Market Access Strategies for ATMPs

What is Market Access?

Market Access focuses on enabling patients to access treatments beneficial to their health. When a treatment ultimately survives the long process of getting approval to go to the market, there is still no guarantee that that treatment will reach the patient to see the fruits of the time, energy, and money invested into that developing treatment. There could be multiple hurdles in getting the treatment to those who need them, such as issues with reimbursement, pricing strategies, and marketing. How would you convince payers that your treatment is superior to the one already on the market? Answering these questions is already challenging for traditional medicine but gets even more complicated and impactful in the case of more advanced therapies, such as Gene Therapy.

What are ATMPs?

Advanced therapy medicinal products (ATMPs) are a class of innovative medicinal products based on genes, tissues or cells, such as somatic cell and somatic gene therapy. ATMPs have the potential to cure, treat and diagnose a variety of diseases, including ones that have been difficult to cure in the past, such as various forms of cancer and Alzheimer’s disease. In addition, using genes, tissues or cells in treatments creates the opportunity for these treatments to be highly patient-specific. ATMPs can be classified into three sub-groups: 

• Gene Therapy Medicinal Products (GMTPs) are medicines that contain an active substance with recombinant nucleic acid used to regulate, replace, repair, add, or delete a genetic sequence. The therapeutic effect must be directly related to the recombinant nucleic acid substance.
• Somatic Cell Therapy is described as medicines containing or consisting of cells or tissues subjected to substantial manipulation in such a way that biological characteristics, functions or properties related to the clinical use have been changed.
• Tissue-engineered products are medicines containing engineered cells used for purposes such as regenerating, repairing or replacing human tissue.

Therapies falling in either of the categories have the potential to be revolutionary innovations for human health. However, there are multiple challenges involved with bringing ATMPs to the market. The development of ATMPs is complicated compared to traditional treatments. Additionally, it is challenging to incorporate ATMPs into legislation regarding market authorization.

Challenges in ATMP Legislation

One of the levels which ATMP developers find challenging is the field of legislation. According to a 2018 survey among ATMP developers (ten Ham et al., 2018), forty percent of ATMP developers in Europe experienced the composition of the regulatory dossier as challenging. In particular, Gene Therapy developers experienced more regulatory challenges. These challenges were mainly because GTMP developers must comply with Genetic Modified Organism (GMO) legislation. Although the purpose of GMO legislation originally was solely the agricultural and food sectors, GTMP developers also have to comply with these legislations on national and local levels. Compliance was described as confusing, delay-causing, and resource-intensive. 

Challenges in ATMP Market Access

Although ATMPs have the potential to treat a variety of diseases previously uncurable, there are still hurdles to overcome in their development. 

Because some of the diseases curable with ATMPs are relatively rare, it can be hard to find ample clinical trial participants. Additionally, the administration of ATMP to patients may sometimes be invasive or require surgery. In such cases, it is ethically not responsible to conduct a controlled, randomized, double-blind clinical trial, especially in the case of a life-threatening disease. 

Because of reasons like this, risk-benefit and cost-effectiveness analyses are challenging. The consensus is that potential toxicities are not as quickly determined in ATMPs as in traditional drugs. 

As a result, risk-benefit and cost-effectiveness analyses are difficult to establish, making risk mitigation even more critical in ATMP clinical trials. To investigate long-term safety and efficacy, we can generate real-world evidence (RWE) from real-world data (RWD).

Other challenges in bringing ATMP therapies to the market are related to reimbursement. Almost half (42%) of large companies surveyed by ten Ham et al. indicate that they experience uncertainty regarding reimbursement in the development process. 

The price of developing a novel therapy is high for both traditional medicine and novel therapies. However, since the patient populations of diseases treated with ATMPs are generally minimal, the cost per patient is also relatively high.

Evidence synthesis & Market Access for ATMPs

In Europe, the value of a drug and other health technologies depends on the institutionalized payers’ subjective perspective, and evidence synthesis strongly contributes to building this perception. The current Health Technology Assessment (HTA) is not designed for ATMPs. HTA is defined as a multidisciplinary activity that systematically examines the technical performance, safety, clinical efficacy and effectiveness, cost, cost-effectiveness, organizational implications, social consequences, and legal and ethical considerations (Velasco et al., 2002). Current aspects that are assessed when valuating a novel therapy include:

• Improvement of quality-adjusted life years.
• Cost-savings to the health system.
• Ease of use.

These aspects typically cover essential features of therapy. However, several relevant key improvements ATMPS can have on patients’ lives are often not included. The Special Task Force of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) has supported the addition of several new aspects that you should consider in the valuation of a novel therapy. Examples of novel aspects are insurance value, real option value, health equity, lifetime burden of illness, caregiver burden, and socioeconomic impact. 

Including these aspects may result in a more accurate valuation of therapies, meeting patients’ unmet needs. However, the limited amount of data regarding cost-offset for healthcare systems and society and long-term benefits remains a challenge, which makes strategies regarding Market Access for ATMPs even more important.

To ensure successful access to the market, identifying barriers regarding reimbursement as early as possible is crucial to ensure Market Access for ATMPs. And at the same time, reducing the time to market of ATMPs, and reimbursing them through an adequate system of pay for value through effective registries, can bring more investment rapidly for the clinical development of ATMPS. Health economists and HTA experts can then play a substantial role in helping European and national HTA bodies and regulatory authorities to make informed decisions for Market Authorization and reimbursement of ATMPs. Providing evidence-based Market Access Strategies built explicitly for ATMP is the only way to speed the development of these breakthrough life science technologies and have the chance to provide sooner than later access to a more significant number of treatments nowadays without a cure.