2.1 Disease State Fundamentals

Understanding a New Disease Area

Serial entrepreneur, Mir Imran of InCube Labs, explains his approach to immersing himself in a new disease

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Serial entrepreneur, Mir Imran of InCube Labs, share some pointers for maximizing the effectiveness of disease research.

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Michael Ackermann, CEO of start-up Oculeve, describes how disease research was fundamental to the development of his company’s product.

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 Getting Started

Regardless of whether or not the innovator has any sort of medical background, an effective assessment of disease state fundamentals can be developed for any given disease state following the steps outlined below.

Assess Anatomy and Physiology

What to Cover

Describe the normal anatomy and physiology of the affected organ(s) and/or system(s).

Where to Look

  • Anatomy Lab – If at all possible, visit an anatomy lab to get a first-hand view of what human organs look like, as well as their relative size and location within the human body.
  • eMedicine – Clinical knowledge base with more than 10,000 physician authors and editors.
  • Harrison’s Online – Contains the complete contents of Harrison’s Principles of Internal Medicine.
  • Medical References – Guyton’s Textbook of Medical Physiology or another standard medical textbook will provide valuable, credible, and detailed information regarding physiology. References such as Netter’s Atlas of Human Anatomy or Gray’s Anatomy of the Human Body are important anatomy references.

Understand the Pathophysiology of the Disease

What to Cover

Address disease function, causal factors, and disease progression.

Where to Look

In addition to the previously listed references, use:

  • PubMed – A database of the U.S. National Library of Medicine that includes more than 16 million citations from MEDLINE and other life science journals back to the 1950s. PubMed general reviews will be particularly helpful in understanding pathophysiology (locate Reviews by selecting this type of article under the “Limits” tab from the main page of the site before beginning a search).
  • Medical References – Texts such as Robbins and Cotran’s Pathologic Basis for Disease (Elsevier, 2010) are useful resources for understanding pathology and pathophysiology.
  • Up-To-Date– A database of evidence-based clinical information.

Understand Clinical Presentation

What to Cover

Profile the patient state associated with a disease.

Where to Look

The New England Journal of Medicine (NEJM) and the Journal of the American Medical Association (JAMA) often have clinically relevant review articles that may provide an up-to-date summary of a disease state and its clinical presentation. Physician and/or patient interviews are another excellent source of information. More basic information can be found on patient advocacy group websites and healthcare company websites.

Assess Clinical Outcomes

What to Cover

Elaborate on the morbidity and mortality rates associated with the disease.

Where to Look

  • eMedicine
  • Harrison’s Online
  • PubMed – Clinical trial outcomes will be helpful (locate Randomized Controlled Trials (RCTs) by selecting this type of article under the “Limits” tab from the main page of the site before beginning the search).
  • Up-To-Date

Gather Epidemiology Data

What to Cover

Outline the incidence and prevalence of the disease, as well as dynamics in the area.

Where to Look

Evaluate the Economic Impact

What to Cover

Determine the overall cost of the disease on the system at large.

Where to Look

  • Medscape – Database of clinical information targeted at physicians.
  • MEPS Data – The Agency for Healthcare Research and Quality (AHRQ) provides data on the health expenditures of 18,000 U.S. households via the Medical Expenditures Panel Survey (MEPS). This data is publicly available for primary analysis.
  • PubMed (Reviews and/or other journal articles)

Assess and Summarize the Information

What to Cover

Capture the most important information gathered through the disease state research and compile it into an overview suitable for the target audience.

Where to Look

Refer back to the resources listed above if gaps are identified or questions arise when developing the summary.