RESUMO
Recent advances in biomedicine are opening the door to new approaches, and treatment and prevention are being transformed by novel medicines based on genetic engineering, innovative cell-based therapies and tissue-engineered products, and combinations of a medical device with embedded cell or tissue components. These advanced therapy medicinal products (ATMPs) hold one of the keys to making a reality of genuinely personalised medicine. There are an estimated 450 companies across the globe working on the development of gene therapies and more than 1,000 clinical trials underway worldwide, and some 20-30 new ATMPs filings are expected in Europe annually over the next 5 years. But challenges confront the sector, complicating the translation from research into patient access. Scientific, clinical development and regulatory issues are compounded by limited experience with clinical and commercial use, limited manufacturing know-how, high costs, and difficulties in accessing development funding and investment. Pricing and reimbursement and market access issues are an additional challenge, particularly in Europe, where unfamiliarity with the technology and uncertainty over the use of real-world evidence induce caution among clinicians, health technology assessment bodies and payers. There is a need for a review of the suitability of the regulatory and market access framework for these products, focused development of data, public/private partnerships, and fuller collaboration governments, doctors, insurers, patients, and pharmaceutical companies. This paper makes specific recommendations for all stakeholders, ranging from early dialogue on potential products, linking of clinical data and patient registries or standardisation of control frameworks, to a comprehensive approach to evidence generation, assessment, pricing, and payment for ATMPs.
RESUMO
We are, understandably, forever hearing about the high cost of bringing innovative new drugs and treatments to the healthcare market, especially medicines for smaller subgroups, and the fact that member state health systems often baulk at the prices. This article will argue that such a bypassing and blocking of innovative medicines and treatments is not only counterproductive when it comes to the health of Europe's patients, but actually fails to take into account the economic arguments. The article seeks to show that the long-term benefit to patients and the economy (health means wealth) will outweigh initial costs down the line. Couple this with a smarter use of information technologies and other resources and it will be possible to get much closer to building sustainable healthcare systems in a Europe struggling under the burden of an ageing population.