Analgesia

  • Premier Insight 236: Fibromyalgia: Gaining Knowledge of a Little-Understood Disease Joining in the elusive pursuit of fibromyalgia pain relief, a small pharmaceutical company engaged Premier Research for a Phase III trial following our completion of the product’s Phase II research. The compound under study: a muscle relaxer on the market for decades, repurposed for the possible treatment of fibromyalgia. The sponsor chose Premier Research largely based on our experience – in analgesia, where we lead the industry with more than 870 trials, and specifically in fibromyalgia, where 15 studies over the past decade put us ahead of all other contract researchers. While experience always counts, the drug company sought our expertise because of the very specific nature of this poorly understood indication.
  • Premier Insight 237: That Was Easy This case is a story with no drama. No sudden crisis. No last-minute rescues. It’s about a clinical trial that ran perfectly from start to finish. And that in itself is a story worth telling. The sponsor needed to test a new nonsteroidal anti-inflammatory drug (NSAID). They had worked with Premier Research before, and knew our track record in analgesia studies: More than 870 pain trials treating over 110,000 patients, including trials for all of the NSAID analgesics on the market today.
  • Dermatology

  • Premier Insight 242: How Ramping Up Communications Helped Overcome a Next-to- Impossible Recruitment Challenge We knew going in that it could be the perfect recruiting nightmare. We were looking for children ages 2 to 12 for a dermatology study that involved long visits and extensive blood draws. We had extremely complex inclusion/exclusion criteria. And most parents wanted nothing to do with it.
  • Premier Insight 270: A Rosacea Trial Failed Many Enrollment Hurdles. Two large, parallel trials with 107 sites spread across the United States. Heavy competition for patients because of multiple competing treatments already on the market. And an advertising strategy that needed to attract more than 1,400 subjects fast enough to meet the sponsor’s aggressive schedule. Those were just some of the challenges we faced when studying a topical drug to treat rosacea. And as with most complex problems, the strategy required a broad set of solutions and experience-based insights.
  • Premier Insight 240: Getting Every Detail Right – With No Time to Think About How Our customer was racing to beat a competitor to registration. One critical part of the timeline: Last patient out to database lock in two weeks. In sister studies that involved 800 patients at 90 sites. And several primary investigators who were heading off on vacation just when we needed their sign-offs on the data. We also had to coordinate the efforts of a central laboratory, PK data people, a data management company, investigators and site staff. That left us with ultimate responsibility but no direct control over the other participants.
  • Hematology & Oncology

