What are the Changes in 2023 for Access to Advanced Therapy Medicinal Products (ATMPs) in France?

ATMPs are the most expensive innovative treatments, thus they require special regulation. Past regulatory measures in France, such as limiting the growth of drug expenditures, the creation of a fund to finance pharmaceutical innovation, the use of performance-based contracts and greater emphasis on medico-economic evaluations in pricing have been contributing to having both universal access to innovative therapies and fair remuneration for innovation. The importance of transparency, public participation in healthcare evaluation, and the challenges of setting drug prices based on their value are not negligible either. Although further negotiations are still necessary to ensure equitable access to medicines and control rising healthcare costs, France has made pioneering steps recently which would be worthy to follow for other states. The Social Security Financing Act for 2023 introduces measures to control face prices, offers a new funding model, and encourages manufacturers to submit reimbursement claims for the full scope of marketing authorizations.

The Reform of the Newborn Screening Policy: Spinal Muscular Atrophy.

One in every 10,000 children is born with SMA and half of them will not even live two years. It is a hereditary genetic disorder, where the muscles die. If it is discovered just after birth, newborns can get the newest medicines to maintain their health. Unlike some other common genetic diseases (e.g. Down-Syndrome), SMA can be screened prior to pregnancy to determine whether the parents are carriers. In Hungary, people have urged reform, due to the baby Zente case, whose story has reached millions. Australia and Germany have also discovered the need for screenings. However, the US has already introduced newborn screening for SMA, far ahead of European countries. National policies should adhere to the same path to contribute to appropriate family planning and to make the treatment available as soon as needed to provide a longer and better life for sick infants.