Veristat Global Policy Insights Q2

Index:  RECENT DEVELOPMENTS: EU | RECENT DEVELOPMENTS: US | other recent developments | Outlook

Topics in focus

USA – Platform technology designation program for drug development

The FDA issued draft guidance on platform technologies.

On 29 May 2024, the US FDA published draft guidance on platform technology, which provides details about the implementation of the Platform Technology Designation Program. Drug sponsors of pharmaceutical products can leverage platform technologies to produce multiple drugs or biological items using a standardized manufacturing process. The document outlines eligibility factors for receiving a platform technology designation, potential benefits of receiving a designation, how to leverage data from designated platform technologies, how to discuss a planned designation request as part of a milestone meeting, the recommended content of a designation request submission, and the review timelines for a designation request. Comments may be submitted by 29 July 2024

USA – FDA guidance on cancer clinical trial eligibility

The FDA issued draft guidance on laboratory values, washout periods and concomitant medications, and performance status for clinical trials.

On April 2024, the FDA issued three draft guidance documents to broaden eligibility in cancer clinical trials.

• The draft guidance on laboratory values for clinical trials advises against overly restrictive criteria, which can hinder patient diversity and participation. Sponsors should tailor eligibility criteria based on the investigational drug’s properties and especially avoid unnecessary exclusions if the drug is not expected to cause specific toxicities.
• Regarding washout periods and concomitant medications, the draft guidance suggests that these factors should be justified with data and should not exclude patients unnecessarily, particularly older ones who are more likely to be on multiple medications.
• The draft guidance on performance status (PS) emphasizes that eligibility criteria based on PS should be inclusive to better reflect the diverse patient population and provide more accurate efficacy and safety outcomes. Alternatives like comprehensive geriatric assessments and patient-reported outcome (PRO) data are recommended to evaluate overall health and predict treatment responses more effectively. The use of wearable devices for collecting objective activity data is also encouraged to enhance clinical assessments.

The consultation on the three draft guidances closed on 25 June 2024.

USA – FDA guidance to enhance bioresearch monitoring inspections

The FDA issued guidance on bioresearch monitoring (BIMO) inspections.

In early June 2024, the FDA issued two draft guidance documents on BIMO inspections, focusing on planning inspections and best communication practices with the FDA before, during, and after inspections. These guidances are applicable to sponsors of new drug applications (NDAs), biologics license applications (BLAs), and supplements, and align with the BIMO technical conformance guide for submitting clinical study information.

The first guidance emphasizes timely submission of accurate data to facilitate early inspection planning, while the second outlines procedures for handling records and communication during inspections, in compliance with the Food and Drug Omnibus Reform Act of 2022. The BIMO program aims to ensure the quality and integrity of data and safeguard the rights of participants in FDA-regulated research through comprehensive monitoring. Companies subject to a BIMO inspection must grant FDA access to all relevant information and facilities and respond promptly to inspection findings with well-organized corrective actions. Comments on the documents can be submitted until 5 August 2024.

USA – Progress on BIOSECURE Act in Congress

Congress makes progress on BIOSECURE Act discussions.

In May 2024, the BIOSECURE Act (H.R. 8333) was discussed by the House Committee on Oversight and Accountability. The Act aims to restrict federal procurement from certain biotechnology companies deemed to be security risks. The House Committee voted on an amended version of the bill, introducing key changes that consider industry concerns raised earlier this year.

Introduced changes include an eight-year transition period for contracts established before the bill becomes effective, a safe harbor clause clarifying that biotechnology equipment or services that were formerly, but are no longer, produced or provided by biotechnology companies of concern are excluded, and a clarification of the scope concerned by the prohibition. These changes to the bill make it more aligned with the Senate version (S.3558).

Separately, the National Security Commission on Emerging Biotechnology expressed strong support for the BIOSECURE Act, emphasizing its importance for national security and urging Congress to promote a robust biotechnology innovation system to complement the bill’s provisions. The legislation now awaits further debate and voting in both the House and Senate.

EU – Results of the EU parliamentary elections

The provisional results of the European Parliament elections are in, showing shifts in party seats.

