This report provides a strategic approach to achieving successful launches for rare-disease medicines. The authors emphasize the importance of addressing patient treatment needs and deploying launch investments strategically for maximum impact. The report also includes a case study of Sanofi Genzyme's activities with Gaucher's disease. The authors recommend that biopharmaceutical companies, particularly those with limited experience in rare-diseases, invest in preparation to achieve successful launches in the rare-disease market.
