Advanced Therapies – the uncharted path of drug development

Advanced therapy medicinal products (ATMPs) are a fast-growing field of innovative and very complex biological products. This novel class of products carry a game changing potential for the treatment of severe conditions, even curative, for which we have no appropriate treatment options today. Translation of an ATMP from research to market authorization is a lot about risk. Your project can be successfully managed if the potential challenges are clearly understood in advance. However, we know that it might not always be the case. At some point you might need urgent solutions.

Success of an ATMP depends on the rigorous control of the manufacturing process and product specifications, which may involve limitations inherent to the complex character of ATMPs. Individual variability of cells used for production may in worst cases confound non-clinical and clinical results. NDA could either provide you with a regulatory roadmap right from the start, or we can select the best experts to help if you have very specific urgent questions regarding CMC, non-clinical, clinical or regulatory aspects. We can also help to generate the scientific rationale for the pharmacovigilance and risk management of your product.

Safety when the models are not enough

Often balance between efficacy and safety is very unique and delicate for each individual ATMP. These unique safety features may be difficult to anticipate before human exposure and mitigation of all risks may not be achievable through standardization and/or quality control of the product. In vivo non-clinical safety studies may be of limited value given the complexity of these products. However, data from such studies are normally required from the regulatory agencies and especially when the foreseen risks are regarded as unacceptable, for instance the risk of tumor formation following treatment with cell based ATMPs and integrational mutagenesis/oncogenesis following treatment with gene therapy products. In such cases regulators will request long-term post-marketing surveillance of efficacy and in particular safety that in many cases exceed the duration traditionally imposed on pharmaceuticals such as 5-15 years. To overcome this challenge setting up disease/treatment specific registries will be needed.

By collaborating with NDA, we can strengthen you with the legal framework, regulatory and scientific guidance to enhance your manufacturing and quality development and non-clinical safety package in order to have your product move faster to clinical evaluation.  NDA could support you to identify any possible gaps in the CMC, non-clinical and clinical data packages, by enhancing your risk analysis and by designing studies needed to address possible shortcomings. 

How to value a potential cure

ATMPs are often one-time treatments which impose potential challenges to the current system of financing, pricing and reimbursement of drugs. Meeting reimbursement requirements for supporting evidence at launch, can be demanding for ATMPs and can in some cases need consideration of new payment models. Engagement with market access stakeholders early in clinical development is needed to understand evidence requirements and how challenges with clinical evidence generation can be addressed and planned accordingly. Early engagement with payers to agree on new payment models can facilitate faster market access. At the NDA we have knowledge regarding the value of life-long benefits and the latest standards in health care payment models.

Just as the progress from ‘one-size fits all’ towards personalized biological medicines continues, the associated regulatory tools will continue to evolve. Our regulatory experts are updated on new technologies and novel scientific findings that in different ways affect the regulatory landscape. As a manufacturer, you will need a solid regulatory strategy to align your ATMP development with the regulatory expectations and with NDA as your trusted partner, we will solve it together.

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