By Chih Wei Teng PhD

With more cell and gene therapies in clinical trials and higher expectations of regulatory approval, can the industry automatically assume commercial success or patients expect equitable access when cell and gene therapies become routine practice? This may not be the case, especially in smaller biotechnology firms that are struggling with complexity and lack of resources. If there’s a mentality of ‘if you build it, they will come’, business planning is an activity that you do just enough to convince the venture capital partners to invest in the company. Afterall, the biggest hurdle that biotechnology firms focus on overcoming is regulatory approval where the criterium are safety, efficacy, consistency and quality. While regulators would like to see more therapies introduced to the market, it is not the regulator’s responsibility to ensure your commercial success. Many of us rely on heuristics or checklists to ensure we do not focus on a few goals at the expense of others.

A couple of months ago, I found myself reading an article from McKinsey & Company titled ‘Eight Imperatives for Launching Cell and Gene Therapies’. [1] The article provided an informative yet concise pathway to guide and validate your commercialisation strategy. Advice included common perspectives on cornerstones of success, such as optimising supply chains, developing an international business model and achieving scalability. Other more complex imperatives include offering innovative payment structures and integrating real-world evidence.

*Figure 1: Eight imperatives from McKinsey & Company needed to launch cell and gene therapies. Alfano et al., 2022*

The McKinsey article resonated with me because Jimmy Yu, who has spent his internship with CCRM Australia, was conducting research to understand the challenges and efforts needed to support equitable access to cell and gene therapies by Australian First Nations, i.e. the Aboriginal communities. The McKinsey article cited a statistic that patients living within 60 miles (97 kilometres) of the treatment centre are twice as likely to receive therapy. [1] This is especially true considering that major treatment centres where most cell and gene therapies are currently being delivered are typically located within metropolitan areas. This could be due to a number of reasons, including available expertise, greater support from allied health and where state-of-the-art infrastructure is located. Cell and gene therapies may require more extended observation periods post-administration, translating to higher travel and accommodation expenses for patients from rural areas. Australian First Nations often live in remote regional communities many times further than 60 miles from the State capital cities. [2]

The issue around health equity is exacerbated further due to the lack of culturally appropriate healthcare services and the historical mistrust of the healthcare system, which has seen many First Nations people avoid seeking medical care until the end stages of their diseases when conditions become too severe to ignore.

*Figure 2: Disease group contribution to total, fatal and non-fatal burden among Indigenous Australians, 2018, Source from AIHW 2022*

As seen from Figure 2 above, the most significant causes of fatalities among First Nations people are 1) accidents and trauma, such as road accidents, falls, and burns, 2) cancer, 3) cardiovascular and 4) infant/congenital. Many of these medical conditions are prime targets for cell and gene therapies. Even in non-fatal areas of musculoskeletal, respiratory, neurological and hearing/vision, regenerative medicine could considerably improve their quality of life.

Through the years, articles have been published about developing business models to treat orphan and neglected diseases that traditional big pharmaceutical companies would often ignore because small market potential makes for difficult business justification. [3] Ironically, targeting orphan diseases is now a business tactic for fast-tracking market approvals by some of these big pharmaceutical companies. In the pursuit of equitable access, this imperative list offers a useful guide during brainstorming sessions to overcome barriers to access, such as remoteness, workforce shortages in remote regions, lack of infrastructure, and communication and culture barriers. Some of the challenges may not seem that unsurmountable. For example, the transportation of cold storage products to remote locations has seen solutions being deployed during the COVID-19 pandemic. Vehicle manufacturers such as Toyota [4] and logistic companies [5] have paved the way. These are good examples of go-to-market models, technological advancements or novel service offerings from business partners that enable biotechnology companies to either go direct or enter into a partnership to optimise the supply chain when we simultaneously address items 5 and 7 from Figure 1.

Your situation may differ from the research and social challenge we are undertaking, but I feel that the list by McKinsey & Co is helpful. Several fundamental concepts are captured in Figure 1 that businesses should remember while juggling many operational, strategic or even 'fire-fighting' activities when bringing a therapy to market. By now, we all agree that the process is the product. While you can reproduce something in the laboratory, it differs significantly during technology transfer and in multiple locations. These processes must be optimised for cost, safety and efficacy. While your business focuses on translating the value of your innovation from the bench to the bedside, you have to ensure that you get sufficient value back from the market to make your business sustainable. This might mean novel methods for measuring success over the long term and value extraction.

Models and checklists are good heuristics that simplify complex processes to make the commercialisation journey manageable. If you have other heuristics you found helpful in your journey, please to share them with me (chihwei.teng@ccrmaustralia.com.au) and I will post it on the CCRM Australia website.

My blog is just one of many covering this topic as part of Signal’s seventh annual blog carnival. Please click here: https://www.signalsblog.ca/blog-carnival-challenges-and-proposed-solutions-to-support-a-deluge-of-new-cgt-approvals to read other bloggers' thoughts.

References

1. Mckinsey: Simon Alfano, Alex Gorham, Alberto Loche, and Pablo Salazar 2022, Eight imperatives for launching cell and gene therapies, McKinsey & Co. website: https://www.mckinsey.com/industries/life-sciences/our-insights/eight-imperatives-for-launching-cell-and-gene-therapies#/

2. Anderson, K., et al., Accessibility of cancer treatment services for Indigenous Australians in the Northern Territory: perspectives of patients and care providers. BMC Health Serv Res, 2021. 21(1): p. 95-95.

3. Institute of Medicine. 2009. Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualised Therapies: Workshop Summary. Washington, DC: The National Academies Press. https://doi.org/10.17226/12219.

4. Zachariah 2021. The Toyota LandCruiser 70 Series has been certified by the World Health Organisation (WHO) to transport COVID-19 vaccines, thanks to a medical-grade refrigerator. (https://www.drive.com.au/news/toyota-landcruiser-70-series-approved-by-who-as-vaccine-transporter/)

5. Truckies praised for logistics behind COVID-19 vaccination roll-out (https://hvia.asn.au/truckies-praised-for-logistics-behind-covid-19-vaccination-roll-out/)