Evolving Landscape ATMPs

By Davide Integlia, General Manager of ISHEO & CRES Trials and Academy

This article explores the evolving landscape of Market Access for Advanced Therapy Medicinal Products (AMTPs). We address what constitutes an AMTP and how their approval is regulated. We also explore how pricing and reimbursement approaches differ for different markets and how the evolving regulatory framework may impact access to these therapies in the future.

AMTPs: A new category of therapeutic products

Advanced Therapy Medicinal Products (AMTPs) are a new category of therapeutic products derived from cells or tissues and potentially treat various diseases. AMTPs are currently developing for various indications, including cancer, cardiovascular disease and neurological disorders.

The development of AMTPs is a complex process, and you must overcome several important hurdles to bring these products to market. One of the biggest challenges is ensuring these products are safe and effective. In addition, AMTPs are often very expensive to produce; access to these treatments will be an important consideration when they become available.

The market access landscape for AMTPs is evolving, and companies currently explore new, different models. One approach is for payers to reimburse these products on a case-by-case basis. However, this approach can be challenging to manage from a budgetary perspective, and there is also the risk that some patients will not have access to these treatments if their insurer does not cover them.

Another considered approach is the creation of specialized funding mechanisms for AMTPs, which could involve the establishment of dedicated budgets or insurance pools specifically for these products. This approach would provide greater certainty around funding and ensure that all patients who could benefit from an AMTP would have access to it.

Whichever approach we take, it is clear that market access for AMTPs will be a complex and challenging issue. Therefore, there is a need for further research in this area to identify the most effective and equitable way to provide access to these potentially life-saving treatments.

The evolving market access landscape for advanced therapy medicinal products (AMTPs) is constantly changing. Therefore, staying up-to-date on this area’s latest news and developments is crucial. Here are some recent headlines that illustrate the current state of affairs:

Differences between Countries

In Europe, reimbursement decisions for AMTPs are made at the national level, meaning each country has its process and criteria making it difficult for companies to gain market access to their products.

The United States Food and Drug Administration (FDA) recently released new guidance on clinical trial design for AMTPs. This guidance intends to help sponsors design clinical trials that will provide adequate data to support FDA approval of these products.

Several countries, including France, Germany, and the United Kingdom, have established national registries for AMTPs. These registries track patient outcomes and collect data on these products’ long-term safety and efficacy.

As the field of AMTPs continues to evolve, so does the market access landscape. By staying informed about the latest developments, companies can be better prepared to navigate this ever-changing landscape on a global level.

Preclinical Research: Data-Generating Activities

Preclinical research is the first stage of a new therapy’s research and development process. It is typically conducted in labs using cell cultures or animal models. The goal of preclinical research is to generate data that will help assess the potential efficacy and safety of the therapy.

Preclinical studies are essential for the development of new therapies. They provide critical data on how a therapy works, its side effects, and whether it is safe and effective. In addition, this data supports clinical trials, the next step in research and development.

There are many types of preclinical studies, each with its strengths and weaknesses. For example, in vitro studies (conducted in lab-grown cells) can provide information on how a therapy works at a cellular level. However, they may not accurately reflect what happens in the human body. Animal studies can provide more realistic data, but there are ethical concerns about using animals for research purposes.

The preclinical research landscape constantly evolves as we develop new technologies and approaches making it an exciting area of research with great potential to improve the lives of patients with serious diseases.

Clinical Development

The clinical development of an AMTP is a critical step in bringing a new therapy to market. Therefore, we need to consider many aspects when designing and conducting clinical trials for AMTPs, including the unique characteristics of the therapy itself, the target patient population, and the regulatory landscape.

There has been a growing trend towards conducting clinical trials in real-world settings rather than controlled laboratory environments in recent years. This change allows for a more accurate assessment of how the therapy works in practice and can help to speed up the development process. However, ensuring that patients enrolled in clinical trials represent the overall target population is vital, as this can impact the trial results.

The regulatory landscape for AMTPs is constantly evolving, and it is vital to stay up-to-date with the latest changes. In particular, there has been an increasing focus on ensuring that AMTPs have a clear benefit/risk profile before they are approved for marketing. This fixation has led to several new initiatives to collect data on patient outcomes following treatment with AMTPs.

As market access for AMTPs becomes increasingly complex, working with experienced partners who understand all aspects of the development process is essential. At Isheo, we have extensive experience in Market Access and can help you navigate every step of the way.

Investigational Medicine Exclusivity (IME)

The sponsor may request exclusivity if a drug is designated as an orphan drug. The FDA may not approve another application for the same drug for seven years if granted. The sponsor may also request market exclusivity for drugs used to treat serious and life-threatening diseases, which, if granted, would prevent the FDA from approving any generic drug version for four years. Finally, if a new clinical investigation is necessary to support the approval of an application or supplement to an approved application, the sponsor may request market exclusivity for five years.

Patent Protection of AMTPs

Patenting an AMTPP is a complex task that requires a detailed understanding of the technology. Three key areas must be addressed when seeking patent protection for an AMTP:

The first is the jurisdiction in which you plan to file your patent application. It is essential to consider whether you want protection in multiple jurisdictions or just one.

The second is the type of patent you wish to file. Two main types of patents can be filed for an AMTP: a product patent and a process patent.

The third is the scope of protection you wish to obtain. This will be determined by the claims you include in your patent application.

It is important to note that there is no such thing as a “worldwide” patent. Instead, patent protection is territorial, meaning it only extends to the jurisdiction in which it is filed and granted. Therefore, it is crucial to carefully consider where you want to seek protection for your AMTPP before filing your application.

Marketing and Commercialization of AMTPs

As the healthcare landscape continues to evolve, so does the landscape of market access for advanced therapy medicinal products (AMTPs). In the past, AMTPs were often developed and commercialized without considering market access. However, as the field has matured, there is an increasing awareness of the importance of market access in determining the success of an AMTP.

There are many factors to consider when marketing and commercializing an AMTP.

First, it is vital to understand the reimbursement landscape for AMTPs. In many countries, AMTPs are not reimbursed by national health systems. This can pose a significant barrier to patient access. Therefore, it is essential to understand the reimbursement landscape before launching an AMTP.

Second, it is vital to consider the pricing of an AMTP. You must carefully consider pricing to ensure that patients have access to the treatment while also ensuring that the company can generate enough revenue to sustain itself long-term.

Third, clearly understanding the target market for an AMPTP is crucial. Who will be using the product? What are their needs? What are their demographics? Answering these questions can help guide marketing and commercialization efforts.

Finally, it is crucial to keep in mind that market access for AMTPs is constantly evolving. It is essential to stay up-to-date on changes in reimbursement policies and regulations governing AMTPs. By

Value-Based Pricing for ATMPs

As the healthcare landscape continues to evolve, so too does how market access for advanced therapy medicinal products (ATMPs) is managed. In recent years, there has been a shift from traditional fee-for-service models to value-based pricing models.

Value-based pricing is a pricing model that considers a product’s clinical and economic value. In other words, it considers the cost of manufacturing and developing the product and the potential benefits that the product may have for patients. This pricing model is becoming increasingly popular for ATMPs, as it offers a more sustainable and equitable way to reimburse companies for their products.