  • Premier Insight 271: Enrolling Sickle Cell Patients Is Tough – So We Got Busy Kick off a study of a drug to treat sickle cell disease and you’re facing a tough climb. Experimental drugs for sickle cell are few and far between, so there is little in the way of clinical trial infrastructure. The patient population is demographically challenging, too, made up largely of socioeconomically disadvantaged people who find it difficult to comply with the rigors of clinical drug trials.
  • Premier Insight 265: Complete Response: Lymphoma Trial A Surprising Success For Five Patients A trans-Atlantic study to evaluate an antibody for treatment of B-cell non-Hodgkins lymphoma is still almost three years from completing patient follow-up, but already it has succeeded beyond expectations in the form of five patients declared disease-free a year and counting. The Phase II trial involved a CD19 antibody that was tested at 26 sites — 22 in Europe and four in the United States. Ninety-two patients enrolled in the study, which set out to evaluate the compound’s efficacy in treating four NHL subtypes: FL (follicular lymphoma), DLBCL (diffuse large B-cell lymphoma), MCL (mantle cell lymphoma), plus a fourth, a type of indolent NHL.
  • Premier Insight 258: Glioblastoma Drug Study Yields ‘Unexpectedly Huge’ Advance A three-nation study of a treatment for recurring glioblastoma showed the therapy more than five times as effective as the typical protocol, an unexpectedly large advance in fighting these aggressive brain tumors. But it was a long road, and the drug’s efficacy was a big factor in extending the trial to five years — nearly twice its expected duration. Yes, recruiting was time-consuming — it took 21 months to find the 84 patients who were ultimately randomized and treated in this Phase II evaluation of an antibody-like human fusion protein. And it took extra effort to ensure that devices used across more than two dozen study sites were accurately calibrated.
  • Premier Insight 260: The Russian Connection: Recruiting Hard-to-Find Patients from a Sepcific HLA Subgroup At first glance, it seemed no more complex than any other global, randomized Phase III study. The devil turned up in the details. I’s always been true: every trial is a learning experience. Every one is different, and every one presents new challenges. And we confirmed that Premier Research can field the kind of teams it takes to win out over almost any challenge, from experienced yet never doctrinaire Project Managers to CRAs expert at motivating as well as monitoring sites. Above all, we learned how important it is to be the kind of people sponsors want to work with, and come back to again and again.
  • Premier Insight 256: A Talent for Resolving Unusually Complex Issues A customer came to us with a seven-year project to develop a novel human antibody from scratch, to treat multiple myeloma. There were no benchmarks, no relevant data on similar compounds because there were no similar compounds. The first challenge: engage key opinion leaders.
  • Premier Insight 259: In Clinical Trials, Success Isn’t Always Where You Expect It When does a clinical trial that falls short of its goal still constitute success? When the experience reveals invaluable lessons in how to avoid a repeat performance — and identifies a highly productive location for siting future studies. A large, multinational study of a drug to control inflammation in patients with recurring high-grade malignant glioma was in trouble. It had been underway for a year, and its data collection was compromised from the start when a requirement that all MRIs be read at a single facility proved unworkable.
  • Medical Device and Diagnostics

  • Premier Insight 252: A Novel Treatment That Worked So Well It Almost Derailed the Study We were testing an autologous chondrocyte transplantation system to repair damaged knee cartilage. The problem was that patients felt great after treatment, and saw no need to make follow-up visits. But the FDA required us to track results for seven years. The challenge: To keep patients involved for seven long years, especially active young patients who moved away from their original locations.
  • Premier Insight 253: Is it Safe or Not? Ask a Panel of Independent Experts When it comes to study data, not everything can be easily measured and reflected by numbers. In a recent trial of a new occluder device for transcatheter closure of secundum atrial septal defects, the primary efficacy endpoint of defect closure was easy to measure in millimeters of possible residual shunt. However, assessment of the safety of the device in terms of postoperative complications and device- or procedure-related adverse events was more complex.
  • Neuroscience

  • Premier Insight 254: Dealing with the Unexpected: Surprisingly Fast Enrollment Normally we’re ready for anything at Premier Research. We anticipate every contingency. This time the patients caught us off guard, by rushing to enroll almost as soon as word of the study got out. That’s because we were dealing with a significant unmet need: Most patients with Binge Eating Disorder (BED) seek help from psychiatrists, nutritionists or obesity specialists. But there was no approved, effective pharmacologic treatment. And patients desperately wanted one.
  • Premier Insight 255: Merging Science and Nuance to Treat Post-Traumatic Stress Disorder Patient enrollment is a key challenge in most clinical trials, and studies of drugs to treat post-traumatic stress disorder require an especially nuanced approach to recruiting. The target population of military service members is inherently circumspect, and the conditions that afflict these men and women add greatly to the challenge.
  • Other

