Case Study Archives

Case Study: Achieving Success in the Largest-Ever Study of a Hemophilia Gene Therapy

At a Glance: Managing a 9-study, 21-year program with over 1,240 patients enrolled

PSI supported the sponsor of a program of studies that allowed hemophilia patients access to a gene therapy product for the first time, including the first gene therapy product to be administered in Brazil. By working closely with sites, building trust, and mitigating staff turnover, PSI was able to achieve FDA and EMA approval for nine studies over the course of a 20+ year program, including the largest-ever study of a hemophilia gene therapy.

Phase 3 study in severe hemophilia A

Sponsor Challenges

The first of its kind to offer gene therapy options to hemophilia patients, this extensive nine-study program required stellar teamwork to mitigate challenges from the outset, including high competition for patients, an intensive patient visit schedule, and complex dosing requirements and lab management. Maintaining consistency in trial oversight over the course of two decades to ensure the highest level of management and governance was crucial for the success of such a trial.

PSI Strategy

Strong site relationships: PSI has been building hemophilia site relationships for over 20 years. PSI knows the investigators and invests time and resources to build close site relationships, so sites want to work with us and prioritize our studies.
Building trust: From the outset, we worked closely with sites, where possible selecting sites that delivered for us on other studies. Additionally, the team implemented meticulous visit planning and site support, using both the Investigator Meeting and co-monitoring visits to build motivation for investigators and help the client gain valuable insights during the program.
Low staff turnover rate: PSI enjoys a low turnover rate – just 15% for the key staff on this portfolio. As we retained our staff, there was significant overlap across the portfolio teams, keeping valuable knowledge of the IMP and maintaining site relationships to deliver recruitment that exceeded the global average.

Program Successes and Outcomes

7 successful FDA Inspections
EMA and FDA approvals granted
Same Project Manager/Project Director for 5 years
Creation of client-specific data and budget tracking system
10+ long-term follow-up studies awarded to PSI based on performance

Learn how PSI can support your hemophilia study at the resources below or contact us today

About Us

For 25 years, we have built trusted relationships with biotech sponsors, with 90% of our customers being repeat and referral.

Hematology Experience

Multiple Myeloma, Lymphomas, Leukemias, and other oncohematological indications. More than 50% of operational staff has hematology and/or oncohematology experience.

ASH 2023

Meet our team of experts at this year’s conference where we will be exhibiting at our booth #3352.

Case Study: How VISIONAL™ is Helping to Accurately Predict Study Enrollment Timelines

Case Study, News article / By Ashley Stufano

At a Glance: AI and ML-powered insights for your next trial

A sponsor of a challenging breast cancer study needed to identify the optimal mix of countries and sites with similar experience and access to a hard-to-recruit subpopulation of breast cancer. Using internal and external data sources, PSI’s VISIONAL™ platform compared thousands of feasible country-site combinations and recommended the optimal enrollment scenario to meet the sponsor’s timelines and cost constraints.

Trial Enrollment Recommendations from VISIONAL™

Sponsor Challenges

The study targeted patients pretreated with recently approved drugs. Our key criteria for country and site selection included local standards of care, historical performance in the indication, and current competition. After the initial planning had begun, the sponsor also requested 20% of patients from Latin America.

How VISIONAL™ Helps Make Strategic Decisions

Working with internal and external data sources and KOLs, PSI feasibility team quickly pulled all available data on standards of care, site performance, and historical enrollment rates, carefully curated for similarity. The dataset included 40+ countries in North America, South America, Europe, Asia-Pacific, and Africa. Using this trove of data, VISIONAL™ compared hundreds of country/site combinations, taking into account study costs and probability of success.

We were able to easily adjust the constraints for regional patient quotas as well as additional requirements from the FDA and EMA after two rounds of protocol review.

Results

1. Optimal Enrollment Scenario

VISIONAL™ recommended the most optimal combination of sites, countries, and timelines based on the current landscape and historical benchmarks, while taking into account all constraints set by the sponsor and the FDA.

2. Cost vs. Speed
The system recommended the fastest and most cost-efficient scenario within the sponsor’s budget range and timelines. We were also able to quickly evaluate alternative options:

Faster scenarios with an acceptable risk level
Longer but less expensive scenarios
Scenarios that buffer for unexpected delays

3. Customizable and Adaptive Reports
The initial work done during the planning stage also provided the sponsor with peace of mind during the study launch with the ability to rapidly model new scenarios based on additional constraints/new requirements.

