Generating Pharmaceutical Sales Before Marketing Approval

Dr. Gene Emmer 

When a patient has a serious illness and there is no approved drug available, the physician may want to try one which has not been authorized for marketing by national health authorities but has shown promise in clinical trials. European Named Patient Programs, like US compassionate use programs, offer physicians access to pharmaceuticals which have not yet been licensed. However, there is one important difference: in Europe an unlicensed drug can be reimbursed. This presents drug-makers with an opportunity to generate revenues while development is still in-progress.

Significant Revenues Are Possible
The additional revenues can be considerable. For example, Pharmion, a US based company focusing on Oncology and Hematology reported dramatic increases in its Thalidomide sales from $1.9 million in 2Q ‘03 to $15.3 million in 2Q ‘04, primarily due to named patient sales in Europe for Multiple Myeloma. Thalidomide sales accounted for approximately 75% of Pharmion's total revenues for the first half of 2004, according to company sources, and were generated while the product awaits marketing approval for this indication. Before receiving European Marketing Approval, Shire's Argylin® for essential thrombocythaemia generated about 5% of its total sales from its European named patient program.

Though thalidomide and Agrylin were licensed in the USA for some indications, pharmaceutical companies do set-up named patient programs and receive full reimbursement for drugs that are not licensed for any indication in any market. Examples include: Insmed's SomatoKine® authorized for named patient use for Primary Lateral Sclerosis, Growth Hormone Insensitivity Syndrome (GHIS) and Severe Insulin Resistance and Protherics' ViperaTAb™ authorized for named patient use for adder snake bites.

Other Benefits of Named Patient Programs
A named patient program can speed uptake after official launch. Physicians, who have had experience before launch, via clinical trials or named patient programs, often become early adopters and references for other physicians once the drug is freely circulating.

Named patient programs, like US compassionate use programs, can increase good-will toward the company because they simplify the process of gaining access for patients in critical need. Smaller companies often can not afford the administrative time and costs of shipping drugs around the world before launch. This can lead to frustration and resentment towards a company that many physicians will remember long after a drug is officially on the market. Creating a formal channel eliminates the unfortunate need of denying requests and risking ill-will later.

A named patient program should be considered an important part of a pre-launch program. It increases awareness to a pharmaceutical's existence, creates excitement, generates good-will and speeds penetration of the product after launch.

Frequent Communication is Necessary
If one of the objectives is to generate revenues, setting up a named patient program is just the beginning. In order to achieve success, physicians need to be aware of the product and what they need to do to get it. Typical methods of informing physicians, such as sales rep visits and ads, may not be appropriate because a license is necessary to market a drug. While physicians are used to simply writing a prescription and being done with it, named patient programs require paper-work that some find tedious. Therefore the company needs to create an appropriate communication plan and work closely with the targeted medical community to keep them informed and simplify the process.

Issues to Consider
You have decided to make a named patient program part of your pre-marketing plan, what now?

• Administration: Do you “go it alone” or work with a company that is experienced at administering named patient programs?

There are several experienced organizations that can assist your company by gaining approval, setting-up the program, doing administration and taking care of physical distribution. If your organization is well resourced and has a pipe-line of products that will require named patient programs, it might be worthwhile acquiring the expertise internally. However, if you have few appropriate products or a stream-lined organization, it is probably best to consider outsourcing.

• Communication: You've set up the program, how do you optimize it?

If you do not have an experienced European marketing group, an organization that is familiar in sales and marketing of pharmaceuticals in Europe can help you to maximize participation in the named patient program. A communication plan, if properly developed and implemented can increase product awareness, but communication concerning an unlicensed product must be done appropriately. This plan should ensure that your entire target group:

• Is fully aware of the product AND the program
• Knows what needs to be done to take advantage of the program
• Has an advocate available to guide them through the process