For a reasoned therapeutic revolution in cell and gene therapy…

For a reasoned therapeutic revolution in cell and gene therapy…

by Pr Jacques MASSOL and Lionel RIOU FRANÇA

Cell and gene therapies are multiplying, and with them the hopes of patients, their families, and healthcare professionals. The aim of these therapies is to respond to major therapeutic needs, for seriously ill patients who are sometimes at a therapeutic dead end. These products are expected to bring about a genuine therapeutic revolution.

For the manufacturers who market them, as well as for patients and professionals, the aim is to make these therapies available as quickly as possible, which explains their often shortened, fragmented, and immature development.

The speed with which they are made available, through the various avenues of early access programs, sometimes even before marketing authorization has been obtained, is partly explained by the fact that evaluation authorities are all the more tolerant of uncertainties in terms of efficacy and safety when these drugs correspond to major therapeutic needs.

Indeed, the data available at the time of application is often limited to interim results from Phase III trials, or simple Phase II trials which, under certain conditions, may be sufficient for market access, as is the case in France with so-called early and compassionate access, outside the common law system.

However, in addition to the uncertainty surrounding their clinical performance, cell and gene therapies are characterized by their high, even very high, cost, which is by far higher than those of traditional drugs. Uncertainty and high prices explain the need for special negotiation/price-fixing methods, such as staggered payments or several types of risk-sharing agreements…

To ensure that these drugs reach the market as quickly and safely as possible, we need to anticipate the requirements for their evaluation (by the various registration and Health Technology Assessment authorities). These data may be independent of the countries targeted for reimbursement. This is the case for safety and often comparative effectiveness data (in Europe, this will be increasingly the case with the generalization of the European Joint Assessment), but it may also depend on the countries targeted for reimbursement (medico-economic studies, ability of national healthcare systems to receive and dispense the drugs in question equitably, measurement and description of target populations, burden of disease, pathology management, quantification of unmet needs, etc.).In order to ensure that we have the data we need, on time, to reach the market, we offer our services in terms of consultancy, preparation for possible managed entry agreements, and carrying out any useful clinical trials and RWE studies. Our approach is characterized by strategic and methodological support, as well as a continuum of data acquisition to ensure consistency, rapidity, and adaptation to the expectations of health authorities.