About Us

For 25 years, we have built trusted relationships with biotech sponsors, with 90% of our customers being repeat and referral.

Therapeutic Areas

Clinical trial sponsors working in oncology, hematology, IBD, infectious diseases, multiple sclerosis, and many rare diseases rely on PSI’s guidance and experience.

Global Reach

Clinical trial sponsors working in oncology, hematology, IBD, infectious diseases, multiple sclerosis, and many rare diseases rely on PSI’s guidance and experience.

up close image of a small clear vial, a CT scan of a brain and a stethoscope

Case Study: Phase 3 Radiopharmaceutical Clinical Trial in Oncology

Case Study, News article / By Ashley Stufano

At a Glance: FDA Approval in 4 Years

Radiopharmaceutical clinical trials face a host of logistical challenges from startup through the clinical phase. A sponsor of a pivotal Phase 3 prostate cancer study investigating a radiopharm product approached PSI for support based on our experience and established network of over 400 global radiopharmaceutical sites. By working closely with our sites and vendors, PSI met or beat all critical milestones, helping our client secure FDA approval for their radiopharmaceutical product in under four years.

Patient Screening and Enrollment Numbers

Sponsor Challenges

The major challenges in global nuclear medicine trials include the complexity of regulatory requirements in each country and the necessity for experienced, highly trained sites either within close proximity to the material’s manufacturing site or with the ability to provide at the site level. The investigational product in this study had a half-life of three days, necessitating diligent time management and site engagement to deliver the study on schedule.

PSI Strategy

To make sure eligible patients didn’t miss these three-day windows, PSI’s CRAs encouraged sites to maintain frequently updated pre-screening logs and regularly discuss the pool of potential patients with the investigators. We also worked closely with sites and the manufacturing facilities to time the inclusion of each patient appropriately, especially around local bank holidays when a manufacturing facility would not be available.

Results

1) Expedited Study Timelines
PSI met or beat all critical milestones during the study, including achieving FPI in the US in less than 3 months and completing NDA submission one month ahead of schedule.

2) 99% Enrolling Sites
Only one out of all initiated sites did not enroll a patient.

3) Successful Regulatory Approval
After passing three FDA inspections without major findings, PSI’s regulatory experts helped our client secure FDA approval in under four years.

“I would personally like to acknowledge and thank the entire PSI team and leadership for the dedication, perseverance and determined support that supported the early completion of the study. Your team have showcased all the capabilities and attributes of a best-in-class CRO for project delivery.”

--Global Head

External Relationship Management

About Us

For 25 years, we have built trusted relationships with biotech sponsors, with 90% of our customers being repeat and referral.

Therapeutic Areas

Clinical trial sponsors working in oncology, hematology, IBD, infectious diseases, multiple sclerosis, and many rare diseases rely on PSI’s guidance and experience.

Global Reach

Clinical trial sponsors working in oncology, hematology, IBD, infectious diseases, multiple sclerosis, and many rare diseases rely on PSI’s guidance and experience.

red blood cells under a microscope viewing with a purple dye contrast

Case Study: Optimizing Feasibility and Start-Up for a Phase III HemOnc Clinical Trial

Case Study, News article / By Ashley Stufano

At a Glance

No trial is ever easy. But with the continued effects of the global pandemic and the war in Ukraine, the sponsor had larger than average challenges. Nonetheless, PSI managed to demonstrate swift feasibility and start-up ahead of schedule, leveraging site relationships, and fostering team consistency and stability. In the end the study completed enrollment of nearly 900 patients on time at as many as 370 sites all around the world. Italy and Australia showed up as the top enrolling countries.

Patient Enrollment Numbers

Sponsor Challenges

Enrolling over 900 patients and in a clinical trial of this complexity would pose a challenge even in the most mundane of years. Add to that continual pandemic consequences and a global conflict, the stage is set for every possible delay and communication breakdown. The sponsor needed a CRO willing to tackle an aggressive recruitment plan and provide a solid partnership in order for the trial to succeed.

Start-Up in Sync

Staying on time means starting on time. Working closely with sites to understand how the pandemic affected their ability to meet site activation, PSI offered them several technical options that allowed for remote site selection visits. PSI managed to identify over 3000 sites and select about 500 sites 3 months ahead of schedule.

