Small biotech companies are responsible for a significant proportion of newly approved medicines, especially for rare diseases. But with limited resources, budget constraints and a lean structure, they face many challenges before they can help patients. Here we look at some of the hurdles, and how small biotech can overcome them.
Emerging biotech companies may be small, but they are powerful in today’s drug development landscape, being behind the biggest share of new FDA-approved products. Their success lies in part in their focus: many small biotech companies opt for niche markets, such as treatments for rare diseases, in order to compete with the bigger companies.
In an interview with Contract Pharma, Christian Dowdeswell, Lonza’s vice president and head of commercial development for small molecules, comments: “The trend of innovation being driven by smaller companies is continuing, tied to the ongoing growth in specialty medicines.” He explained that small biotech saw a rise in funding in recent years, adding that “The tendency to develop therapies for niche indications with small patient populations (~67% of FDA approvals in 2020) is undoubtedly a factor here as well.”
Although this can put small and emerging biotech in a good position on the market, it also comes with a range of challenges related to the size of the companies and the interventions they develop. Here we look at some of the biggest pain points and share Siron Clinical’s insights to help address them.
Overcoming budget constraints
Financing is a major consideration for every clinical trial, regardless of the size of the company. But following the recent increase in available funding, biotech now faces soaring interest rates and tightening budgets in an unsettled global economy.
This has major implications for clinical trials: in one study, 22% of failed phase III trials failed because of a lack of funding.
But small biotech have flexibility and adaptability on their side. Many can operate in an agile way, enabling them to make changes to their approaches and save costs. They may also accelerate the timeline of the clinical trial, to ensure their funding lasts the duration. To make this work, they need efficiency, and this means working with the right partners.
Facing complex regulatory hurdles
The global clinical trial regulatory landscape is constantly evolving, and where big biotech has the in-house legal expertise to keep up with changes, smaller companies might find this more challenging. What’s more, regulatory requirements can be costly, especially if mistakes are made in the process, setting back progress. According to a paper in Contemporary Clinical Trials Communications, “particularly in the United States, the cost of complying with an increasing regulatory burden is also impactful, necessitating more staff, storage, and financial outlay.”
The key to ensuring compliance and navigating requirements efficiently and effectively is to partner with expert service providers who have international and local regulatory knowledge. As Dowdeswell notes in his interview with Contract Pharma, “smaller companies may look to outsource ‘soup to nuts,’” including seeking regulatory support. “Often these companies look to their service providers to guide them through some of the nuances of the clinical process in a consultative capacity,” he adds.
Filling resource and expertise gaps
While smaller companies tend to be leaner, this means they may have gaps in resources, knowledge and experience. According to a review paper in the New England Journal of Medicine (NEJM), small companies need to be innovative and drive for success to overcome these challenges. The authors note: “many small companies may choose to partner with larger companies to add resources and experience.”
Resource gaps could be anything from expertise in designing the trial protocol to publishing the results. Small biotech companies often have in-house technical expertise when it comes to the drug they are developing, but they may face particular pain points during regulatory submission, participant recruitment, monitoring and statistical analysis.
Outsourcing can help here. However, although many small companies outsource the expertise they lack in-house, this process itself is not without its challenges: selecting the right suppliers and partners and managing those relationships can be difficult too. Biotech companies may turn to expert vendor selection partners for supplier management support.
Recruiting the right participants
Recruitment and enrollment are among the biggest obstacles for most trials to overcome. According to one UK study, only 31% of clinical trials met their enrollment goals, putting them at risk of failure before they even get started. If the drug on trial is for a rare disease, which is the case for many small biotech companies, this obstacle can be even more significant, particularly due to the small pool of people suitable to participate in the trial. Although their small size, limited resources and potentially more challenging therapeutic area may make recruitment very challenging for small biotech, they can take steps to ensure they recruit a diverse group of participants that will give them generalizable results. In particular, they should focus on personal, clear and transparent communication throughout the process. If this is challenging from a resource perspective, companies may also turn to a partner service provider for support.
Partnering for success
Small biotech is a fundamental part of the broader clinical trials world, and their success is vital to ensure patients get the treatments they need. Even the leanest biotech can run a clinical trial efficiently and effectively, utilizing technology and decentralized approaches to limit costs, for example. And with the right partner, they can tap into decades of experience as well as global and local expertise across the whole clinical trial process.
Are you running a clinical trial with limited resources? You don’t have to do everything yourself – find out why, and contact Siron Clinical for support.