Category Archives: Blog

Although current limitations in neuropsychological testing may discourage development of treatments for central nervous system (CNS) disorders, some researchers see these unmet needs as an opportunity for innovation. Take a look at three strategies that are improving the validity and viability of neuropsychological testing. Rater Training Success or failure of a CNS trial can come down toRead more

A variety of factors can make it difficult to conduct traditional full-scale clinical trials for new treatments of rare cancers. Consequently, because so little information is available, treatments for rare oncology patients are inadequate or nonexistent. Luckily for both patients and researchers, the FDA and the European Medicines Agency (EMA) have introduced regulations that expedite review and approval of certain investigative drugs. Expedited FDARead more

Central nervous system (CNS) disorders are a diverse group of conditions that include psychiatric, neurological and substance abuse disorders. Unfortunately for patients, treatment options for CNS disorders are often limited (or non-existent). To make matters worse, comparatively few CNS drugs are in the development pipeline. What aspects of CNS disorders contribute to lagging drug development? Read on toRead more

A rare disease, also referred to as an orphan disease, by definition affects a small percentage of the population. Most are genetic and appear early in life, with one in three children with a rare disease dying before their fifth birthday. While the definition of “rare” varies from country to country, the US defines a rare disease asRead more

A rare cancer is often defined as one with an incidence of less than 15 per 100,000. The patient populations of these conditions are so small that traditional clinical trial design strategies can be unfeasible. But, with such a low incidence, just why is rare oncology research so important? Why Rare Oncology Needs Innovation Keep in mindRead more

A target product profile (TPP) is a strategic process tool that streamlines medical device development through cross-functional strategic alignment. It’s a living document that defines the value proposition and key differentiators of an intended commercial product while assisting in product development strategy. A TPP may take a lot of time and resources to craft, but it’s a strategy that promises a majorRead more

When developing treatments for a relatively common illness or disease, patient recruitment is relatively straightforward.  Screening and data collection can generally be done in a centralized location in a highly controlled environment. Now, imagine the concentration of people with the disease is not high enough in your local area, or even your country for thatRead more

Visit the iTunes App Store or Google Play and you will find the smartphone app PoopMD+: A New Parent’s Guide to Baby Poop and Pediatric Liver Disease Study from Johns Hopkins. PoopMD+ is an imaging and clinical survey-based app developed to help screen for a rare but life-threatening liver disorder in babies and collect feedbackRead more

This week’s Tales from the Road is from Angi Robison, Executive Director, Strategic Development Department, Rare Disease & Pediatrics, who gives us a bit about what she took away from the World Congress Patient Advocacy Summit back in October. Greetings from the road! I just had an excellent two days at the World Congress Patient AdvocacyRead more

A target product profile is a document that presents a polished explanation and aggregation of all relevant information needed in validating product development. This common template can be used for all products across a company portfolio to guide and align regulatory, preclinical, clinical, marketing, and health economic outcomes and reimbursement strategies. The concept of the TPP was originally developedRead more

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