Additionally, feasibility projections were actually reflected in enrollment, not merely a best-case scenario or wishful thinking. This is due in large part to knowing the sites. Today, PSI would have also added machine-learning-powered tool, VISIONAL™, to generate the most optimal enrollment scenarios that could potentially further reduce the study time and cost.

Racalibrating the Geo-mix

Not every CRO can offer flexibility and adaptability, especially in a short amount of time. During enrollment, a change of strategy on the sponsor side called for more sites, and swiftly. In order to do this, countries with initially smaller projected sites and patients had to ramp up with little to no interruption. The only way this could be accomplished was with previously established solid global site relationships and coordination and stellar communication on every level of the study team.

Managing Motivation, Staffing, & Velocity

To motivate teams on a country level, PSI staff focused on maintaining a sense of competition between teams. This helped create a friendly sense of excitement as countries saw their recruitment numbers grow.

Continuity of the study team also played an enormous role in the study’s success. This not only helped keep the study on track when the client had changes to their own staff but provided a sense of consistency and reliability throughout the course of the trial at every level. Key roles project supervisor, project manager and other key staff stayed on for the whole of two years of study duration, something almost unheard of in this ever-changing industry. They are still with PSI running the trial as we write these lines.

The PSI Advantage

For the sixth year in a row, PSI CRO has been named a CRO Leadership Award winner in the categories of Expertise, Quality, and Reliability in the Overall (combined Big and Small pharma) respondent group. The CRO Leadership Awards are presented by Life Sciences Leader and Clinical Leader based on research conducted by ISR Reports. The awards recognize CROs that are voted by sponsors to meet or exceed expectations.

About Us

For 25 years, we have built trusted relationships with biotech sponsors, with 90% of our customers being repeat and referral.

Oncology Experience

Including multiple myeloma, lymphomas, leukemias, and other oncohematological indications.

ASH 2023

Meet our team of experts at this year’s conference where we will be exhibiting at our booth #3352.

Strategies for Working with NGS Testing on Site in China

Case Study, Video / By Ashley Stufano

Did you know that NGS testing in China can be expedited with the right site relationships?

As treatment options move towards enhanced personalization, NGS testing, or specific gene mutation testing, has become increasingly common as part of modern oncology and hematology clinical trials. When PSI Shanghai was contacted about a specific oncology trial requiring NGS testing, the team was able to contact 24 sites with relevant indication experience, and within 3 days, received interest from 15 of those sites to participate.

In order to accelerate patient enrollment, PSI engages a trusted network of highly qualified sites to ensure that each trial’s needs are met with specific experience. Learn more about NGS testing in China with Lisa Lu, Country Manager.

If you have a phase 2/3 clinical trial requiring NGS testing, consider clinical trial sites in China. Around the world, PSI CRO is working to ensure studies deliver on time and on budget. Discover more about our oncology and hematology experience here or contact us to speak with an expert today.

Transcript:

Did you know the NGS testing status in China?
Modern Oncology and hematology trials often require specific mutation testing, as we all move to a more personalized
medicine in time.
While this NGS technology is well established in China, it is not used in every study yet.
We still have capacity here. Let me share with you some recent cases.
We recently had a request for a specific oncology trial to be run in China. The protocol required sites being able to run the
NGS testing in screening phase. PSI’s Shanghai team contacted 24 sites with study experience in this indication.
Overall, the sites’ attitude was positive.We only had 3 days to check our sites. Among all of them, 15 sites expressed immediate interest and had the relevant
NGS experience and equipment.
The results show that NGS testing is not a show stopper for any modern trials which need this measured in China.
Looking forward to seeing you in Shanghai.

How to Boost Patient Enrollment in a Global IBD Trial

Case Study / By Paul Mirek

For one of the largest-ever global IBD trials, we knew our sponsor’s success depended on identifying the right sites and keeping them engaged.

Click to read How a Global IBD Program Boosted Enrollment & Strengthened Site Relationships Partnering WIth PSI CRO

Download the case study as a PDF.

Details:

Therapeutic Area: IBD

Geography: 40 countries

Sites: 400

Patients: 800 (planned)

Current Status:

Patient enrollment on schedule
PSI is on track to enroll 500 patients by year-end

How to Engage Investigators & Patients in the Highly Competitive IBD Landscape

After a large pharmaceutical company delivered enrollment 60 days ahead of schedule for a Phase 2 inflammatory bowel disease (IBD) study with PSI CRO, the sponsor faced a new challenge: one of the largest-ever IBD programs in the same indication. Competition for IBD patients is fierce, and we knew success depended on not only identifying the right sites but also keeping them engaged throughout the duration.

