts associated with application of artificial intelligence (AI) to pharmaceutical manufacturing.

The focus is on advanced manufacturing. i.e. innovative pharmaceutical manufacturing technologies and approaches that may improve the reliability and robustness of the manufacturing process and the resilience of the supply chain. Artificial intelligence may play an important role in monitoring and controlling advanced manufacturing processes. The discussion paper addresses the main areas of consideration with respect to the FDA’s existing risk-based regulatory framework, as emerged from a comprehensive analysis of existing regulatory requirements applicable to the approval of drugs manufactured using AI technologies. The document focuses on drug products that would be marketed under a new drug application (NDA), abbreviated new drug application (ANDA), or biologic license application (BLA).

Industrial associations joint statement on the proposed Product Liability Directive revision

Regulatory

Giuliana Miglierini

-

15 June 2023

0

Industrial associations joint statement on the proposed Product Liability Directive revision

The Federation of the European Pharmaceutical Industry Associations (EFPIA) and MedTech Europe (representing manufacturers of medical technologies) are among the industrial associations that jointly signed a statement on the Product Liability Directive (PLD) revision proposed by the EU Commission. The PDL directive was originally published in 1985 and revised in 1999; it addresses the product liability regime in order to balance manufacturers’ interest to innovate and consumers’ fair access to compensation.

According to the 11 associations that signed to joint statement, the current proposal might pose a risk for the European competitiveness, as it might result in a increased number of litigations and speculative claims. Instead of just addressing the challenges posed by new technologies such as AI and Internet of Things (IoT) devices, the proposals would involve a complete revision of the current framework. According to the industrial associations, the undergoing discussions by the EU institutions would be too rapid to achieve final satisfactory rules. This may result on a higher burden on national judicial and court systems to accommodate the new and unfamiliar rules, as well as in a low confidence in, or incentivise, manufacturing in Europe.

The statement also highlights six point of attention that should be considered in the revision of the PLD directive, including the liability of software, sufficient protection against malicious mass-claims and third-party litigation funding, the narrowing of key concepts (e.g. the definition of “damage”), safeguards on evidence disclosure orders, limiting the alleviation of the burden of proof, and reinstating monetary upper and lower thresholds.

Source: EFPIA

Associations’ comments on the Citizens Initiative to phase-out animal testing

Legislation

Giuliana Miglierini

-

6 June 2023

0

Associations’ comments on the Citizens Initiative to phase-out animal testing

The European Citizens Initiative “Save cruelty free cosmetics” asks for a legislative intervention, including the revision of the EU chemicals regulation and a roadmap to phase-out all animal testing for cosmetic ingredients in the EU before the end of the current legislative term. The BioMed Alliance, representing the European medical and research community, expressed a negative opinion on the Citizens Initiative. In a statement published in its website, the association highlights the detrimental effect the phase-out may have on biomedical research. The request for EU policy makers is, instead, to adopt a more gradual and longer-term approach to the reduction of the use of animals in health. The BioMed Alliance also recalls the many alternative non-animal methods under development, including in vitro, ex vivo, organoid and in silico models, and the fact the current regulatory pathway needs animal data before proceeding towards human studies.

EFPIA, representing the research-based industry, recalled from its website the successful voluntary non-profit collaboration launched in 2022 between four pharmaceutical companies (supported by EFPIA) and the European Chemicals Agency. Three further companies signed up to participate to the pilot project, and over 500 tests – containing physicochemical, toxicological and ecotoxicological substance data – have been uploaded containing information about the hazard properties of 94 substances. All data are publicly available and can be used to develop new methodologies so to decreasing the need for the use of animals in research.

