Over the past four decades, and especially in recent years, progress in the development of cell and gene therapies has reached unprecedented levels. In gene therapy alone, between 1989 and 2015 there were more than 2,330 clinical research programs targeting almost 50 different indications.As more cell and gene therapies reach commercial stage, industry insiders expect research in the sector to continue to expand in the coming years. For patients, clinicians, and health systems, new cell and gene therapies bring the promise of historic and transformative advances in the treatment of many serious diseases.
While ushering in a new era of hope in healthcare, cell and gene therapies also present many important considerations for stakeholders, including manufacturers, payers, regulators, patients, and clinicians, as well as entire health systems. For example, many new cell and gene therapies have highly complex manufacturing, distribution, and administration requirements that can drive up both cost and risk. In addition, while curing diseases remains the Holy Grail in drug development overall, current pricing and reimbursement models may not be easily adapted for some higher-cost drugs or for those involving a single dose or short course of a curative therapy.
Cécile Matthews quoted in Pink Sheet article following study on non-oncology orphan drug pricing in France
Following a recent study on non-oncology orphan drug pricing in France, CRA’s Cécile Matthews was interviewed by reporter Francesca Bruce. The interview was...