The sponsor engaged PSI to manage 400 sites and enroll 800 patients across 40 countries. With the length and size of the study, many sites went silent after the initial excitement. The onset of the COVID-19 pandemic introduced a whole new set of extraordinary challenges, which was especially noticeable during the summer months – already a typically slow period, but also when many countries implemented additional restrictions to slow the spread of new variants that made enrollment even more difficult.

To re-energize the study team and keep enrollment on track, the sponsor introduced a new incentive campaign with PSI modeled after the Summer Olympics to foster a sense of friendly competition.

3 Steps to Empower Your Sites

Create a compelling, inclusive campaign to motivate sites

PSI divided the study teams to stand for their respective countries and introduced the following scoring system:

Gold medal: Awarded for every subject randomization
Silver medal: Awarded for every subject screening
Bronze medal: Awarded for every subject rescreening

The project team developed creative materials and branding to engage the teams during the campaign and made sure that countries of all sizes stayed visible within the competition thanks to an averaged rating system.

Invest time into building close relationships with the site teams

To be truly patient-centric, a CRO needs to first be site-centric. Our mission is to make the site’s life in clinical trials as trouble-free as possible. It’s not easy, because clinical trial protocols are typically overcomplicated by all sorts of requirements that add work to the site staff already exhausted by routine medical practice. That’s why we are focused on building site support processes, working closely with the site staff to prevent and fix any signs of screening and enrollment delays.

PSI works carefully with each investigator to increase the percentage of patients entering the study, providing training and resources so the site teams understand the best moment to screen patients for the study and that screen failure patients could be reassessed. The project team provides extra support to sites and patients with personal protective equipment, implements additional procedures for direct shipment of the drug to patients’ homes, identifies a big network of local labs to minimize trips during the pandemic, and arranges comfortable and safe conditions for patients’ travel or travel reimbursement.

Build on a long-standing partnership

With the same leadership team in place since 2015 for the program, including the same Global PM and Co-Manager from the Phase 2 Study, the PSI teams draw on their experience from previous studies for this client during the competition. PSI Country Managers and Clinical Operations colleagues are also engaged to help the project teams come together to meet a common goal. It’s great to have a stable global team on such a complex project.

Sign up for more IBD insights from PSI CRO.

Strategies for Expediting Study Startup in Australia With Barrie Koh

Leave a Comment / Case Study, Video / By Paul Mirek

Did you know that Phase 2 and 3 study startup times in Australia can be expedited with the right site relationships? In order to accelerate the first patient in, PSI engages private sites and medical centers to ensure an average startup timeline of only five months. Learn more about study startup in Australia with Barrie Koh, Country Manager.

Around the world, PSI CRO is working to ensure studies deliver on time and on budget. Find the nearest office to you and start a conversation.

Transcript:

Hi, I’m Barrie. Did you know that Australia is quick to start up?

Everyone in the industry knows that it is easy to get an approval for an early-phase study in Australia. It is supported by streamlined regulatory processes, and you just need fewer documents for submissions. What most don’t realize is that startup in Phase 2 and Phase 3 isn’t that different.

The challenge is that most clients have a habit of going back to the same key opinion leaders or the huge institutions. they have lots of studies and they are heavily bureaucratic. To get to the first patient in quickly, PSI has been engaging the private sites, which are much more agile, only working with four to five huge private medical centers and a dozen more smaller sites.

With this particular approach, five months is feasible and it is consistent across all phases, all therapeutic areas and drugs classes. To cite you an example, in a recent microbiome study, we actually managed to achieve the first patient in in five and a half months at a private site down in Sunshine Coast. And this came in two weeks ahead of our planned first patient in.

Another example is a gene therapy study that we’re currently running. We were the first CRO to apply for a gene therapy license (in Australia). So we actually worked with each other to try to move this forward and this was done under four months itself and ever since then, we’ve completed four more gene therapy studies.

How to Apply for Phase 1 and Phase 3 Studies in China at the Same Time With Lisa Lu

Leave a Comment / Case Study, Video / By Paul Mirek

Did you know you may be able to run Phase 1 and Phase 3 studies in China in parallel, particularly for urgent medical needs?

