Delivering advanced therapies: the big pharma approach

Tarnowski, Joseph; Krishna, Delfi; Jespers, Laurent; Ketkar, Amol; Haddock, Rachel; Imrie, Jessica; Kili, S

doi:10.1038/gt.2017.65

Cited by 9 publications

(6 citation statements)

References 9 publications

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“…For example, basic research in Niemann-Pick disease type C, an enzyme deficiency leading to organ failure, led to treatments for Ebola [52], and basic research in cherubism, a disease causing bone loss of the jaw, may lead to treatments for arthritis [53]. Development of gene therapies for patients with rare genetic disorders allows the therapy to be studied in patients with a clearly defined condition, thereby informing research on more complex diseases while providing crucial therapy for patients with rare disorders [54]. Assessments of value must also take into account this larger societal benefit.…”

Section: Patient Perspectives On Proposed Methods For Determining Primentioning

confidence: 99%

A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies

White¹

2019

Gene Ther

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Gene-based therapies are changing the landscape of medicine for patients with rare diseases. These one-time, potentially curative treatments pose a challenge in the US healthcare model, where high prices and insurance coverage variation may prevent patients from receiving life-altering therapies. Questioning of high prices occurs when patients, payers, and policy makers hold divergent views on the value and uncertainty of therapies. The key for patients is that high prices need to be justified, and companies need to partner authentically with patients. Companies should not automatically assume that the combination of the "gene therapy" label and a small patient population justifies high prices. To speed up the development process and potentially reduce costs, patients want the industry to improve clinical trial efficiency by sharing data, including natural history studies and failed trial results. From the patient perspective, current value assessment frameworks disadvantage people with disabilities, may not accurately reflect patients' and societal views, and omit necessary factors such as impact on caregivers, lost productivity, and the future value of innovation. Value determination methods need to provide fair incentives and outcomes to industry, payers, regulators, and especially patients-the courageous pioneers who need equitable and sustainable access to life-changing gene-based therapies.

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Section: Patient Perspectives On Proposed Methods For Determining Primentioning

confidence: 99%

A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies

White¹

2019

Gene Ther

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“…El tipo de negocio de las empresas farmacéuticas está cambiando y con la pandemia del SARCS-COv2 se ha acelerado esa transformación. El auge de los medicamentos biológicos y personalizados, a través de terapia celular o génica (Tarnowski et al 2017), con los altos costos de su desarrollo, a la par con la dificultad de desarrollar nuevas moléculas, está llevando a las farmacéuticas a plantearse diversas formas de adaptación, entre ellas hacer un cuidadoso balance entre el mercadeo y los costos de investigación (Márquez y Marveya 2019) y en no pocos casos invertir más en marketing que en investigación y desarrollo (Ponce 2018).…”

Section: El Código Trust: Un Medio Para Impedir El Doble Estándar En ...unclassified

Las empresas en la investigación sobre las innovaciones en medicamentos

Gamboa-Bernal

2022

Rev. Col. Bioet.

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Propósito/Contexto. El papel de la industria farmacéutica se puede examinar desde múltiples ángulos. La presencia de estas empresas durante la pandemia ha sido obligada por la necesidad de contar, en el menor tiempo posible, con vacunas capaces de prevenir los contagios y medicamentos efectivos de tratar a los afectados, es decir, el desarrollo de productos innovadores. Metodología/Enfoque. Desde una perspectiva bioética se reflexiona sobre cuatro temas conexos con el rol de estas empresas en circunstancias análogas: las responsabilidades de los patrocinadores en los procesos de investigación; la contratación con las organizaciones de investigación por contrato (CRO); la relación con los investigadores y la interacción con los comités de ética en investigación (CEI) y, finalmente, sobre una manera de contener el doble estándar en investigación. Resultados/Hallazgos. Los patrocinadores tienen responsabilidades precisas que están orientadas por documentos y pautas internacionales. También hay una necesidad de tener una mayor claridad en el trabajo de las CRO y los investigadores pueden mejorar la interacción con los CEI; además, debe evitarse el dumping ético. Discusión/Conclusiones/Contribuciones. Se concluye que la industria farmacéutica tiene un papel de corresponsabilidad en los procesos de producción de medicamentos innovadores con los demás actores, con quienes ha de mantener un diálogo transparente y fluido, y debe orientarse no solo por las guías de buenas prácticas clínicas, sino principalmente por la Declaración de Helsinki. Un medio puede ayudar en la eliminación del doble estándar en investigación: el Código Trust, así las empresas farmacéuticas pueden contribuir a mejorar la cultura ética de la investigación.

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“…However, it was withdrawn from the EU market because its 1 USD million price caused extremely limited usage in major EU countries where reimbursement was lacking. This is indicative that the price of orphan ATMPs must be determined by finding the right balance between medical needs, clinical benefits, appropriate commercial returns, and healthcare affordability [54].…”

Section: Reexamine the Reimbursement Of Atmpsmentioning

confidence: 99%

Current state of developing advanced therapies for rare diseases in the European Union

Qiu

Wang

Dabbous

et al. 2020

Expert Opinion on Orphan Drugs

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Objective: Advanced Therapy Medicinal Products (ATMPs) present significant therapeutic advantages for inherited rare diseases. However, the development of orphan ATMPs is challenging due to their complexity and unpredictable biological activity. This study aims to comprehensively describe the current state of orphan ATMPs in Europe. Methods: Orphan drugs (ODs) granted by European Commission until March 2020 were investigated. The characteristic of diseases and ATMPs were extracted from the public summary reports of ODs from Committee for Orphan Medicinal Products. The methodology for the pivotal studies of ATMPs was extracted. Results: A total of 274 ATMPs were identified, covering 116 rare diseases, with metabolic, optical, and oncologic diseases being the most targeted. A total of 158 ATMPs were indicated for life-threatening diseases, 129 ATMPs targeted diseases currently lacking authorized or satisfactory treatment available. Twenty-eight ATMPs are being investigated in the phase II/IIII or phase III studies. The median patient size of pivotal studies was 127, 15 were open-label studies, 8 were single-arm trials, and 14 reported surrogate outcomes. Conclusion: There are rapid growths in developing ATMPs for life-threatening diseases with high unmet clinical needs. Optimizing the study methodology and exploring innovative design to facilitate the market access is paramount.

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Delivering advanced therapies: the big pharma approach

Cited by 9 publications

References 9 publications

A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies

A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies

Las empresas en la investigación sobre las innovaciones en medicamentos

Current state of developing advanced therapies for rare diseases in the European Union

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