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The R&D Process

Every day, scientists in the biopharmaceutical industry research the molecular underpinnings of disease, screen compounds against new disease targets, and conduct clinical trials with thousands of patients atlocations around the globe. The driving goal of the sector is to find new medicines that improve medical care and address unmet medical needs.


In 2010, despite the challenging economic environment, the sector maintained its strong support for innovation. Biopharmaceutical companies invested an estimated $67.4 billion in the search for new medicines. Developing a new medicine is a long and complex process, with many setbacks and challenges. The R & D process is becoming increasingly difficult, expensive, time-consuming and risky, costing $1.3 billion on average.


Researchers are working to find new ways to approach the R&D process to make it more efficient while maintaining the highest safety and efficacy standards. The process is evolving to make use of the latest statistical techniques and research tools.


The R&D Process


Only about one in six drug candidates that enter clinical trials are ultimately submitted to and approved by the FDA, according to a study of the 50 largest companies – many candidates fail as late as phase 3 trials.For the small share of drug candidates that do become approved drugs, it takes about 10 to 15 years from the initial discovery to availability for treating patients. The process requires both flashes of inspiration and persistent dedication. Researchers must creatively tackle unforeseen challenges and thoroughly collect data on all aspects of the drug’s safety and efficacy.


Drug Discovery and Preclinical Testing


Extensive basic research lays the groundwork for understanding the disease to be treated and, if possible, the underlying cause. Researchers may contribute to this work from across sectors, including academic institutions, government labs and biopharmaceutical companies.


With this knowledge, biopharmaceutical researchers look for a molecule or “lead compound” that may alter the disease course. They may screen compound libraries, develop a molecule from scratch or use some substance from nature as the starting point. After many safety and efficacy tests, they often redesign the most promising compounds to optimize their disease-fighting properties. Often, hundreds of variations are pursued.


The Research and Development Process


The next step is to test the optimized compounds in the laboratory to find the most effectiveness lead with a safety profile that supports initial introduction into humans. Scientists try to determine how a compound works describes its safety profile


If the compound appears to be safe and effective, the company submits an Investigational New Drug Application to the FDA to seek approval to begin clinical trials.


Clinical Trials


A critical part of the R&D process is clinical research, the study of a pharmaceutical product in people. Clinical research involves both potential benefits and potential risks to the participants, and research-based biopharmaceutical companies place great importance on respecting and protecting the safety of research participants, ensuring scientific integrity, and disclosing clinical trial results.


Before a trial begins, researchers develop a protocol, or plan, for the trial, laying out exactly what information they are collecting and how patients’ safety will be protected. The clinical trials process lasts an average of six to seven years and usually involves thousands of patients and several different phases of research:


Phase 1 trials (20 to 100 volunteers) – Phase 1 trials are usually performed in healthy volunteers. These studies are designed to determine if a drug is safe in humans, what the safe dosing range is, and if the drug should move on further testing.


Phase 2 trials (100 to 500 patients) – In phase 2 trials, researchers study patients with the disease or condition in question, and also identify common, short-term side effects associated with the treatment.


Phase 3 trials (1,000 to 5,000 patients) – Phase 3 trials study a drug in a much larger patient population and allow researchers to collect data on a drug’s safety and efficacy for the evaluation of the overall benefit-risk profile of the treatment for a particular patient population. Phase 3 trials are the longest trials, and often take place in literally hundreds of sites across the United States and throughout the world.


New Drug Application/ Biologic License Application


If clinical trial findings indicate that a drug is both safe and effective, the company files a New Drug Application (NDA) or a Biologic License Application (BLA) with the FDA to request the medicine be reviewed for approval. The FDA reviews the application, which can run 100,000 pages or more, to assess the data from all testing done since the beginning of the process. The FDA uses these data to determine whether a drug’s or biologic’s benefits outweigh any risks, what information should be included in the medicine’s labelling, and whether the proposed manufacturing process is appropriate.


Post-Approval Research and Monitoring


Research does not end with FDA approval. Companies continue to monitor the safety of the product as long as it is available to patients, and often conduct research on new potential benefits of the medicine in other disease areas or patient populations. For the entire life of the medicine, teams of scientists and physicians collect safety data on a daily basis and report potential problems to the FDA. For example, the FDA requires:


Reports on safety issues every three months for the first three years after approval; annual reports as long as the medicine is marketed.

Adverse events reports within 15 days of event (seven days for a life threatening event).


In addition, the FDA may require companies to conduct “Phase 4” studies as a condition of approval to evaluate the long-term safety and effectiveness of a medicine or its effects on a subset of patients.


Companies often also continue to research expanded uses and benefits of a medicine after approval, leading to growing understanding of the full benefits of a given treatment. For instance, the medicine may be used earlier in the disease process, for different diseases, in combination with another medicine, or in combination with specific biomarkers to better predict response to treatment. As research accumulates after approval, new benefits of a medicine are identified.


Advancing R&D Methods


Research and development of new medicines is not a static process. Researchers are always looking for new and better ways to innovate. As the process has become more complex, expensive, and time-consuming in recent years, researchers have redoubled their efforts to improve the R&D process. They are using new technologies and more sophisticated methods for analyzing data to make the process more efficient while still maintaining the highest safety standards.


SOURCE: Pharmaceutical Research and Manufacturers of America, Pharmaceutical Industry Profile 2011






We have been piloting campaigns that we hope would ensure the integrity of pharmaceutical products to safeguard the public against fake and substandard medicines.


Foremost to these campaigns is our active involvement in the Safe Medicines Network or SMN which is a multi-stakeholder partnership that advocates for the provisions of safe and quality medicines. Under SMN are various committees. PHAP has been tasked to lead the SMN Enforcement Committee which will create a database of counterfeiting developments. Through the efforts of SMN, we were able to designate a hotline for the reporting of fake medicines. Four our information, the hotline is 1-800-10-FAKEMED. Since there is a need to beef up awareness about the fake medicines menace,the SMN has also been conducting information and education campaigns in the judiciary and local government units in the provinces.


Robust investment in research and development (R&D) by biopharmaceutical companies has resulted in advances and discoveries unlike anything we’ve seen before. In the last decade we have invested half a trillion dollars, and these investments are just beginning to pay o , opening the door to entirely new ways to tackle some of the most complex and di cult to treat diseases of our time.i


The progress we’re seeing today is revolutionizing how we treat disease, saving patients’ lives and improving quality of life and public health across a broad range of chronic and

rare conditions. In this new era of medicine, many diseases previously regarded as deadly are now manageable and even curable. And today, there are about 7,000 medicines in clinical development around the world.ii Across the medicines in the pipeline, 74 percent in clinical development have the potential to be

rst-in-class treatments.iii The future has never been brighter as researchers are exploring new frontiers that just a few years ago may have been regarded as science ction, but are now transforming patients’ lives.


This new era of medicine isn’t just good news for our health— it’s good news for our health care system and society. New innovative medicines keep patients healthy and out of the hospital, reducing the need for costly emergency room visits, hospital stays, surgeries and long-term care, ultimately saving money for patients and the nation’s health system. (source:


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