Hear Lisa Lu, PSI CRO’s Country Manager in China, discuss how to determine if your study may qualify and how PSI’s local experts can help.

Around the world, PSI CRO is working to ensure studies deliver on time and on budget. Find the nearest office to you and start a conversation.

Transcript:

Hello, my name is Lisa Lu. I’m the country manager for PSI in China and I’m located in Shanghai. Let me share with you some relevant aspects for running clinical trials in China.

Did you know that for new drugs coming to China, it is needed to conduct a PK study in a Chinese population locally? One can apply for an IND for Phase 1 and Phase 3 studies at the same time.

Based on NMPA’s feedback, you can potentially run Phase 1 and Phase 3 studies in parallel. Urgent medical needs like hematology studies, and lethal diseases without any drugs available at the moment, have a higher chance in running Phase 1 and 3 studies at the same time.

Usually, a PK study needs 20 to 30 healthy volunteer subjects or patients, depending on the different indication and medicine.

How Do India Study Startup Timelines Compare? With Radhika Bobba

Leave a Comment / Case Study, Video / By Paul Mirek

How did we achieve site activation for a radiopharmaceutical study in India in less than 5 months? It turns out India study startup timelines are faster than many European countries.

Learn more in this video with Radhika Bobba, PSI CRO’s Regional Director, India and Far East Country Management.

Around the world, PSI CRO is working to ensure studies deliver on time and on budget. Find the nearest office to you and start a conversation.

Transcript:

India startup timelines are always discussed. “India is so slow, it’s so difficult to set up a study, it’s so difficult to start a study.” Let me give you a recent example.

We submitted a dossier for a radiopharmaceutical molecule in oncology on the 3rd of August, 2021. We received approval for this particular dossier on the 2nd of December, 2021 from a regulatory authority, the DCGI. It took us five months. The first site was initiated on the 14th of December, 2021, and the same site was activated on the 28th of December, 2021.

5 months, is it so long?

Yes, we are definitely not the fastest. We are definitely not the slowest. We can be compared to other countries in Europe. We can be compared to France, Italy, Germany, Switzerland. We are faster than Romania.

Debunking Latin American Study Startup Myths With Oscar Podesta

Leave a Comment / Case Study, Video / By Paul Mirek

Did you know that startup timelines for some countries in Latin America are comparable to those of many in Europe?

Hear from Oscar Podesta, PSI’s Head of Latin America, as he shares insights debunking some of the common myths about startup duration in the region.

Around the world, PSI CRO is working to ensure studies deliver on time and on budget. Find the nearest office to you and start a conversation.

Transcript:

Hi, my name is Oscar Podesta. I’ve been responsible for the Latin American region at PSI for almost 10 years now, and I wanted to briefly address one of the most frequent comments I receive about my countries here that is related to the startup timelines.

I think that these days we can call it a myth around Latin American countries being too slow to get started in clinical research trials, but I do remember those years where this was true—I mean when Latin American countries used to take just too long to get activated, but that’s not the case anymore, and it has not been the case for quite some time already.

These days, if you look only at statutory timelines, we can say that countries like Panama or Chile can be activated in less than six months, which is comparable in line with many countries in Europe. And even looking at bigger countries, like Argentina, for instance, our statutory timelines there are comparable with Germany, actually could be even shorter than the UK.

So, looking at overall just First Site Activated in countries in the region, our timelines are not longer than the rest of the world in general. But I think we have to look at that in more detail. And if we look at the data we have at PSI, our track record and metrics, and we actually look at the average time to activate all sites in a country—not just the first site activated, but all sites—that provides even more interesting information.

At the end of the day, we need all sites activated to make the enrollment for the study, so it is important to have the whole country active. And looking at those data at PSI, we find out that countries like Argentina, Chile, Panama, and Peru are actually shorter than several western European countries.

And let’s take another step into that and look at the bigger countries in the region, like Brazil or Mexico. Brazil right now is hitting more than 200 million people living there. So it’s huge, as everybody knows. And historically, everybody would say, “No, I wouldn’t go with the study to Brazil because it takes too long”—and it’s not the case. It’s not the case.

Right now there are a lot of indications, especially rare diseases, that are branded fast-track review by the authorities in Brazil, and a rare disease study can easily be activated in Brazil, maybe in six months, which puts the country in direct competition to pretty much any country in the world. So, the myth or the statement about Latin American countries being too slow, I don’t think it’s anymore not even fair to say.