Short Time to Market and Forward Planning Will Enable Cell Therapies to Deliver R&D Pipeline Value.
Hum Gene Ther
; 32(9-10): 433-445, 2021 May.
Article
em En
| MEDLINE
| ID: mdl-33023309
ABSTRACT
There is considerable industry excitement about the curative potential of cell and gene therapies, but significant challenges remain in designing cost-effective treatments that are accessible globally. We have taken a modeling-based approach to define the cost and value drivers for cell therapy assets during pharmaceutical drug development. We have created a model development program for a lentiviral modified ex vivo autologous T cell therapy for Oncology indications. Using internal and external benchmarks, we have estimated the total out-of-pocket cost of development for an Oncology cell therapy asset from target identification to filing of marketing application to be $500-600 million. Our model indicates that both clinical and Chemistry Manufacturing and Controls (CMC) cost of development for cell therapies are higher due to unique considerations of ex vivo autologous cell therapies. We have computed a threshold revenue-generating patient number for our model asset that enables selection of assets that can address high unmet medical need and generate pipeline value. Using statistical approaches, we identified that short time to market (<5 years) and reduced commercial cost of goods (<$65,000 per dose) will be essential in developing competitive assets and we propose solutions to reduce both. We emphasize that teams must proactively plan alternate development scenarios with clear articulation of path to value generation and greater patient access. We recommend using a modeling-based approach to enable data driven go/no-go decisions during multigenerational cell therapy development.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Contexto em Saúde:
1_ASSA2030
Base de dados:
MEDLINE
Assunto principal:
Custos de Cuidados de Saúde
/
Terapia Baseada em Transplante de Células e Tecidos
Tipo de estudo:
Prognostic_studies
Limite:
Humans
Idioma:
En
Revista:
Hum Gene Ther
Ano de publicação:
2021
Tipo de documento:
Article