Advanced Therapy Medicinal Products

Advanced therapy medicinal products (ATMPs)/cellular and gene therapy (CGT) products are a rapidly growing innovative class of biopharmaceuticals because of their potential to offer therapeutic breakthroughs for many ailments, especially life-threatening cancers and rare serious inherited disorders for which conventional medicine and various other modalities have not been completely effective.

These products are being increasingly researched and emerging as promising therapies because of their unique ability to address unmet medical needs; health authorities across the globe are actively encouraging their development. Because of the nature, complexity, and novelty of these products, the industry and regulatory authorities are constantly contributing toward evolving the guidelines and approach to handle these products. The classification of cell and gene products is complex; moreover, the respective terminologies also vary in the United States and the European Union.

ATMP’s Classification as per EMA

Gene therapy medicines
  • Gene therapy medicines Contain genes that lead to a therapeutic, prophylactic, or diagnostic effect. They work by inserting ‘recombinant’ genes into the body, usually to treat a variety of diseases, including genetic disorders, cancer or long-term diseases. A recombinant gene is a stretch of DNA that is created in the laboratory, bringing together DNA from different sources.
Somatic-cell therapy medicines
  • Somatic-cell therapy medicines Contain cells or tissues that have been manipulated to change their biological characteristics or cells or tissues not intended to be used for the same essential functions in the body. They can be used to cure, diagnose or prevent diseases.
Tissue-engineered medicines
  • Tissue-engineered medicines Contain cells or tissues that have been modified so that they can be used to repair, regenerate, or replace human tissue.
Combined ATMPs (cATMPs)
  • Combined ATMPs (cATMPs) Some ATMPs may also contain one or more medical devices as an integral part of the medicine, which are referred to as combined ATMPs. An example of this is cells embedded in a biodegradable matrix or scaffold.1

ATMP’s Classification as Per EMA

Gene therapy medicines

Gene therapy medicines Contain genes that lead to a therapeutic, prophylactic, or diagnostic effect. They work by inserting ‘recombinant’ genes into the body, usually to treat a variety of diseases, including genetic disorders, cancer or long-term diseases. A recombinant gene is a stretch of DNA that is created in the laboratory, bringing together DNA from different sources.

Somatic-cell therapy medicines

Contain cells or tissues that have been manipulated to change their biological characteristics or cells or tissues not intended to be used for the same essential functions in the body. They can be used to cure, diagnose or prevent diseases.

Tissue-engineered medicines

Contain cells or tissues that have been modified so that they can be used to repair, regenerate, or replace human tissue.

Combined ATMPs (cATMPs)

Contain cells or tissues that have been modified so that they can be used to repair, regenerate, or replace human tissue.

Definitions of Cellular and Gene Therapies as per US FDA

Human gene therapy product

FDA generally considers human gene therapy products to include all products that mediate their effects by transcription or translation of transferred genetic material or by specifically altering host (human) genetic sequences. Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use. All gene therapies are considered as ATMPs.

Somatic cell therapy/cellular products

Somatic cell therapy is defined as any autologous, allogeneic, or xenogeneic cells that have been propagated, expanded, selected, pharmacologically treated, or otherwise altered in biological characteristics ex vivo to be administered to humans and applicable to the prevention, treatment, cure, diagnosis, or mitigation of disease or injuries.

Combination products

A combination product is a product composed of any combination of a drug and a device; a biological product and a device; a drug and a biological product; or a drug, device, and a biological product

ATMPs: The Future of Medication

Major Safety Challenges Faced by ATMP Companies

Clinical Safety and Pharmacovigilance

For sponsors, early planning of setting up a pharmacovigilance system with a clear direction for what can be outsourced and what will be handled by internal teams may be valuable. The in-house focus at organizational level should be on strategic initiatives and predictive decision-building and a responsive safety strategy for successful clinical development and approval.