The elections of the European Parliament resulted in a shift to the right, as anticipated by the polls. However, a coalition of center-right (EPP), liberal, and socialist groups is likely to secure the 361 votes needed for a majority in the 720-seat EU Parliament to elect top EU officials and pass legislation. The EPP remains the largest, gaining 11 seats, while the greens and liberals each lose about 20 seats compared to 2019. The next plenary session of the new legislative term will be held from 16 to 19 July 2024.

Key health-related priorities of the main political groups at the EU Parliament:

• EPP: Aims to build on the success of the Beating Cancer Plan and supports an action plan for cardiovascular diseases as well as Parkinson's and Alzheimer's.
• Socialists and Democrats (S&D): Focus on universal access to quality health, fair and transparent medicine pricing, and European cooperation on health threats. 
• Alliance of Liberals and Democrats (ALDE): Less concrete references to health with passing references to digitalization in healthcare and attracting specialists in various fields.
• European Conservatives and Reformists (ECR): No mention of health or consider health a national competency but the voting records reveal support for health files.
• The European Greens: Light mention of health priorities but the voting record demonstrates a strong support for health files.
• Patriots for Europe/Identity and Democracy group (ID): A new political group was created on 8 July 2024, bringing together the previous ID group, France’s Rassemblement National (RN), Hungary’s Fidesz party, Italy’s Lega, Austria’s FPÖ, inter alia. Priorities have not yet been made available. 

EU – Substances of Human Origin (SoHO) Regulation adopted

The revised SoHO legislation applicable to blood, tissues, and cells is final.

The SoHO Regulation was adopted by the Council of the EU on 27 May 2024. It aims to enhance the safety of blood, tissues, and cells in healthcare and seeks to achieve its objectives by:

• Establishing an EU-level SoHO coordination board to assist Member States in implementing the regulation
• Introducing standardized procedures for the authorization and assessment of SoHO preparations across the EU
• Mandating Member States to appoint a national SoHO authority to ensure unified oversight
• Creating an EU IT platform for the exchange of information and activities related to SoHO
• Defining a common standard for voluntary and unpaid donations

The Regulation was signed by both the Council and the European Parliament Presidents on 13 June 2024 and will take effect following its publication in the EU’s Official Journal.

EU – Adoption of AI Act & launch of AI Office

The EU groundbreaking AI Act is final, and the European AI Office has been established.

The Artificial Intelligence (AI) Act received final approval from the Council of the EU on 21 May 2024, marking a significant milestone in the regulation of AI systems across Europe. Key provisions of the new law include:

• Registration of high-risk AI systems in an EU database
• Assessment of AI systems' impact on fundamental rights
• Penalties for infringements based on a percentage of a company's turnover
• Regulatory sandboxes to facilitate the testing of innovative AI systems

The Regulation will be signed by both the Council and the European Parliament and will take effect following its publication in the EU’s Official Journal.

The European Commission established the AI Office, which will play a pivotal role in regulating AI developments within the EU, ensuring the coherent implementation of the AI Act through activities such as:

• Collaborating with Member States and experts
• Working with the European Artificial Intelligence Board
• Supporting governance bodies and conducting AI model evaluations
• Preparing guidelines and coordinating codes of practice

Other recent developments

USA – FDA unveils initiative on drug safety

In June 2024, the FDA's Center for Drug Evaluation and Research (CDER) launched the Emerging Drug Safety Technology Program (EDSTP). The EDSTP focuses on the use of AI and other emerging technologies in pharmacovigilance (PV) and is part of CDER’s multifaceted approach to enhance mutual learning of where and how specific innovations, such as AI, can best be used across the drug product lifecycle. The EDSTP has three goals:

• Serve as the central point of contact for discussion between industry and CDER on the use of AI and other emerging technologies in pharmacovigilance (PV)
• Enable knowledge management and knowledge transfer within FDA specific to the context of AI or other emerging technologies used in PV
• Understand the context of the use of AI and other emerging technologies in PV to inform potential regulatory and policy approaches within PV

To further these goals, CDER also introduced the Emerging Drug Safety Technology Meeting (EDSTM) Program, which offers a collaborative environment for industry experts, researchers, and technology developers to present their innovations related to the use of AI and other emerging technologies and its potential application in PV, and receive feedback from the FDA.