  • Premier Insight 250: A Study That Wasn’t Working Until Premier Research Stepped In A customer called on us to rescue a study that just wasn’t going well. (It happens a lot.) The challenge was to deliver data the company needed to make a go-/no-go decision at a major internal meeting that couldn’t be rescheduled. The problems stemmed, in part, from a failure to communicate between a large, somewhat impersonal CRO and a small sponsor that was used to close personal relationships and more or less constant interaction with the team managing its studies.
  • Premier Insight 251: It Helps to Know What’s Going to Happen Next We were conducting a Phase IIb trial of a new treatment for nonalcoholic steatohepatitis, or NASH. And we knew three things: We knew that hepatologists would jump at the chance to enroll their patients, because the only existing treatment for NASH is the well-known lose weight – eat healthy – exercise more regime. This was the first simple, oral, once-daily treatment ever developed for the disease.
  • Premier Insight 235: It’s About How the Social System Works, Not Just the Science The assignment sounded simple enough: Recruit 24 patients for a Phase I proof-of-concept study of inflammatory bowel disease. The first CRO that received the assignment showed how difficult it really was: They were able to recruit only nine patients in a year and a half. At that point, the sponsor asked Premier Research to step in. We recruited and randomized 15 patients in six weeks.
  • Premier Insight 269: Recruitment in Reverse: Motivating Patients to Find the Study For a fertility study, we needed to recruit young women who had had at least three unexplained miscarriages and who were currently trying to conceive. The field of prospective recruits was huge: all women 18 to 37 years old living in the United Kingdom. The challenge: cost-effectively reach, screen, and randomize 150 patients. The solution: motivate them to reach us by replacing patient recruitment with a wide-reaching study marketing campaign.
  • Premier Insight 243: The Question Was a Real Puzzle – and the Answer Changed Everything Things were complicated enough to start. The customer, conducting a Phase II/III dose-finding/pivotal study for an adult growth hormone drug, changed its protocol midstream in response to regulatory feedback and was rethinking other aspects of the trial, adding to its complexity. For weeks, the sponsor’s requirements changed almost daily, severely testing an incumbent lab vendor that was already struggling to provide real-time data to support 21 titration paths. That’s when Premier Research stepped in and made a critical discovery that changed everything.
  • Pediatrics

  • Premier Insight 242: How Ramping Up Communications Helped Overcome a Next-to- Impossible Recruitment Challenge We knew going in that it could be the perfect recruiting nightmare. We were looking for children ages 2 to 12 for a dermatology study that involved long visits and extensive blood draws. We had extremely complex inclusion/exclusion criteria. And most parents wanted nothing to do with it.
  • Premier Insight 263: The Case of the Elusive Protocol There wasn’t any magic to our success. It was a matter of patiently adapting to changes, communicating clearly and frequently with all concerned, and maintaining our focus throughout. It also helped that we had regulatory specialists in each country to handle submissions on their home ground. As a result, we were able to balance the need for unusual flexibility with steadfast adherence to high ethical and scientific standards. It all goes to show: Good problem solvers are good at solving problems that are very different from the norm.
  • Premier Insight 238: Back On Track: Data Rescue On Pediatric Rare Disease Trial Imagine rebuilding the engine in an ambulance carrying a critically ill patient to the hospital. Got it? Then you have a pretty good idea what it took to put this pediatric rare disease trial back on track. A pharma company developing a drug to treat urea cycle disorder struggled with a CRO that was not effectively managing the study data. As trouble mounted, the CRO quit, stranding the project at a critical point in the development cycle.
  • Rare Disease

