Jeff Antos, The Weinberg Group

I have the privilege of regularly speaking with emerging pharma, biotech and device executives, all of whom ask variations of the same question: “What’s the best way to get my product approved and marketed?” Of course, each executive brings his or her own specific expertise to the project; one might be a clinical physician, another a PhD scientist, another an intellectual property attorney, etc. Because no one CEO has all the skills necessary to successfully launch a drug, virtual companies bring in expertise from outside organizations.

Often, an emerging company needs to select the best product candidate from a number of possible compounds and indications in its portfolio. In order to narrow the options down to just one or two compounds, a number of elements must be considered. The remainder of this article provides a brief method for winnowing the list down to a navigable set of possibilities.

There are six interconnected development elements that must be considered and successfully executed to bring a drug or device to market. As a memory aid, I use the acronym PRIMES: Production, Regulatory, Investment, Marketing, Exclusivity and Science. The dictionary definition of primes is “To prepare someone for a situation or task, typically by supplying relevant information,” which seems appropriate.

Each element impacts some or all of the other elements. For example, if we are considering orphan drug exclusivity for our product, that will impact production (small volume), regulatory (application for Orphan Drug Designation), investment (enhanced by ODD), marketing (to a small, defined segment, perhaps with advocates) and science (efficacy must be demonstrated on a smaller patient sample).

Production: Can we manufacture the product?

Most emerging companies use the services of a contract manufacturing organization (CMOs). Some of these organizations also assist in product development and are referred to as CDMOs. In selecting a CMO, make sure that it is familiar with your product type and route of administration. Volume is also important; your forecasted output should match the capacity of the CMO.

Regulatory: What is the path for Agency (FDA, EMA, etc.) approval?

Since the objective of the drug development process is Agency approval, the regulatory plan is of paramount importance, defining key meetings and submissions to FDA/EMA, while integrating nonclinical and clinical plans. A variety of scientific disciplines are necessary to develop and execute the plan: nonclinical/toxicology, medical/clinical, Chemistry Manufacturing Controls (CMC) and biopharmaceutics/pharmacology. We sometimes refer to this key document as a “Roadmap to Approval.” It is essential that we have an optimal plan before presenting to Regulatory Agencies or investors. Inevitably the plan will change during development. Perhaps Dwight Eisenhower put it best, “I have always found that plans are useless, but planning is indispensable.”

To achieve each milestone in the drug/development process, you will make an investment which will diminish your risk and increase the value of your asset. At each successful milestone completion, investment and value increase as risk diminishes. Early funding is usually provided via grants or individual investors. Private equity firms typically wait until Phase II of development, when risk is lower and potential rewards are greater. In reality, this means that achieving the opening of an Investigational New Drug (IND) application is of paramount importance enabling improved funding and licensing opportunities.

Marketing: What is the commercial value of the product?

Early in the process, it is important to get a sense of the commercial value of the product. This is particularly important if you are trying to select one product and/or indication from a number of possibilities. A standard is often selected for comparison purposes, e.g. mature annual sales five years after product launch. Numerous variables affect the commercial value including NDA vs. ANDA, exclusivity/patent protection, disease prevalence, competing products, unmet need, etc. It’s also useful to know the commercial value of existing therapies.

Exclusivity: Do I have patent or other protection?

It is critical to get as much patent protection as possible early in the development process, engaging an intellectual property attorney who specializes in drugs or devices.

The ultimate value of your product will be based on its patent protection and other exclusivity afforded by Regulatory Agencies. A helpful summary on the topic is provided by FDA at https://www.fda.gov/media/92548/download

Of course, this is the key question and the reason clinical trials are conducted. The 505(b)(2) provisions of the Food, Drug, and Cosmetic Act enable sponsors to use prior approvals and/or scientific literature to make the case for safety and efficacy, a great benefit considering that clinical trial costs range from $10,000 to $50,000 per patient. Clinical trials are typically conducted by clinical research organizations (CROs). Try to choose one that best fits your therapeutic area, stage of development and corporate size.

As you move forward in your pharma development, keep PRIMES in mind to make sure that you are considering all of the elements necessary for your success.

Jeff Antos is Vice President at the Weinberg Group, a ProPharma Group company (www.WeinbergGroup.com). For almost 40 years, The Weinberg Group has served the global pharma industry, providing companies of every size with regulatory and compliance support.

Articles

Guidance for companies starting out on the commercialization path

PRIMES: The six key elements of successful drug/device development

Since the objective of the drug development process is Agency approval, the regulatory plan is of paramount importance, defining key meetings and submissions to FDA/EMA, while integrating nonclinical and clinical plans. A variety of scientific disciplines are necessary to develop and execute the plan: nonclinical/toxicology, medical/clinical, Chemistry Manufacturing Controls (CMC) and biopharmaceutics/pharmacology. We sometimes refer to this key document as a “Roadmap to Approval.” It is essential that we have an optimal plan before presenting to regulatory agencies or investors. Inevitably the plan will change during development. Perhaps Dwight Eisenhower put it best, “I have always found that plans are useless, but planning is indispensable.”

To achieve each milestone in the drug development process, you will make an investment which will diminish your risk and increase the value of your asset. At each successful milestone completion, investment and value increase as risk diminishes. Early funding is usually provided via grants or individual investors. Private equity firms typically wait until Phase II of development, when risk is lower and potential rewards are greater. In reality, this means that achieving the opening of an Investigational New Drug (IND) application is of paramount importance enabling improved funding and licensing opportunities.

The ultimate value of your product will be based on its patent protection and other exclusivity afforded by Regulatory Agencies. A helpful summary on the topic is provided by FDA at 