APCER’s Safety and Pharmacovigilance experts are aware of an increased risk of undesirable and unpredictable outcomes that may present as delayed adverse event(s). It may not be possible to collect all safety and efficacy data during clinical investigations, considering the limited number of subjects enrolled in clinical trials for advanced therapies. Therefore, APCER Life Sciences (hereinafter referred to as APCER) can help with collection and analysis of safety data for maintaining the overall risk–benefit balance, additional pharmacovigilance activities for long-term monitoring (LTFU) for safe and effective use, and measures to minimize risks (risk evaluation and mitigation strategies [REMS], risk management plan [RMP], and additional risk-minimization measures [aRMMs]), with highly specific data on quality, safety, and efficacy.

Risk Management

An efficient risk minimization system establishes a methodological framework for the pharmacovigilance plan and the characterization of risk–benefit analysis for any ATMP. While creating a risk management strategy, the risks that may occur due to the nature of ATMPs need to be considered. To address important safety concerns and improve the risk–benefit profile of certain ATMPs, aRMMs and REMS need to be implemented in addition to routine measures in the European Union and the United States, respectively.

 APCER can support in end-to-end risk management activities, including implementation, tracking, assessment, and modification of the implemented risk management systems. APCER’s team of experts can support in preparation and implementation of various RMM tools, such as educational material, and control access tools as required.

Medical Information and Medical Affairs

Clinical administration of advanced therapies requires specialized medical centers and highly qualified personnel. Physician training in clinical procedures specific to these products must be coordinated with product marketing and handling and use, as well as with post-market surveillance reporting. APCER’s Medical Information and Medical Affairs team provides the medical resources you need to listen to the market, respond to opportunities, and communicate with various stakeholders, including patients and healthcare professionals, promptly and accurately.

Medical Writing

APCER’s Medical Writing department has an experienced team of writers with clinical, regulatory, and scientific writing expertise to help companies document efficacy and safety data and stay in compliance worldwide throughout the product life cycle.

APCER’s Medical Writing team with experience across diverse therapeutic areas can provide ATMP/CGT development support at all phases throughout the life cycle of these products. The team can also assist in authoring and reviewing clinical and regulatory documents, such as protocols, investigator’s brochures, clinical study reports, layman summaries and individual patient narratives, briefing documents for interaction with regulatory agencies, applications for various drug designations and expedited approval pathways (priority review, ODD, BTD, etc.), and eCTD submission modules for IND or CTA and BLA or MAA application filing.

The Medical Writing team analyzes, interprets, and summarizes scientific data and helps the sponsors with publications and presentation at appropriate forums. Additionally, RMMs for these products may include HCP educational material and patient wallet card.

Regulatory Affairs

Considering the nature and complexity of these products, an appropriate classification of advanced therapies is very crucial for regulatory oversight, quality assurance, and patient safety.

Owing to the complexity and novelty of ATMP/CGT, The European Union and the United States have published multiple guidelines for specific expectations and created regulatory pathways for approval. Both EU and US regulatory agencies allow requests for informal scientific advisory meetings throughout the product life cycle. ATMP/CGT developers are encouraged to consult the agencies for identifying and assessing the regulatory risk associated with the product. Joint US FDA and EMA advice could also be sought; this strategy is mostly useful to obtain advice on clinical trial design and the selection of clinical end points.

Regulatory Team

APCER’s Regulatory team is capable to provide support to biopharmaceutical companies during all phases of the product life cycle. The foray of services begins with the classification of ATMPs. The team can prepare the package by compiling the required formats followed by submission to the EU regulators. The team provides support for authoring and verifying the quality information required to be included in the Orphan Drug Designation Request along with the compilation and submission of the request to the US FDA. Similarly, the team can compile pre-submission and submission requests in coordination with the medical writing and clinical experts and submit the ODD requests to EMA. The team provides support for requesting and participating in different kinds of advice meetings with US and EU regulators, viz. PDUFA and INTERACT Meetings with the US FDA and Scientific Advice Meetings with the EMA. The team has the capability to compile meeting requests, as well as meeting/briefing packages in compliance with the latest regulatory expectations in close coordination with the medical writing and clinical experts and submit them to the regulatory authorities via appropriate routes. During the clinical trial phase(s), The team can author and review the quality/CMC sections of the IND and IMPD for the US and EU markets. The compilation of the complete IND/CTA package, as well as electronic publishing of the package (wherever required), followed by the submission of the application to the regulators is conducted via appropriate routes.

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