Professionals focused on drug development are charged with making the most of their resources, maximizing the value of their work, and bringing effective products to market as quickly as they can without compromising quality or safety. It is a long list of responsibilities, and development professionals struggle to balance all of them.
Sameena Sharif, senior vice president of product strategy for Regulatory Professionals, a division of contract research organization (CRO) Premier Research, recently spoke with Outsourcing-Pharma about the benefits of taking a holistic approach to building drug development programs. Adopting such a strategy and philosophy can streamline the process and lead to better outcomes, she said.
“There are a couple of approaches that I think people are leveraging today, or have been heading toward," she told us. “One such approach is leveraging the one drug, one target, and the second is utilizing a multi-target drug discovery strategy. Traditional drug discovery platforms are based on the backbone of the disease targeting a particular aspect of the disease, a receptor, upstream or downstream.”
Holistic design starts off with designing a target product profile that considers the end goal early in the development planning. Also, part of a successful holistic approach, Sharif suggested, is asking thoughtful, appropriate questions at the outset, making sure to consider outcome-focused impactful issues at every stage of development.
“I think starting out with a target product profile is key,” she said. “What do you want your drug to look like? Who do you want to treat? What indication do you want to go after?”
Then, she added, spend time weighing all the pieces of the equation.
“Considering the patient population in question, within those populations and indications, what is that product going to look like?” she suggested. “Even if those thoughts are aspirational, at the very beginning, it’s helpful to keep those things in mind.”
The idea is not to come up with definitive predictions that answer questions ahead of time, she pointed out, but to help guide the process. It can be beneficial to craft a statistical analysis strategy at the early stages, for example, but consider being flexible enough to make adjustments if necessary.
“As the environment changes, you can update the target product profile information,” she said. “As data becomes available, you can change those things.”
Consider the market
Additionally, Sharif suggested, consider the environment and market the end product will be introduced into.
“Having a diagnostic component allows you to carve out a drug that doesn’t have a similar mechanism of action, she said. "It allows you to conduct development in a much smaller targeted population. Think about where the industry is heading. The majority of diseases have multiple presentations and multiple opportunities for tackling treatment by leveraging a multi-drug approach, which has been seen with basket studies.”
With drugs intended to treat rare diseases especially, Sharif said, designers can benefit by shifting from traditional thinking and approaches.
“The more you segment, the smaller the population gets,” she commented. “The model of the blockbuster drug, wanting to make big money, is now not as relevant. Today, there are more data points focusing organizations on rare disease. It’s all about the science and the biology, and shifting the needle a little.”
Sharif also advised drug developers to consider the myriad global regulatory bodies and their potential impact, soliciting advice early in the regions where the product likely will be marketed.
“This does impact your timeline and requires some additional thought on what you can include in your study design,” she said. “Try if you can to stagger the input so you can incorporate as much of the feedback from one agency before interacting with another.”
While Sharif commented both holistic and traditional approaches tend to lead to the same regulatory compliance goal, the former can streamline the process.
“The holistic approach enables asking downstream questions earlier, such as asking about subsequent studies and endpoints, so that this is visible to the agencies,” she said. “Responses from the agency, however, may be similar; it’s data-dependent and a review issue.”
Overall, Sharif suggested, the keys to successfully adopting a holistic strategy is to think broadly, and deeply.
“The bottom line is, you really have to have think through the implications of your initial design on your long-term product development strategy,” she said. “Achieving a broader approach, across multiple geographies, means designing the program as more than just a Phase I study.”