Source: EU Commission, BioMed Alliance, EFPIA

EU Commission, proposed new rules for SPCs and compulsory licensing

Regulatory

Giuliana Miglierini

-

31 May 2023

0

EU Commission, proposed new rules for SPCs and compulsory licensing

Supplementary Protection Certificates (SPCs), compulsory licensing and Standard Essential Patents (SEPs) are central to the reform of the EU’s intellectual property (IP) legislation announced on 27 April 2023. The package is comprehensive of several Regulations; it will undergo assessment by the EU Council and Parliament before adoption. The proposed provisions will act in coordination with the Unitary Patent system which enter into force on 1 June 2023. The creation of a unitary SPC aims to overcome the current fragmentation, leading to differences in the expiry dates for patents and frequent litigations. The unitary SPC would be granted by the EUIPO, upon assessment of a single application. SPCs for each of the Member States designated in the application would also be granted.

The proposals on compulsory licensing during emergency or crisis situation aims to facilitate access to critical medicines and products. The new rules would be activated only upon the previous activation of another specific EU crisis instruments. A fair remuneration of IP owners is also included in the proposal. SEPs are mainly used by the ICT industry, as they support the development of standard-compliant products and technologies for the Internet of Things. SEP holders are committed to license these patents on fair, reasonable and non-discriminatory (FRAND) terms, allowing access to market to implementers. The proposed SEP licensing framework aims to create a balanced system and improve transparency and predictability, thus incentivising good faith negotiations between interested parties.

Source: European Commission

EU Parliament, a study on investments for Covid-19 Vaccines

R&D

Giuliana Miglierini

-

23 May 2023

0

EU Parliament, a study on investments for Covid-19 Vaccines

A study prepared for the special COVI committee of the European Parliament shares light on how the great amount of funds for the development of Covid-19 vaccines was used for R&D and the expansion of the production capacity. Nine different vaccines have been examined, and comparisons have been also made with the US situation. Outcomes of the analysis may provide critical insight to address existing and future challenges relating to variants and R&D incentives. According to the study, combined support in various form by external funds was in the range of €9 billion. Other approx. €21 billion supported Advanced Purchase Agreements (APAs). The estimated R&D expenditures sustained by companies for Covid-19 vaccines is in the range of €4-5 billion for the period 2020-2021. Funds made available by European institutions and national governments were mainly used to de-risk corporate investment. The US provided a greater funding to R&D that the EU plus Member States (respectively, 24.7% vs 1%).

Avoiding fragmentation and duplication of funding for R&D on Covid-19 vaccines, and ensuring public support for clinical development of the next generation vaccines (or vaccines protecting against unknown coronaviruses) are the main lessons learnt, together with the need to create a favourable regulatory and infrastructural environment for clinical trials and a careful examination of the conditionalities of future R&D grants and de-risking mechanisms.

Source: M. Florio, S. Gamba, C. Pancotti “Mapping of long-term public and private investments in the development of Covid-19 vaccines”, Study PE 740.072, EU Parliament, Policy Department for Economic, Scientific and Quality of Life Policies Directorate-General for Internal Policies, March 2023

The proposals for the reform of the pharmaceutical legislation

Legislation

Giuliana Miglierini

-

11 May 2023

0

The proposals for the reform of the pharmaceutical legislation

The European Commission has announced on 26 April 2023 its proposals for the revision of the pharmaceutical legislation. The package is comprehensive of a Directive, a Regulation and a Council Recommendation on antibiotic resistance; it will now undergo scrutiny by the EU Parliament and Council before adoption and entry into force. The objective of the EU Commission is to create a Single Market for medicines in the EU. Among major proposals, pharmaceutical companies should benefit of a 2 years extension of the market protection in case they launch a new product in all Member States at the same time. Incentives are also planned for medicines for rare diseases, unmet medical needs, comparative clinical trials, or new therapeutic indications. The standard period of regulatory protection would be 8 years, extending up to 12 years with the incentives (13 for rare diseases).

Simplification of the regulatory framework should reduce the maximum time to evaluate a dossier to 180 days, thanks also to electronic submissions. New procedures are planned to speed up approval of generics and biosimilars. Renewal of marketing authorisations should be abolished in many cases. Shortages and repurposing of medicines already on the market are also addressed. The EU Commission also supports a higher sustainability of pharmaceutical products in line with the objectives of the European Green Deal, to be tackled by stricter environmental risk assessment and measures to fight antimicrobial resistance.

