Unveiling the Cost of an Ophthalmology Clinical Trial
My father once told me that when it comes to any project, “You can have it fast, you can have it good, you can have it cheap: pick two.” For most of my life, and with most things, my dad was right.
When it comes to clinical research trials, however, I have yet to meet a sponsor willing to compromise on time, quality, or cost and with stakes so high, I don’t think they should. With careful planning and consideration, including the choice of a strategic and collaborative CRO, ophthalmic sponsors can run successful and safe clinical trials on time and within a reasonable budget.
How much does it cost to run a clinical trial?
The answer is, “it depends.” In a study conducted by a group of scientists from the nonprofit Institute for Safe Medication Practices (ISMP), the median cost of a set of 225 clinical trials from 2015-2017 was $19 million, with the estimated median cost for a pivotal clinical trial at $48 million . When looking at ophthalmology specifically, the study found the median cost to be about $36 million. But those are all estimates based on the median study in a field of clinical trials. How do you know where your study will end up falling on this continuum?
Conducting an ophthalmology clinical trial involves various elements and components, each contributing thousands to millions of dollars to the overall cost. Protocol development, site selection, patient recruitment, data management, regulatory compliance, and statistical support are just a few key factors that require financial consideration.
Although some study parameters are fixed, understanding each cost driver will help you plan and budget effectively, ensuring the successful execution of your trial. Let’s consider an average ophthalmology study and break down a few of the top considerations when it comes to getting the most from your study budget.
1. Regulatory Strategy and Operations
One of the best methods for establishing a baseline scope and budget for your study is to engage regulatory experts from an early stage to develop a strategic pathway to your target market. In addition to preparing the required documents, the right regulatory partner can prepare you for and facilitate meetings with regulators and help navigate key challenges on your regulatory pathway.
To begin with, be prepared to share the scientific and commercial objectives for your ophthalmology study with your CRO partner along with the open questions and variables you are willing to explore. Many therapeutic area specific CROs have deep domain knowledge, years of internal expertise, and external strategic partners to bring to your study’s design and execution strategy. Sharing the “why” will help guide your CRO toward a smooth, effective, and efficient pathway.
2. Ophthalmology Study Design and Protocol Development
The protocol is the foundation of your clinical trial. The study design, inclusion and exclusion criteria and parameters for execution including the visit schedule and schedule of procedures are the sources from which the cost will flow.
In the study mentioned above, the largest single influence on estimated cost was the number of patients needed to establish the treatment effects and the second-ranked influence on the estimated cost of pivotal trials was the number of clinic visits required for screening, baseline randomization, treatment and benefit assessment.
Further, in a recent global site survey by the Tufts Center for the Study of Drug Development, protocol execution burden was among the top five factors impacting their decision to participate in a clinical trial with between 67%-72% of sites rating it a major influence in their decision.
Developing a research design and protocol that will pass muster with regulators while appealing to ophthalmology sites, investigators, and patients is essential to running a successful clinical trial. Investing in an experienced CRO who can guide you and foster collaboration among investigators, medical experts, statisticians, and regulatory professionals will ensure you get your protocol right the first time and avoid costly and time-consuming protocol amendments.
3. Ophthalmology Site Selection, Feasibility Assessment, and Management
The right mix of investigators and research sites is a key piece of any successful clinical trial. A good CRO partner will understand the landscape of ophthalmology sites and be able to leverage a dedicated site relations team to carefully select and engage a custom network of sites.
Almost all CROs use syndicated data sets of historic data for site selection and enrollment forecasting. However, ophthalmology-dedicated CROs will have first-hand knowledge of how sites are set up and their interests, capabilities, equipment, performance records and staffing levels to help ensure the selected sites will deliver on time and within budget.
Site relations is about more than just selection though. Your CRO’s knowledge and experience will also come into play when it comes to budgeting, contracting, training, and initiation through monitoring, management, and close out. In the global site survey by Tufts, 70% of respondents said that conducting a clinical trial has become considerably more difficult over the last five years.
Having a relationship focused CRO with a dedicated site relations team can greatly impact your results. This experience will carry through the study and allow you to benefit from strong site relationships should course correction be required.
4. Ophthalmology Patient Recruitment and Retention
Because ophthalmology is a niche area with many rare diseases, patient recruitment often requires extensive time and adds to the complexity of the study. Recruiting a sufficient number of eligible patients is vital to the success of a clinical trial. In the Tufts global site survey, availability of eligible patients was one of the top two considerations for sites’ decision to participate in the study at all.
Patient recruitment and retention are among the most complex and potentially expensive components of a study, so you’ll want to ensure that your product development partner has a solid strategy to ensure success.
Costs associated with patient recruitment include advertising, screening, pre-screening, and enrollment. Retention efforts, such as patient education, reimbursement for travel expenses, and compensations for participation, also contribute to the overall cost.
You will see costs for recruitment and retention appear for central efforts – for the study overall – as well as additional ad hoc efforts that might be required at the site level. Pay attention to make sure your CRO is clear about the standard costs that come with planned pre-screening, screening and enrollment efforts versus those that might be required should things not go as planned.
