By Ed Miseta, Chief Editor, Clinical Leader
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Research firm Industry Standard Research recently asked 329 respondents from sponsor organizations to share their thoughts about a variety of current trends in the clinical development space. ISR analyzed responses by company size to understand how perspectives of respondents at large sponsors (those spending over $1B annually on R&D) differ from those at non-large organizations. Respondents were provided a list of trend-related statements and asked to rate their level of agreement with each.
I took this opportunity to speak with Rebecca McAvoy, VP of market research at ISR, to discuss the findings and gain a deeper understanding of what the results might mean for both large and small companies in the industry.
Ed Miseta: 87 percent of large sponsor companies believe their organization’s interest in mobile health applications will increase over the next two years. In non-large companies, that number drops to just 60 percent. Any insights on the difference in numbers? Are these technologies simply more affordable and accessible to the large organizations?
Rebecca McAvoy: Yes, that is my take on it. Large companies are more likely to have the internal resources along with the knowledge to create these applications and more likely to have the funds to outsource this work. There is a lot more room to “experiment” inside a large company.
Miseta: The FDA and sponsor companies are taking a much closer look at diverse patient populations. Again, it seems the large companies are more focused on this challenge than smaller ones. Any thoughts on that disparity?
McAvoy: I believe this is similar to the situation we discussed about mobile health. Large companies have the resources to reach into broader geographies and focus on diversity. And large companies are also more likely to be running larger trials where diversity becomes more of an issue.
Miseta: Patient recruitment and retention remains a challenge for most companies. Are more companies engaging patient recruitment firms to help with this challenge?
McAvoy: Going directly to sites is still the most common, and effective, route to find patients. We have asked this question about increasing the use of specialized patient recruitment companies for the past five years. We have seen very little change in terms of the percentage of respondents planning to increase their use of patient recruitment companies. Each year, approximately 70 percent to 75 percent of respondents say their organization will increase the use of patient recruitment companies in the next year or two years. So theoretically, the use of patient recruitment companies would be increasing but we don’t currently have additional data to support that.
Miseta: 58 percent of respondents reported virtual trials will be a major component in their clinical trials in the next two years. Will the COVID-19 pandemic cause those numbers to increase significantly, in both large and small sponsor companies?
McAvoy: Recent research respondents have shared that their companies have made big changes to implement virtual components to trials (telehealth, home health visits instead of site visits). Not only does this keep patients safe during the pandemic but it is easier and more comfortable for sick patients to not come into the site as frequently, pandemic-related issues aside. Many anticipate that these virtual components will continue to be used after the pandemic is under control. The data you reference was collected in Q4 2019 and I would imagine when we ask the same question again in a few months, we will see a sizable uptick.
Miseta: More than half of the large companies surveyed believe they will conduct a clinical trial in the next two years where most of the activities occur in a patient’s home. What will be the biggest challenge companies face in making this vision a reality?
McAvoy: There are many difficulties associated with this. Inertia regarding trying something new, uncertainty over whether regulators will approve new methods of data collection, data privacy, logistics (everything from supplying the study drug to making sure at home devices are charged to educating patients on how to use data capture devices or apps), patient compliance, patient safety, collecting patient samples, etc. Despite those challenges, my guess is these numbers will see an uptick during our next collection of this data as well.
Miseta: More than 50 percent of survey respondents believe clinical supplies and logistics is one of the most complicated parts of their clinical trials. With the technologies we have today, why is this still so much of a challenge for companies? And do you expect the rise in virtual trials will mean more medications being shipped direct to patients?
McAvoy: Clinical trial supplies and logistics have a lot of moving parts. Supply forecasting is tricky and is tied to patient enrollment which can be unpredictable. Companies must balance not producing too much of a drug so that it won’t be used before it expires with making sure that there is enough drug on hand to reach trial subjects when needed. Often there are temperature controls and storage to account for. And, of course, there are regulatory/local customs requirements in different countries around the globe. To complicate things even more is the fact that in many trials variable dosing based on patient weight or other markers means variable shipments. Finally, a lot of hands are involved in passing drugs back and forth. That creates a lot of opportunities for delays or errors in handling. New technologies have simplified some aspects of logistics, but many of these challenges will never be eliminated.
Miseta: 63 percent of respondents noted their organizations will increase their focus on rare diseases and orphan drugs. Is this further indication that the days of blockbuster drugs are over, and that the future of drug development lies in precision medicine?
McAvoy: Respondents have been sharing that they are seeing more and more activity in the rare disease space. Some common diseases and cancers are being sub-typed by biomarker status or other markers into rare disease subtypes. However, there are challenges with that as well. Prices for the rare disease treatments are astronomical and respondents have hypothesized they are unsustainable. Reimbursement is challenging. If the prices go down, smaller biotechs may not be able to recoup their development costs which could lead to more partnerships with or acquisitions by large companies. Conducting rare disease trials is very difficult, every patient is so critical, and it takes time. Some large pharma companies are willing to let smaller firms take on that initial risk until they get past the proof-of-concept stage.