'Brain Waves' posts

Defining Tertiary Care for Neurological Diseases

Health care for patients with neurological diseases in the United States occurs across mul­tiple levels, ranging from primary to tertiary care and, less common­ly, quaternary care.

The World Health Organization has defined primary care as the point where first medical contact occurs, and where coordination and continu­ity of medical services is managed. Primary care focuses on a broad range of ser­vices, rather than the diagnosis and treatment of diseases of a specific organ system, and thus primary-care providers (PCPs) have varying levels of comfort in the treatment of neurologi­cal disorders.

Secondary care is provided by specialists, such as general neurologists, who focus on dis­eases affecting a specific organ system, usually upon referral by a PCP. Both primary care and secondary care are provided in an outpatient setting or a general hospital setting.

Tertiary neurological care is provid­ed by subspecialists who treat a subset of conditions that affect the nervous system. Examples include:

Unfundable: Clinical trials for rare diseases and orphan drugs

The study of acetazolamide for idiopathic intracranial hypertension described on page 9 of the Spring issue of BrainWaves raises an important question about a neglected aspect of clinical trials: How do investigators obtain funding to study the efficacy of a drug that has no commercial potential?

Only 300 of the approximately 7,000 known human diseases are of interest to the biopharmaceutical industry from the standpoint of prevalence or commercial potential, according to the Office of Rare Diseases Research (ORDR) at the National Institutes of Health (NIH). The Orphan Drug Act of 1983 sought to use patent protection and tax incentives to promote development of drugs for diseases with a small market. In this context, the word “orphan” refers to a condition or drug that has not been “adopted” by the pharmaceutical industry because of limited commercial potential and a rare disease is one that affects 200,000 or fewer Americans. In the twenty years following enactment of the ODA, 249 orphan drugs received marketing authoriza¬tion, compared to 10 in the preceding decade, with the most notable being Amgen’s erythropoietin.

Commercial potential alone does not determine value

Low prevalence does not equate to limited commercial potential, of course, because pricing must also be considered.

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