  • Premier Insight 271: Enrolling Sickle Cell Patients Is Tough – So We Got Busy Kick off a study of a drug to treat sickle cell disease and you’re facing a tough climb. Experimental drugs for sickle cell are few and far between, so there is little in the way of clinical trial infrastructure. The patient population is demographically challenging, too, made up largely of socioeconomically disadvantaged people who find it difficult to comply with the rigors of clinical drug trials.
  • Premier Insight 265: Complete Response: Lymphoma Trial A Surprising Success For Five Patients A trans-Atlantic study to evaluate an antibody for treatment of B-cell non-Hodgkins lymphoma is still almost three years from completing patient follow-up, but already it has succeeded beyond expectations in the form of five patients declared disease-free a year and counting. The Phase II trial involved a CD19 antibody that was tested at 26 sites — 22 in Europe and four in the United States. Ninety-two patients enrolled in the study, which set out to evaluate the compound’s efficacy in treating four NHL subtypes: FL (follicular lymphoma), DLBCL (diffuse large B-cell lymphoma), MCL (mantle cell lymphoma), plus a fourth, a type of indolent NHL.
  • Premier Insight 263: The Case of the Elusive Protocol There wasn’t any magic to our success. It was a matter of patiently adapting to changes, communicating clearly and frequently with all concerned, and maintaining our focus throughout. It also helped that we had regulatory specialists in each country to handle submissions on their home ground. As a result, we were able to balance the need for unusual flexibility with steadfast adherence to high ethical and scientific standards. It all goes to show: Good problem solvers are good at solving problems that are very different from the norm.
  • Premier Insight 238: Back On Track: Data Rescue On Pediatric Rare Disease Trial Imagine rebuilding the engine in an ambulance carrying a critically ill patient to the hospital. Got it? Then you have a pretty good idea what it took to put this pediatric rare disease trial back on track. A pharma company developing a drug to treat urea cycle disorder struggled with a CRO that was not effectively managing the study data. As trouble mounted, the CRO quit, stranding the project at a critical point in the development cycle.
  • Premier Insight 267: From KOLs to Patient Advocates, Using Every Tool to Fight a Rare Disease The indication: a basal cell carcinoma known as Gorlin syndrome, a disease that manifests itself in large numbers of benign tumors that grow on various parts of the body. The sponsor: a very small, virtual company consisting of a handful of contractors pursuing success in the form of a topical gel, which had only ever been tried as an oral drug in the past. This had demonstrated several undesirable side effects for patients as an oral medication, and the only other alternative available was to undergo numerous surgeries to physically remove the tumors.
  • Regulatory

  • Premier Insight 268: When Other CROs Said No, We Said Yes – to the Nearly Impossible The client asked the nearly impossible — a task so daunting that two other CROs had simply said “no, thanks.” Completing a new drug application typically takes a year to a year and a half, but the time available here was six months. And the short timeline was only the half of it. So we got busy. Really busy.
  • Services

  • Premier Insight 238: Back On Track: Data Rescue On Pediatric Rare Disease Trial Imagine rebuilding the engine in an ambulance carrying a critically ill patient to the hospital. Got it? Then you have a pretty good idea what it took to put this pediatric rare disease trial back on track. A pharma company developing a drug to treat urea cycle disorder struggled with a CRO that was not effectively managing the study data. As trouble mounted, the CRO quit, stranding the project at a critical point in the development cycle.
  • Premier Insight 244: Data Management: A Growing Challenge, Made Easier
  • Strategic Sourcing

  • Premier Insight 249: A High Unmet Need that No One Wanted to Talk About What do you do when patients are too embarrassed to talk about what’s wrong with them? It was a trial for fecal incontinence, and patients wanted help – they just didn’t want to admit it.
  • Premier Insight 245: Getting Creative: A Hybrid Answer When the Conventional Won’t Do When the going gets tough, the tough get ... creative. The search for the best resourcing solution sometimes defies a single answer. That’s when Premier Research combines outsourcing, insourcing, and geographic flexibility to devise a hybrid solution.
  • Premier Insight 247: Forty Percent Savings in Four Weeks: Yeah, We’re That Good A U.S. pharmaceutical company needed an immediate outsourcing solution to replace an underperforming vendor. Not only was time critical, cost was, too. These new data specialists would be located offshore to reduce the sponsor’s overhead. Oh, and did we mention it had to be done fast?
  • Premier Insight 246: Transfer and Re-Engage: A Powerful Path to Flexibility and Cost Savings Functional outsourcing can produce significant savings and greatly increase flexibility in assigning resources. But any big change in staffing requires care to avoid personnel disruptions, keep projects on track, and comply with laws governing use of contract employees.
  • Premier Insight 248: Outsourcing: You’re Now Free to Do What You Do Best Pharma, biotech, and medical device companies are increasingly turning to contract research organizations to outsource a wide range of activities through functional service provider (FSP) arrangements. FSPs increase agility and drive down costs while offering cafeteria-style flexibility — giving clients wide latitude in choosing which services to farm out and which to keep in house.