USA – FDA, Health Canada, and MHRA identify further principles for ML in medical devices

The FDA, Health Canada, and the Medicines and Healthcare products Regulatory Authority (MHRA) have further identified guiding principles for transparency for machine learning-enabled medical devices (MLMDs). These principles aim to ensure that such devices are safe, effective, and reliable for public use. Key aspects of the guiding principles include:

• Transparency: Developers are encouraged to clearly document and communicate the functionality and limitations of machine learning algorithms used in medical devices
• Patient Safety: Emphasis is placed on continuous monitoring and updating of algorithms to maintain safety and effectiveness over time
• Ethical Considerations: Principles call for the fair and unbiased use of data, ensuring that machine learning models do not perpetuate or exacerbate existing health disparities

It is stated that, by adhering to these principles, developers can contribute to a more trustworthy and robust ecosystem for machine learning-enabled medical devices.

Furthermore, the FDA has recently updated various pages addressing AI-related topics in healthcare, including:

• Addressing the Limitations of Medical Data in AI
• Identifying and Measuring AI Bias for Enhancing Health Equity
• Evaluation Methods for AI-enabled Medical Devices: Performance Assessment and Uncertainty Quantification
• Performance Evaluation Methods for Evolving AI-Enabled Medical Devices
• Regulatory Evaluation of New Artificial Intelligence AI Uses for Improving and Automating Medical Practices
• Methods for Effective Post-market Monitoring of AI-Enabled Medical Devices
• Artificial Intelligence Program: Research on AI/ML-Based Medical Devices

USA – FDA new guidance and requirements relevant for cell and gene therapy products

Over the last quarter, the FDA released several documents that are relevant to cell and gene therapies. In April 2024, the FDA released draft guidance detailing safety testing protocols for human allogeneic cells used in cell-based medical products. This guidance provides recommendations for sponsors on supporting an Investigational New Drug (IND) application or a Biologics License Application (BLA). It emphasizes a risk-based analysis, considering factors such as cell expansion potential and the reagents used in cell culture. Comments can be submitted until 29 July 2024.

A revised draft guidance on promotional labeling and advertising considerations for prescription biological reference and biosimilar products was published in April 2024. It focuses on ensuring that promotional communications for biologics and biosimilars are accurate, truthful, and non-misleading. The FDA seeks to refine how data and information are presented in promotional materials.

Separately, the FDA released draft guidance for the use of human- and animal-derived materials in cell and gene therapies (CGTs) and tissue-engineered medical products (TEMPs). This guidance provides safety, quality, and identity recommendations for these materials and outlines required Chemistry, Manufacturing, and Controls (CMC) information for investigational new drug applications. Stakeholders can comment on the draft until 29 July 2024.

USA – FDA launches CDER Center for Clinical Trial Innovation

In May 2024, the FDA’s CDER launched the Center for Clinical Trial Innovation (C3TI) to promote clinical trial innovation through enhanced internal and external communication and collaboration. C3TI will be a central hub within CDER that supports innovative approaches to clinical trials that are designed to improve the efficiency of drug development. Initial projects will focus on point-of-care trials, Bayesian analyses, and selective safety data collection.

USA – Rules and guidance for IVDs and public health emergencies

In April 2024, the FDA issued a final rule clarifying that in vitro diagnostics (IVDs), including those manufactured by laboratories, are classified as medical devices under the FD&C Act. This rule initiates a four-year phase-out of the FDA's general enforcement discretion for laboratory developed tests (LDTs) and introduces targeted enforcement discretion policies for certain IVD categories.

Separately, the "Coordinated Agency Response Enhancement Act" (CARE Act) was introduced in the House of Representatives in April 2024. The bill aims to establish an after-action program and a risk communication strategy within the Public Health Service Act to improve responses to public health emergencies and enhance communication about health risks

Draft guidance for more diversity in clinical studies

The US FDA published a draft guidance on 26 June 2024, to improve enrollment of participants from underrepresented populations in clinical studies. The guidance relies on Diversity Action Plans (DAPs), which sponsors must submit for certain clinical studies. The guidance further details:

• Form, content, and use of DAPs
• Applicable medicinal products and clinical studies
• Timing and process for submitting DAPs
• Process/criteria according to which the FDA will dispense waivers for the submission of action plans

Online or written comments can be submitted until 26 September 2024.

