Expert Insights

Expert Insights

Bringing Advanced Therapies to Novel Territories

Bringing Advanced Therapies to Novel Territories
04 November 2020

Rare disease research is inherently challenging. Though it’s not uncommon to open one country with one site for one patient when working in low- to middle-income countries – defined by a gross national income (GNI) from below $1,035 to $4,045 per capita – there are additional factors that must be considered for cost-effectiveness and feasibility, such as:

  • Lack of validated outcome measures
  • Disease burden
  • Lack of global standard of care consensus
  • Cross-border enrollment patient travel

Additional challenges range from vague or nonexistent regulations or pathways for initiating a clinical trial; lack of financial support for novel therapies; often onerous regulatory requirements for approval of drugs or clinical trials; lack of reimbursement, defined timelines for approval, administrative infrastructure, and knowledge about diagnosis and drug treatment options; and minimal support for Phase I/first-in-human trials.

As daunting as these challenges seem, there are compelling reasons to tackle them to bring advanced therapies to novel territories. With a population of more than six billion, low- to middle-income countries have about 360-480 million patients with rare diseases, and these patients can experience the most significant benefit from treatment, given that there may not be other ways of standard of care. Furthermore, physicians and sites are typically very eager for current research and novel therapies.

Assessing a Country Holistically

Instead of determining viability based on the number of available patients, there are some areas of investigation that can help determine if a country is an option for your trial, such as:

  • Human subject protection: While there are levels of protection laws and regulations that exist globally, there are variances in those laws. It’s important to ask how laws and regulations differ from neighboring countries that might not be low- or middle-income countries, to have a risk assessment strategy in place, if necessary, to provide additional details or protection in terms of considerations like patient consent forms.
  • Biomedical regulations: If you’re developing a cell and gene therapy, it might be important to ask if it’s the first time it’s been done in that country. If it has been, you’ll know you’re not the first company to try a novel therapy in that country. However, If the response is no, ensure that you want to be the first to try that therapy in that country and to try that investigational product in that country with its accompanying challenges. Cross-border enrollment isn’t allowed everywhere; for instance, there are some upper-middle and high-income countries that do not allow cross-border enrollment, and we can assume the same for low- and middle-income countries as well.
  • Logistics and materials transfer storage: How far away is the site from the airport? Are they able to leverage the airport like we do in Europe and the U.S. in terms of shipping samples, etc.? Are there customs requirements such as import/export requirements?
  • Safety and stability: It’s important to understand historically how safe it is for locals of neighboring countries and for foreigners to visit that country for things like monitoring. What does the political landscape look like?
  • Necessary infrastructure: When performing this holistic review of countries, try to account for as many issues as you can. Are there any operational considerations you need to understand in terms of monitoring? Are there any manufacturing concerns? Does the site have the equipment in place to properly store equipment?

Weighing the risks and benefits of bringing advanced therapies to novel territories from a holistic view is critical to success. But it’s important to also evaluate if it makes sense for your company – your corporate identity – to target low- and middle-income countries, especially if you’re working in an indication that’s very saturated. Finally, by understanding the country holistically and understanding approaches to feasibility and site selection, you’ll have a much clearer picture to determine how that country can benefit your program and your pipeline.

For more information on rare and orphan disease clinical trials, please visit: https://www.synteract.com/Therapeutic-Expertise/Rare-Orphan-Diseases

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