The Collaborate | Innovate Challenge calls on participating organizations to submit innovative concepts that translate patient insights into improvements in the drug development process. Read more from Sanofi’s own Marc Bonnefoi, DVM, Ph.D., Head of the North America Research and Development (R&D) Hub for Sanofi to learn more about why involving patients is so critical and what the major stages in the drug development process are.
Q: What are your top priorities at Sanofi R&D?
A: At Sanofi R&D, our main objective is to discover and develop healthcare solutions to help improve the health of people worldwide, and we’re trying to do this faster than ever before. Today, that is a lot easier said than done as the healthcare solutions we are working to develop are aimed at treating very complex diseases.
In my role as Head of the North America R&D Hub, my priority is to ensure we are leveraging our R&D resources in the region as effectively as possible to focus on R&D projects that will have a meaningful impact on the lives of patients and their families. We have passionate, experienced teams at Sanofi and my job is to help them succeed.
Q: How has the R&D process evolved since you started working in the pharmaceutical industry?
A: For many years, we had a drug in search of a patient. We screened scores of molecules to see if they had any effect on different models of medical conditions. Today, we begin with an understanding of the underlying cause of a given disease and work to develop a solution to interfere with that process. We are trying to integrate a translational approach to our R&D efforts, applying the knowledge from patient populations much earlier in our drug discovery and development processes and identifying earlier indicators of whether or not a potential treatment will be successful. At Sanofi, we are truly putting the patient first in all of our R&D efforts so that a new product has medical value as well as scientific value.
Q: Are there any other aspects of R&D that you think are important for consumers to know about or better understand?
A: In spite of remarkable scientific progress, our capacity to translate those advances into health benefits generally decreased since the 90’s. In the biopharmaceutical sector, the success rate has dropped from 1 in 8 to 1 in 14, and the length of development has doubled. On average, it has taken 10 to 15 years to develop the drugs that were recently approved. On the other hand, some recent statistics, such as number of drugs approved and cycle time, suggest that we may be at a tipping point. Patients, their family and society at large should continue to expect more from R&D: it is our main driver.
Q: Can you give us an overview of the R&D process?
A: Research and development (R&D) is an integral part of the drug development process. Developing new drug compounds to bring into the market is a long process, often taking more than ten years and costing more than $1 billion. These time and money investments are made with no guarantee of success.
The main stages of drug development include (1) pre-clinical testing, (2) the Investigational New Drug application (IND), (3) clinical trials, and (4) the New Drug Application (NDA).
(1) Pre-clinical Testing
Most drugs that undergo preclinical testing never even make it to clinical trials. The drugs that do must undergo the FDA’s rigorous evaluation process, which scrutinizes all data. They look at everything from the experimental evidence that the target (the biological structure or process that the drug is supposed to modify) is relevant to the disease to potential side effects to the conditions under which the drug is manufactured.
(2) Investigational New Drug Application (IND)
Even before a drug can begin clinical trials, drug companies must show the FDA results of pre-clinical testing in vitro (i.e. in cell cultures) or in laboratory animals, and what they propose to do for human testing. At this stage, the FDA decides whether it is reasonably safe for the company to move forward with testing the drug in humans.
The information gathered during the pre-clinical testing is called an “Investigational New Drug” application or IND. When the FDA approves the IND application, development progresses to the clinical trial phase.
(3) Clinical Trials
Clinical trials involve three steps:
• Phase I trials – Conducted usually in healthy subjects, these tests determine safety and dosing
• Phase II trials – Conducted to get an initial reading of effective and to further explore safety in small numbers of sick patients
• Phase III trials – Large, pivotal trials conducted to determine safety and efficacy in a sufficiently large numbers of patients
The drug development process continues even after human clinical trials begin. In addition to the tests required to move a new drug into the clinic for the first time, it is also important to ensure that the medicine can be manufactured in a reliable manner in the future, and to determine potential risks associated with long-term treatment or special conditions (e.g. pregnancy, multiple medications taken at the same time, aging).
(4) New Drug Application (NDA)
A drug can be submitted for marketing approval in the various countries where it will be sold if it:
• Emerges from clinical trials with an acceptable benefit/risk profile (medical value for the patient and society vs. potential side effects)
• Can be shown to be reliably manufactured
In the US, this process is called a “New Drug Application” or NDA. Most drug compounds fail this stage, either because they have some unacceptable toxicity, or simply do not show effectiveness in the desired disease indication during clinical trials.
If a drug compound’s NDA is approved, it proceeds to large-scale manufacturing, distribution, and continued testing.
Input from patient communities is crucial to the drug development process. Scientists and clinicians are able to identify indicators of a potential treatment success rate sooner when they apply the knowledge from patient populations much earlier in drug discovery and development processes. We hope you’ll leverage this knowledge and feel inspired to collaborate with fellow organizations to propose new and innovative patient-centered approaches to the drug development process.
Submit your concepts to Collaborate | Innovate before 11:59pm EST on February 23. Follow the Challenge on Twitter and Facebook.