EU – Progress on the revision of the EU pharmaceutical framework

The European Parliament adopted its position on revising the EU pharmaceutical framework in April 2024. MEPs endorsed measures from the Committee on Environment, Public Health and Food Safety (ENVI), including simplified EMA marketing authorization, regulatory sandboxes, and management of shortages.

Key provisions include a minimum 7.5 years of regulatory data protection, up to 11 years of market exclusivity for orphan drugs addressing high medical needs, and flexible access to treatments across Member States. Transferable data exclusivity vouchers for antimicrobials are also maintained. Inter-institutional negotiations will commence after the European elections.

 On 21 June 2024, the Employment, Social Policy, Health and Consumer Affairs Council (EPSCO) convened to deliberate on the incentive system within the pharmaceutical package. The discussion focused on potential adjustments to regulatory data protection periods, market access incentives, and incentives addressing unmet medical needs. While considerable progress was made, certain issues remain unresolved:

• Most ministers expressed support for modulation, provided the framework was clear, predictable, and respected the need for innovation, while a notable minority supported the current framework
• Several ministers stressed that access should not be linked to pricing and reimbursement, taking into account differences between national systems
• All ministers agreed that a solution was needed for access, yet there were mixed views on how this should be achieved, with some expressing support for an approach that incentivized companies to facilitate access, while others preferred to impose an obligation on companies
• Ministers expressed support for an unmet medical need (UMN) incentive but felt that the definition and criteria of this incentive should be further discussed, specifically regarding achieving legal certainty and objectivity in its application

EU – Recent developments around MDR and IVDR

On 30 May 2024, the Council of the EU adopted a regulation extending transitional periods for certain IVDs, allowing a phased roll-out of the EUDAMED database, and mandating prior notice for supply interruptions. The regulation amends the In Vitro Diagnostic Medical Device Regulations (MDR and IVDR). This regulation entered into force on 9 July 2024, after its publication in the EU Official Journal. Key changes include:

Extending IVDR transition periods until:

• 31 December 2027 for class D IVDs
• 31 December 2028 for class C IVDs
• 31 December 2029 for class B IVDs and class A sterile IVDs
• Gradual roll-out of EUDAMED modules, with device registration beginning in the second hall of 2025
• Requiring manufacturers to inform public authorities and clients about potential supply interruptions of critical devices/IVDs

Voices in favor of wider reforms of the MDR and IVDR are getting louder. Most recently, European People's Party (EPP) Member of the European Parliament (MEP) Peter Liese echoed industry concerns and put forward several proposals to revise the legal framework, including:

• Establishing a new European Medical Devices Office
• Changing the recertification timeline for high-risk devices (Class III/most Class IIb implantables)
• Abolishing recertification of lower-risk devices by Notified Bodies
• Making targeted amendments to ensure the MDR functions well for Small and Medium Enterprises (SMEs)
• Implementing good administration principles for Notified Bodies
• Streamlining the designation and surveillance process of Notified Bodies

Separately, the Commission updated and published revised guidance Medical Device Coordination Group (MDCG) 2022-4 on 24 May 2024, focusing on appropriate surveillance of medical devices, specifically legacy devices certified under the Medical Device or Active Implantable Medical Devices Directives (MDD or AIMDD). The Commission also released several form templates for preliminary re-assessment reviews of MDCG guidance documents by Notified Bodies:

• Preliminary re-assessment review template - MDR: MDCG 2024-6 and MDCG 2024-7
• Preliminary assessment review template - IVDR: MDCG 2024-8 and MDCG 2024-9

EU – Launch of Critical Medicines Alliance

The Critical Medicines Alliance to address medicines shortages was officially launched by the Health Emergency Preparedness and Response Authority (HERA) in collaboration with the Belgian Presidency of the EU Council, as announced by the European Commission on 30 April 2024. The Alliance operates on a five-year plan with currently over 250 registered members and welcomes new members on a rolling basis.

