08 August 2012

Shot in the arm: for the Patient?,
      for Pharma?, or for Both!?

- New vaccines and drugs are developed and made available only after rigorous study, stringent methodologies, human effort, and enormous cost, not to mention long nights in the lab. From a good idea or concept, to the marketing phase of a new treatment, every step is laden with eureka moments as well as downsides. Considering that we are in the age of new technology and discovery, the number of new drugs approved has declined progressively in the past decades. Billions of dollars are allocated for research and development, and yet, the proportion of budget to production has not been compensatory. As a matter of fact, all the “drug dollars” do not necessarily imply phenomenal pharmaceutical developments. Some things have lagged behind:definitely not ideas, but rather the management of resources, methods, and the sub-optimal analyses of mountains of data. But that is changing now, with the advent of number crunching SaaS (Software-as-a-Service), PaaS (Platform-as-a-Service) , and the systematic analysis of data that is either structured, loosely structured, or unstructured. This is being done using new algorithms, improved programming languages, hardware, and interfaces.

- In Medical School of past days, prospective M.D.s were taught how to concoct cough syrup almost like an alchemist’s recipe. (Do not try this at home!) Of course, eventually the proud Medical Student would be beaming with an explorer’s wonder. Well, as it goes, you could buy these compounds pre-packaged, over-the-counter at your favorite drugstore. But it was a great achievement to be able to explore one’s own process of discovery. This element of curiosity combined with guided instructions (and trial and error, or experimentation) would be the hallmark of Pharma R&D. Nowadays, pharmaceutical set-ups “hire” IT companies who specialize in the management of data and costs that are an ever-present challenge. Budgets may be tighter now due to the recession and this predicament actually compels drug developers to work smarter and safer.

- Doctors and Pharma Professionals now team up with Medical Informatics groups to make the most of the development process. This may mean that a potential drug is either dropped totally at some point in the trial, thereby not wasting any resources, or it continues towards the progressive trial phases. The phases start with drug design and drug discovery, go on to animal testing, then begin with testing in only a few selected human subjects. A good protocol design is necessary. If there has been no peril to the patient/s or "failure" of a drug candidate, or any undesirable effect, they then expand to test in many study participants. If initial data look promising, the drug company can start working on submission of preliminary data to the Drug Safety and Regulatory Boards. All this effort leads towards cure or amelioration of the pathologic condition, without sacrificing patient safety. Investigator/s' biases have to be set aside completely. (This may be debatable, considering nobody has absolute zero biases.) Financial disclosure must be revealed to authorities or to the public readership. Premature conclusions should not be announced to the news media carelessly. This has led to public confusion and even panic at times.

- With these goals in mind, Doctors, and Pharmacologists (as well as Toxicologists) are now embracing Medical Informatics well beyond the scope of the basic Electronic Health Record. The enhanced abilities of drug development teams enable them to demonstrate to sponsors, giving them a  better understanding of the scope and significance of the drug in mind. In effect, it is basically a kind of business proposal geared towards a humane purpose and the rationalization of costs. Return on Investment (ROI) in terms of helping the community that needs the drug, and the financial reward are both imperative in the eyes of sponsors and stakeholders of the company. After all, we are all partners in this endeavor.

- The development of genomics and use of biomarkers have consequently led to the process of pharmacogenomics. In this process, the likelihood of cure is increased due to the pinpoint target concept. Genomics is moving slowly but steadily year by year now. And let’s not forget stem cell technology. Accumulating evidence is demonstrating that we need a paradigm shift in mode of treatment. Possibly, within 5 years, we will see a jump in the number of biological, proteomic, and nanovector (and virusvector) treatments, all of these pointing to highly defined molecular targets. Nevertheless, whether chemical, or molecular, Big Data from clinical trials and post-market vigilance is posing a tremendous challenge to analysts. When it comes to data crunching, we have the offline spreadsheet type of tools, or programs like SPSS and SAS, where we carry out our robust statistical calculations. Now, online and cloud based services can equip us to handle mountains of data. Some of these programs are geared towards clinical trials, while others such as OpenEpi, analyze epidemiologic trends. You can find a number of free statistics programs here. Clinical trial protocols have become far more complex during the past decade. Both clinical and epidemiologic study data are currently subjected to more rigorous scrutiny by either new algorithms or graphic mathematical procedures like meta-analyses. New programming languages are utilized and expanded with the purpose of making all the data more relevant. Languages like R, Ruby, Python, Java (Jython too), and even Matlab are being used in ways that enhance our analytic processes.

- For example, one data analysis group of consultants is Medidata Solutions providing cost databases for clinical trial analysis and “advanced reporting and analytics to drive clinical development efficiencies.“  ModgraphConsultants Ltd. an international software company specializing in the pharmaceutical industry, is offering software solutions in chemistry, cheminformatics and molecular modeling. Pharmaceutical departments are mostly serving drug development projects. Systematic and secure document handling is of utmost importance. These documents, recording all the processes and developments of the drug in focus, have to be treated just like any top-level dossier. This leads to an improvement in production and lesser time-to-market. Drug companies as well as the Regulatory Safety Boards in each country and continent also have to consider post-market information, and adverse drug effects (ADE) recording and collating. An example of such a service is the Infotenha Group who, with their software suite as their complete solution, “manage information dealing with regulatory affairs, quality assurance / quality control, Research & Development and Pharmacovigilance.”

Drug companies have to increasingly adhere to auditing activities and security protocols in their IT ecosystems. The U.S. F.D.A. has laid out Guidelines for clinical trials . You can check out this excellent article on MRM.This ensures that no stone is unturned and no leak unplugged. Drug discovery is going through exciting times and new treatments and cures are becoming more promising, provided that the Medical and Pharma Communities meld with IT folks, and cooperate in a new unified paradigm. At any rate, I think that this approach will save us unnecessary losses, and push us further on towards great heights.

- Hippocrates (c. 460 BC – c. 370 BC)
         (Translated from the original Greek)
Vita brevis,
ars longa,
occasio praeceps,
experimentum periculosum,
iudicium difficile.

Life is short,
and Art long,
opportunity fleeting,
            experience perilous.
 - Keep moving forward and change the world. And remember, the patient is THE MOST IMPORTANT CLIENT! They deserve our very best.

- Fernando Yaakov Lalana, M.D.

No comments:

Post a Comment