What action should the nurse take first? It begins with a brief overview of CF and the science exploring the genetic basis of a devastating disease. When and Which Ones are Necessary? It enabled continuing research, wide-spread CF diagnostic testing and newborn and carrier screening, and facilitated development of CF therapeutics. Joan Germana, May 22, It is also worth noting that the drug resulted from a partnership between a disease advocacy organization and a for-profit firm, and the three-month priority approval process at FDA was expedited by trials that involved patients, ages 6 to DiSante recalls that they only had 2—3 days to complete the patent application so that it could be filed before they could publicly confirm that the gene had been identified.

Cook-Deegan R, Heaney C. In that instance, the U of M was informed that a laboratory was advertising CF testing, while not adhering to quality control standards or the professional medical guidelines for testing and counseling. However, the main challenge posed by conferring exclusivity to a gene therapy company was that there were several potential venues through which exclusivity could be granted: Had CF diagnostic testing not become as accessible as it was, these life expectancy improvements may have been less impressive or happened later. Indeed, DiSante recalls receiving many phone calls from biotechnology companies interested in taking out exclusive licenses for gene therapy research. Wide access to genetic testing and screening made it easier for states and hospitals to implement newborn screening programs; earlier detection of CF meant that patients could be started on nutritional supplementation sooner; and medical care providers could more aggressively intervene to prevent lung infections, a leading cause of death among CF patients. The publisher’s final edited version of this article is available at LES Nouv.

But the U of M did not want to exclusively license the gene itself, because that would block development of alternative delivery and insertion systems for gene transfer, as well as using the CFTR gene or CFTR protein as therapeutic targets.

Cystic Fibrosis Patents: A Case Study of Successful Licensing

Mutated CFTR protein results in a buildup of thick, viscous mucus in the lungs, digestive tract, and reproductive system. Generic Biotechnology Drugs Posted on: Exclusive licensing is quite common as an incentive to startups, and in this case a particular vector system was covered.


hesi case study cystic fibrosis

Identifying the genetic basis of cystic fibrosis Cystic fibrosis CF is a genetic disorder long known to be inherited as an autosomal recessive character, and to be highly variable in its severity, duration, and spectrum of symptoms. Impact of gene patents and licensing practices on fibross to genetic testing for hereditary hemochromatosis.

When the CFF was founded ina child born with CF was not expected to survive until elementary school; in contrast, the life expectancy today is over 37 years, and is increasing at the rate of about one year per year. Which documentation further supports the diagnosis of CF? Considerations for therapeutics were entirely different. From — it fibrksis also co-funded by the US Department of Energy.

hesi case study cystic fibrosis

The two clinical trials thus required access to patients and their families, a drug-development team, and rigorous clinical efficacy and safety trials that drew heavily on the resources and organization of the collaborating partners, as well as illustrating the new model of therapeutics developed for genomic subtypes.

A stucy days after the party, Sarah came down with common cold symptoms that rapidly deteriorated into a serious lung infection. DiSante recalls that they only had 2—3 days to complete the patent application so that it could be filed before they could publicly confirm that the gene had been identified. Managed Care Considerations Posted on: It is common enough to attract attention, and indeed the success of ivacaftor shows there was sufficient commercial interest to develop a therapeutic for a genetic subtype of low prevalence earning Orphan Drug designation.

Despite these efforts, she has encountered many lung infections that cwse deteriorated her lung function over time. What are standards of care? Ethical Choices Posted on: Prior to this analysis, there was an assumption that with CF being a rare, orphan disease that any CF treatment would not generate much revenue.


PRIME® Clinical Case Study: Cystic Fibrosis Amidst the Zest for Life in Youth

No notification letter was sent, and apparently the laboratory quietly withdrew from the market, or at least stopped advertising its CF testing service so publicly. Srudy statement indicates a correct understanding of the etiology of CF? Not much was known about whether a potential protein-based therapeutic could be developed, since the function of the CFTR gene was not yet known, other than hints it was an ion channel for chloride. Which nursing intervention would be appropriate?

Discuss the importance of playing games that require Debbie to do physical activity such as running, jumping, or skipping.

Cystic Fibrosis Patents: A Case Study of Successful Licensing

Cawe the Public Interest: Sarah spends upwards of an hour and a half every day receiving breathing treatments and chest physiotherapy in order to maintain her lung health.

Although this particular licensing strategy is currently only used by the U of M with respect to the CFTR patent, Ritchie does draw from it to help draft other licensing agreements with other entities:. Three days ago, Sarah was diagnosed with a pulmonary infection, which presented only 3 months after a prior pulmonary infection treated with IV antibiotics.

FDA approves Kalydeco to treat rare form of cystic fibrosis.

hesi case study cystic fibrosis

Therefore, patenting made sense to the scientists, their nonprofit institutions, and disease advocacy groups. While the U of M administers all U. Join our CME mailing list! The inclusion and active participation of the CFF patient advocacy organization was another important factor in the initial patenting and licensing discussions. Subscribe to RSS Feed. Update on carrier screening for cystic fibrosis. Sarah had never felt so alive and normal. The clause was incorporated into every license the U of M has issued since.