EU – Update on clinical trial framework

18 June 2024, the new version of the Clinical Trial Information System’s (CTIS’) public portal was officially launched. The new version of the portal coincides with the application of the revised transparency rules for the CTIS. One of the key changes is the earlier availability of information on authorized clinical trials. Importantly, the new rules eliminate the previously available deferral mechanism, which allowed clinical trial sponsors to delay publishing certain data and documents for up to seven years after a trial’s completion to protect commercially confidential information.

Switzerland – Launch of talks on bilateral relations

In a significant development for Swiss-European Union relations, the Federal Council of Switzerland finalized its negotiating mandate by consultations with various stakeholders including Parliament, cantons, and socio-economic partners. Following preparatory discussions, the EU Council authorized bilateral negotiations based on the EU Commission's recommendation put forth on 20 December 2023. With both sides equipped with negotiating mandates, formal discussions commenced in March, marking the beginning of what promises to be a comprehensive negotiation process. The negotiations will cover multiple facets simultaneously, aiming to address various elements of the bilateral package. Both Switzerland and the EU are committed to reaching a comprehensive agreement by the end of 2024 underscoring the shared commitment to strengthen bilateral ties and navigate key issues affecting both parties and progress towards a comprehensive and mutually beneficial agreement.

Switzerland – Upcoming changes to clinical trials legislation

Amendments to the Swiss legislation on clinical trials are expected to come into force on 1 November 2024. Recently adopted by the Federal Council on 7 June 2024, these changes will affect the four implementing Ordinances associated with the Human Research Act (HRA). These ordinances provide detailed specifications on ethical, scientific, and legal requirements essential for conducting human research in Switzerland:

• ClinO (Ordinance on Clinical Trials): This Ordinance governs clinical trials, excluding those involving medical devices, ensuring rigorous standards for participant safety and research integrity
• ClinO-MD (Ordinance on Clinical Trials with Medical Devices): This Ordinance specifically addresses clinical trials involving medical devices, ensuring compliance with stringent regulations to protect participants and ensure data reliability
• HRO (Ordinance on Human Research): This Ordinance covers all other forms of human research not classified under clinical trials, emphasizing ethical principles and scientific validity in research methodologies
• OrgO-HRA (Ordinance on Organisational Aspects of the Human Research Act): This Ordinance focuses on the organizational framework and procedural aspects of conducting human research, ensuring consistency and adherence to legal standards

UK – MHRA issues strategic approach to AI

On 2 May 2024, the MHRA outlined its strategic approach to AI, focusing on principles such as safety, transparency, fairness, governance, and contestability. The MHRA plans to issue further guidance on AI as a medical device, including best practices for development and deployment. In perspective to these developments, the MHRA also launched a regulatory sandbox for AI as a medical device (AIaMD) called AI Airlock.

UK – Updates on medical devices and IVDs

On 21 May 2024, the MHRA announced a step towards enhancing the safety of high-risk IVDs and launched a four-week consultation aimed at gathering feedback from stakeholders. The consultation seeks input from stakeholders across the healthcare and medical device sectors, including manufacturers, healthcare professionals, and patient groups. The goal is to refine regulations and ensure that high-risk IVDs meet the highest safety standards, thereby protecting patient health and improving diagnostic accuracy.

The MHRA also unveiled a proposed framework for the international recognition of medical devices in May 2024. This framework represents a pivotal advancement in the regulatory landscape for medical devices in Great Britain. By aligning with international standards, the MHRA aims to:

• Ensure that medical devices, including high-risk IVDs, are safe and effective for public use
• Enable faster access to cutting-edge medical technologies and innovations in the Medtech sector
• Increase Great Britain’s attractivity as a market for medical device manufacturers

Outlook for Q3/Q4 2024

European Union

• Nomination of Commissioners and Commission President
• Nominations of MEPs in committees and (co-) rapporteurship & European Parliament Presidents
• Council of the EU’s negotiation position and start of interinstitutional negotiations of the pharmaceutical legislation package

Switzerland

• Revision of the Therapeutic Products Act (TPA)

USA

• Meeting of the Oncologic Drugs Advisory Committee Meeting Announcement (25 July 2024)
• Adoption of guidance on AI drug development

Global

• Adoption of ICH M13A BE testing guideline
• Final negotiations on WHO Pandemic Agreement

OUTLOOK: EU | OUTLOOK: US | OUTLOOK: Switzerland | Outlook: UK