Source: European Commission

Towards an EU Drugs Agency

Giuliana Miglierini

-

4 May 2023

0

The existing European Monitoring Centre for Drugs and Drug Addiction may soon become a fully-fledged EU Drugs Agency, following the provisional agreement on a proposed regulation reached by the Council presidency and the European Parliament. The agreement has now to be approved by both the European Council and Parliament before undergoing the formal adoption procedure.

The new EU Drugs Agency is expected to act more efficiently in tackling the illegal drugs market ad drug addictions at both EU and global level. The new legislative framework would allow the agency to improve the response capacity to new health and security challenges posed by illegal drugs. It would also provide support to member states.

Under the enhanced mandate, the EU Drug Agency would not only continue to collect, analyse and disseminate data. It would be also involved in developing general health and security threat assessment capabilities to rapidly identify new threats, and in conducting regular foresight exercises to identify future challenges. Poly-substance use could be a specific target, together with strengthening the cooperation with the national focal points, and the creation of a network of laboratories which will enable the agency to access forensic and toxicological information. The agency should also become competent for the development of evidence-based interventions, and its role in the area of international cooperation should be clarified.

Source: European Commission

MHRA’s new framework for clinical studies

Regulatory

Giuliana Miglierini

-

27 April 2023

0

MHRA’s new framework for clinical studies

UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has announced its new framework for clinical trials, based upon the results of a consultation which collected more than 2,000 responses. The final goal of the revision, which comes 20 years after the last changes of UK’s clinical trial system, is to allow a faster and easier approval and to run clinical trials in the UK, so to increase the attractiveness of the country for clinical research. As highlighted in the “Government response to consultation on legislative proposals for clinical trials” document, the reform of the UK clinical trials regulatory framework will continue focusing on patients and their safety as the main objective of all clinical trials. These should become more proportionate, streamlined and flexible without compromising on safety. The reform is expected to integrate the regulatory and ethics reviews of clinical trial applications. For trials where the risk is similar to that of standard medical care, for example, the “notification scheme” will enable a clinical trial to be approved without the need for a regulatory review.

Faster times to recruit patients will also be part of the reform, based on the results of a pilot phase. The review process of applications should end up in a maximum 30 days in general, with maximum 10 calendar days for a decision to be granted once the regulator has received any final information. The new regulatory framework shall accomodate all different types of trials and innovative designs, including decentralised trials, and it will align to ICH Good Clinical Practice principles so to facilitate international multi-site trials. A legal mandate to register the trial in a World Health Organisation (WHO) public register, and a requirement to publish a summary of results within 12 months of the end of the trial would support transparency of the entire process.

Mid-term achievements of EMA’s Regulatory Science Strategy to 2025

Regulatory

Giuliana Miglierini

-

20 April 2023

0

Mid-term achievements of EMA’s Regulatory Science Strategy to 2025

After five years from its launch, the European Medicines Agency published an interim report on the mid-term achievements of the Regulatory Science Strategy to 2025.

“The achievements highlighted in this report demonstrate that we have made considerable progress in advancing regulatory science to build a more adaptive regulatory system that encourages innovation in human and veterinary medicines,” said Emer Cooke, EMA’s Executive Director.

Five main areas of innovation for human medicines and three for veterinary products have been identified. Areas of progress for the human domain include fostering innovation in clinical trials; promoting use of high-quality, real-world data in decision making; reinforcing patient relevance in evidence generation; contributing to health technology assessment bodies’ (HTA) preparedness and downstream decision making for innovative medicines; and supporting developments in precision medicine, biomarkers and ‘omics. In the veterinary domain, actions were taken to transform the regulatory framework for innovative veterinary medicines. The development of new approaches to improve the benefit-risk assessment of veterinary medicinal products and the collaboration with stakeholders to modernise veterinary pharmacoepidemiology and pharmacovigilance were other key areas of attention.

Giuliana Miglierini

Newsstand