Featured Trial Design Editorial

  1. Rare Diseases Require Unique Approaches In Clinical Trial Design

    The vast majority of rare diseases have no approved treatments available, despite the fact that research related to these diseases has expanded significantly in the past 20 years. A key reason may be the many unique challenges researchers face when developing drugs to treat rare diseases, including difficulties designing and executing effective and appropriate clinical trials.

  2. AMO Pharma Addresses Rare Childhood Disease

    “Rare diseases are bad enough, but the ones that affect children are even more heartbreaking,” says Mike Snape, CEO of AMO Pharma, a virtual biotech focused on rare disease. “The medical need in these areas is greater than ever. They also present challenges for drug developers, especially with clinical trials.”

  3. Samus Therapeutics Expands Clinical Development Program

    Samus Therapeutics is a privately-held, biopharmaceutical company focused on developing novel therapeutics and diagnostics targeting the epichaperome, a foundational protein complex emergent from multiple disease states, including oncology and neurology. “We are the ‘anti-epichaperome company,’ says Jonathan Lewis, the company’s executive chairman and CEO. “Following various forms of cellular stress, chaperome units are rewired into the epichaperome network. Targeting and disrupting the epichaperome in cancer results in cell death  and in neurologic diseases, neuronal survival,  with no apparent effect on normal cells.”

  4. 3 Protocol Design Strategies To Enhance Patient Recruitment

    How many of the clinical trials that your company has conducted have met the initial recruitment timelines? How often have you seen recruitment issues increase costs or cause delays in your company’s operational objectives? Nearly 80 percent of clinical trials experience delays or even early termination due to recruitment issues.1 These delays not only negatively affect the sponsor company but also keep patients from getting potentially life-saving treatments in a timely manner (if at all).

  5. Google And Apple Compete For Clinical Trial Supremacy

    The Apple Watch, along with Apple’s ResearchKit, has already found a home in the clinical space. But it just got some competition. In April 2017, we saw the introduction of the Verily Study Watch. If you don’t know, Verily (formerly Google Life Sciences) is the research organization of Alphabet Inc. devoted to the study of life sciences. According to Verily, the Study Watch is the next step in the company’s targeted efforts to create new tools for unobtrusive biosensing.

  6. Clinical News Roundup: Why All The Love For Adaptive Trials?

    Clinical News Roundup for the week of April 10, 2017 with information on Scott Gottlieb and adaptive approaches, preparing for epidemic clinical trials, proposed tax law changes for clinical research, and using selfies to improve medicine adherence.  

  7. Finding The Right CRO For Rare Disease Trials

    CRO selection is a challenge for every company outsourcing clinical trials. When the trial you are about to launch is for a rare genetic disease and has 30 patients spread across multiple countries and continents, the challenges are multiplied. This was the situation for Cerenis Therapeutics, which recently launched a Phase 3 trial for patients having very low levels of high-density lipoprotein.

  8. Lilly Makes It Easier For Physicians To Become Investigators

    As the global head of clinical innovation for Eli Lilly and Company, Katherine Vandebelt knows there are many things pharma needs to do better. No drug can make it to market without first navigating the clinical trials process, which creates a burden on the physicians involved. Therefore, it should come as no surprise to anyone involved in the process that many physicians will oversee one trial and then opt to no longer take part in them.

  9. Are Outdated Pricing Models Hurting Clinical Research?

    Two issues that always seem to throw a wrench into sponsor/CROs relationships are changes to staffing and pricing. Protocol changes can disrupt trials for CROs and cause price increases for sponsors. Staffing changes can create new learning curves, delay trials, and cause sponsors to feel they were misled. Are penalties an easy way to fix both?

  10. PatientsLikeMe And Duke Bring Novel Approach To ALS Trial

    Can the peptide Lunasin help to reverse the debilitating effects of ALS? After one patient (and three experts) noted it could, PatientsLikeMe and the Duke ALS Clinic decided to get involved. Their novel trial will feature an unlimited number of patients, sharing of all patient data, and electronic patient reported outcomes. It also eliminated the use of a placebo.