Furthermore, retention efforts, such as patient support and education, reimbursement for travel expenses, and compensations for participation should be considered as separate costs.
Because ophthalmology is a complex therapeutic space with many common and rare diseases, a product development partner with domain knowledge and experience can save both time and money when it comes to setting and navigating your optimal patient recruitment and retention journey.
5. Data Management, Biostatistics and Statistical Programming
Managing and analyzing the data collected during a clinical trial is a critical component that impacts the timeline, quality, and cost of your clinical trial. From the costs of the various software packages, such as EDC to statistical analysis packages, to the services required to support system setup, database programming, data cleaning, and data analysis, safeguarding data integrity and driving proof of your results can be expensive.
That said, nothing can be as frustrating as spending millions of dollars on a study only to have to deal with costly and time-consuming mistakes with your data. Early and frequent biostatistics engagement can help identify gaps in your development strategy and in individual protocols. This often helps avoid errors and sidestep costly delays or inaccuracies that may cause regulators to challenge marketing applications.
Although this seems like an area where ophthalmology experience might not be as vital as other areas of the study, having a cohesive team of data managers, biostatisticians, and statistical programmers who can participate in the overall regulatory strategy is a worthwhile asset.
6. Ophthalmology Study Management and Clinical Monitoring
To optimize the execution of your study, choose a CRO that has ophthalmic research experience across the team, including at the study management and clinical monitoring levels. Certain CROs will have staff who have done trials in anterior and posterior segments, device trials, cell and gene therapy and rare diseases.
Also look for a CRO with internal education programs and Standard Operating Procedures (SOPs) that ensure that all staff receive not only GCP training but ophthalmology training as well. Your study management and clinical monitoring staff will be acting as an extension of you with sites and investigators and you will want to make sure to map the right path to reach study endpoints while growing your relationships with important stakeholders.
From site initiation to close out and each monitoring visit in between, your study management and clinical monitoring team serves to ensure data integrity, supply and equipment logistics, site communication and documentation, risk management, and several other valuable services.
According to the Tufts study, providing CRAs that are better qualified was the second and third most important factor ensuring sites’ long-term operating viability.
Selecting a CRO with the right staff will save time and money while assuring optimal results and strong ongoing relationships with your ophthalmology sites. Ask your study team to engage regularly to assess and manage risks to your timeline and budget.
7. Ophthalmology Medical Monitoring and Safety Oversight
The best CROs provide access to ophthalmology medical experts to provide research coaching from protocol development through the final CSR, leveraging expertise and ensuring patient safety throughout the study.
Having subject matter expertise in ophthalmology will help medical monitors do their jobs most efficiently as they will be able to educate investigators on protocol, patient eligibility, and safety reporting and will have valuable awareness of AE issues in other, ongoing trials with similar products. Understanding the ophthalmology space will also facilitate review and management of possible AEs, SUSARs, dose escalation rules and dose-limiting toxicities, coding, safety and clinical study reports.
Some CROs will include pharmacovigilance services as part of a larger clinical or medical safety offering. Whether these services are offered from internal or external resources, make sure the offering is integrated and seamless so you are inspection ready.
8. Additional Costs to Consider and Control
In addition to the priority items discussed above, it’s important to consider and control a few other budget items that might impact your study’s quality, cost, and timeline.
- Ancillary services encompass laboratory tests, diagnostic procedures, imaging, and other services required during the trial. Costs arise from the outsourcing or utilization of these services and are optimally controlled by working with a CRO that has longstanding relationships with its vendors and partners.
- Overhead and administrative costs are often budgeted to cover expenses related to CRO project management, insurance, quality control, legal services, communication, and documentation. Shipping and logistics costs can also be included. In general, these should amount to no more than 25% of the budget. Encourage your CRO to be transparent about these costs and the guidelines you can put in place to keep them to a minimum.
- Drug product – if the clinical trial involves testing a new drug or medical device, costs will arise to manufacture or procure the investigational product. This can include manufacturing, packaging, labeling, and distribution costs and expenses for drug accountability, storage, and disposal Biologics add an additional level of complexity, which again drives costs. An experienced CRO can help you choose and manage the right vendor to provide efficient support in this area.
In closing, your CRO partner can help you go a long way in affecting your final study cost. It can be tempting to choose the lowest bid when faced with cost constraints but, to use another phrase from my dad, “You seldom improve quality by cutting costs, but you can often cut costs by improving quality.”
The right partner will help you get things done right the first time and help you save time and money in the long run. Experienced CROs accurately forecast costs and expenses from the outset and provide transparent and regular budget reviews and proactive cost-control strategies to ensure the efficient utilization of your money and resources.
Before you go….
Want to learn how to maximize the likelihood of success of your ophthalmic study? Check out our quick guide, stacked with advice from experts in ophthalmology and clinical operations.
 Institute for Safe Medication Practices https://bmjopen.bmj.com/content/10/6/e038863.full#F1
 Tufts Center for the Study of Drug Development https://www.clinicalleader.com/doc/clinical-sites-are-optimistic-despite-growing-challenges-0001
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