Cell and Gene Therapy in a Nutshell

Cell and gene therapies are a groundbreaking new concept that offers many patients with rare diseases a healing potential. pharmaLevers has tried to summarize the most important findings for the market access of Cell and Gene Therapies: ?

Not all about Cure

For some, healing means a lifelong treatment effect – for others, a multi-year benefit. One-shot therapy is possible, but for certain diseases and vectors, multiple doses may be required.?

It works

Despite many uncertainties, the good news is that more than twenty cell and gene therapies have received market approval so far. Similarly, cell and gene therapies are usually reimbursed based on special agreements.?

Economically but limited

the cumulative budget impact may not be affordable

Despite a high price, many cell and gene therapies have a reasonable price-performance ratio and are therefore economical. However, the associated full costs for the health system are cumulatively hardly affordable, which is why the full benefits can usually not be rewarded. ?

Value & Uncertainty

a combination of managed entry agreements is more attractive to payers than annuity payments. ?

There is currently uncertainty about the degree of efficacy and benefit duration. Therefore, cell and gene therapies are usually reimbursed conditionally. The upfront risk for the payers is considerable. Therefore, managed entry agreements are used. A combination of Managed Entry Agreements is more attractive to payers than annuity payments. ?

Decisive Approaches

Compared to other therapies, payers have greater influence and decision-making power in cell and gene therapies, as hospital reimbursement can also be decisive in addition to the product price. Since these are high-price patients, price negotiations and performance controlling are a good investment for the payers.

payers have greater influence and decision-making power in cell and gene therapies

• There is no one-size-fits-all approach. Cell and gene therapies can vary considerably in terms of disease, required vector, cure level, dosage, and long-term benefits for the healthcare system.
• It is important that stakeholders learn a differentiated assessment and appraisal. It also follows that a specific combination of managed entry agreements should be chosen for each cell and gene therapy.

In the following newsletters, the statements listed above are specified in more detail.

Comments, additions, and corrections are useful