NorthWest Biotherapeutics Inc (NWBO)

[SkyLimit2022]

LC,

Thanks for asking about the Specials Program again. Revenue might not be the most relevant consideration on this topic as the Specials data might have greater value in the long run…

The compassionate use cases generated some astonishing data, and those data may be included in the regulatory filings!

“Compassionate use data, also known as expanded access or named patient programs, can be included in regulatory submissions for drug approval. Compassionate use refers to the use of an investigational drug outside of a clinical trial to treat a patient with a serious or life-threatening illness for whom no other treatment options are available.

Regulatory agencies, such as the U.S. Food and Drug Administration (FDA), recognize that compassionate use programs can provide valuable information about a drug's safety and potential efficacy. However, the inclusion of compassionate use data in regulatory submissions is subject to certain conditions:

1. **Data Quality and Relevance:**
- The compassionate use data must meet the standards of quality, relevance, and reliability expected in clinical trials. Robust data collection and reporting practices are crucial.

2. **Consistency with Regulatory Requirements:**
- The data collected under compassionate use should align with the regulatory requirements for the type of approval sought (e.g., New Drug Application, Biologics License Application).

3. **Ethical Considerations:**
- Ethical considerations surrounding patient safety and informed consent are essential. The compassionate use program should adhere to ethical standards, and patients should be well-informed about the risks and benefits.

4. **Corroboration with Controlled Clinical Trial Data:**
- While compassionate use data can be informative, regulatory agencies typically prioritize data from well-designed, controlled clinical trials. Compassionate use data may complement such trial data but is not a substitute for it.

5. **Regulatory Agency Consultation:**
- It's advisable to consult with the relevant regulatory agency early in the drug development process to discuss the potential inclusion of compassionate use data in regulatory submissions. Agencies may provide guidance on how this data can be integrated appropriately.” —ChatGPT

https://www.prnewswire.com/news-releases/northwest-biotherapeutics-announces-that-a-marketing-authorization-application-has-been-submitted-to-the-uk-mhra-for-dcvax-l-for-glioblastoma-302021038.html

https://www.theguardian.com/science/2022/nov/17/im-just-carrying-on-vaccine-gives-brain-cancer-patient-years-of-extra-life

https://www.braintumourresearch.org/stories/in-hope/in-hope-stories/kat-charles

https://braintumourresearch.org/en-us/blogs/research-campaigning-news/astonishing-results-for-brain-tumour-vaccine-trial