Biotechnology

1. Biotechnology Presented by Megan Hopkins-Brady

2. Objectives: To define how new developments in biotechnology are revolutionizing the healthcare industry. New drugs discovered and developed by the Biotechnology Industry are being used to treat diseases, that were previously not available; this technology is novel compared with traditional pharmaceuticals. These drugs are modified versions of naturally occurring substances in the body.

3. Biotechnology • Biotechnology is a field of applied biology that involves the use of living organisms and bioprocesses in engineering, technology, medicine and other fields requiring bio-products. Modern use similar term includes genetic engineering as well as cell- and tissue culture technologies. • Biotechnology draws on the pure biological sciences (genetics, microbiology, animal cell culture, molecular biology, biochemistry, embryology, cell biology) and in many instances is also dependent on knowledge and methods from outside the sphere of biology (chemical engineering, bioprocess engineering, information technology, bio-robotics).

4. Biotechnology Vs Pharmaceuticals • Most traditional pharmaceutical drugs are relatively simple molecules that have been found primarily through trial and error to treat the symptoms of a disease or illness. • Biopharmaceuticals are large biological molecules known as proteins and these usually target the underlying mechanisms and pathways of a malady; Biopharmaceuticals can deal with targets in humans that may not be accessible with traditional medicines. A patient typically is dosed with a small molecule via a tablet while a large molecule is typically injected.

5. Biotechnology Vs Pharmaceuticals • Biotechnology is also commonly associated with landmark breakthroughs in new medical therapies to treat hepatitis B, hepatitis C, cancers, arthritis, hemophilia, bone fractures, multiple sclerosis, and cardiovascular disorders.

6. Technologies • Hardware used in Biotechnology • High throughput screening techniques to discover drugs • Animal models to test if drugs have potential therapeutic benefits • Recombinant DNA Technology used to produce drugs • Bioreactors and chromatography to manufacture biotechnology drugs for clinical trials and patients • Software • Statistical analysis software such as Minitab or SAS

7. Hardware: Bioreactors • Large steel tank • Cells are grown in mixed environment where nutrient levels and pH, temperature and oxygen levels are controlled • While the cells are growing, they produce the protein of interest in large quantities. • The protein can be easily purified from the cells and other proteins using purification techniques such as chromatography

8. Software: Statistical Analysis • Used to analyze large datasets • Used to determine statistically if drug has positive effects on patients • Used various statistical methods such as correlation, regression and ANOVA to perform analysis • Different packages exist: Examples include Excel, SAS, Minitab and JMP

9. Minitab software • Statistical software package • Allows easy input and analysis of large volumes of numerical data • Also allows the user to design clinical trial studies in order to determine what number of patients are needs and what groups to study • For example, the program allows the user to run tests of significance (such as ANOVA, t-test, f-test) and correlation/regression analysis. • Such analysis allows the user to determine if the effect is statistically significant.

10. Information System Review • Computerized data information systems are routinely used to store and analyze clinical trial data • The Food and Drug Administration guidelines requires that firms conducting clinical trials have appropriate systems in place (FDA, 2007) • The computerized systems should be designed: (1) to satisfy the processes assigned to these systems for use in the specific study protocol (e.g., record data in metric units, blind the study), and (2) to prevent errors in data creation, modification, maintenance, archiving, retrieval, or transmission (e.g., inadvertently un-blinding a study).

11. Information System: Clinical Trials • Used by clinical trail administrators, nurses, physicians, study directors and data analysts • System must be capable of accurately capturing and analyzing all aspects of clinical trial data

12. CliniProteus • CliniProteus is a scalable and flexible integrated solution intended to cater to the needs of the clinical research organization of any size (Matura et al, 2007) • XML based flexible clinical trials data management framework in .NET environment that can be used for efficient design and deployment of Electronic case-report forms (eCRFs) to efficiently collate data and analyze information from multi-site clinical trials.

13. CliniProteus Features • The system has the following functionality to support clinical trials: • End User Administration • Patient registration • eCRF • Verification of eCRF data entry • Maintain inclusion/exclusion criteria, adverse event log • Visit management • Shipping & handling of Samples • Clinical sample data integration • Customized reports • Research analytics.

14. Advantages of Biotechnology Trend • New or improved cures and therapies for previously untreated diseases being developed. Major indications include • Autoimmune diseases such as arthritis, MS and Crohns Disease • Cancer and oncology • Neuro-degenerative conditions such as Parkinson’s and Alzheimer's • Infectious diseases such as Hepatitis, AIDs and common flu • Filling pipelines of pharmaceutical companies and creating new Biotechnology companies.

15. Disadvantages of Biotechnology Trend • Relatively unproven drugs and technologies • Low success rates of drugs in clinical trials which cost a lot of money • Some concerns over the safety of the drugs • Expensive to produce • Drugs are difficult to produce relative to conventional small-molecule therapies.

16. Ethical/Legal issues • Ethical • Some companies developing therapies using stem cells derived from pre-term birth tissue • Use of animals for pre-clinical studies to determine if drugs works in animal models • Legal • Drugs and technologies are typically patented, protecting the rights of the discoverer, but not allowing other companies to develop drugs, creating potential monopoly. • Potential to sue company if safety issues observed in the field that were not previously seen in clinical trials.

17. Informatics Competencies Required • Nursing professionals working in clinical trials with the drugs would need to be familiar with the following • Data acquisition system used in the clinic to capture data from the trial including patient information, drug lot numbers, patient clinical data, etc… • System used to analyze data to determine if drug was working

18. Functions and Responsibilities of IN Working with this trend • Collect and enter relevant patient information into data system; typically the clinical trial protocol will have instructions on what data to enter. • Administer the therapeutic drug according to the protocol • Collect samples from the patient and store appropriately • Monitor patients symptoms and progress over the duration of the trial. Enter relevant data into data acquisition system.

19. Summary • Biotechnology companies are developing novel cures and therapies for previously difficult-to-treat diseases • Such therapies promise to revolutionize the field of medicine • Nursing professionals should be kept informed of developments in this important field.

20. APA Reference Guidelines FDA, Guidance For Industry: Computerized Systems used in Clinical Investigations, March 2007. Mathura, VS; Rangereddy, M; Gupta, P; Mullen, M; Bioinformation. 2007; 2(4): 163–165. Robbins-Roth, C. (2001). From alchemy to IPO. Cambridge, Massachusetts: Perseus Publishing. Wikipedia: The free encyclopedia. (2004, July 22). FL: Wikimedia Foundation, Inc. Retrieved September 18, 2010, from http://www.wikipedia.org