Mapping the pathway

The U.S. pharma market is the largest pharma market worldwide, and continues to grow. According to GlobalData, it was worth around $395.2 billion in 2014, and is estimated to rise to $548.4 billion by 2020. This make it a very attractive one for biopharma, and provides the opportunity to improve patient outcomes for a large population.

The benefits offered by the U.S. market include support for the biopharma industry from the government, and access to a well-educated and experienced workforce. Innovation is welcomed in the U.S., particularly where a product or a device offers cost savings through efficacy, safety and improvements in patient outcomes, and this is backed by a well-managed intellectual property system, transparent patent protection processes, and market exclusivity for orphan drugs.

Drug development is expensive, with estimates of the costs of moving a drug from the bench through clinical trials to the market ranging from $92 million to $2.6 billion. In order to make the most of the U.S. market, companies need to be able to establish a balance between a return on investment, with money to put back into drug development, and setting a price that supports market access by getting the right drug to the right patient.

The route to success: Plan early

There are many steps involved in launching a product in the U.S., in particular for one that is manufactured in another country. Ideally, the process of gaining market access should begin at least a year before launch, and the key to success is early planning.

The challenges include:

  • Protecting the IP
  • Understanding the process and regulations involved in importation
  • Learning about the market landscape and payers, including where the product fits in the market, and who will use the product
  • Getting the product onto the third payer formulary
  • Gaining a favorable reimbursement position with minimal or no restrictions
  • Proving product value to allow reimbursement and to support negotiations to reduce levels of co-pays

Take a structured approach

Creating a structured approach to the market access process ensures that everything needed to launch a product is accomplished and nothing is overlooked. It is important to know the prescriber landscape and patterns of use, and this drives the choice of distribution model, such as creating a limited distribution model for a drug for a rare condition prescribed by tertiary physicians, rather than primary or secondary healthcare professionals.

Distribution models also need to be tailored to the needs of the drug, the patient and the company, for example taking into account sales volumes. For rare conditions, such as infantile hemangioma, which was a topic of a recent Quintiles webinar, pharmacies are unlikely to keep therapeutics in stock. Ensuring that specialty pharmacy partners are in place means that prescribing physicians can provide patients with access to a treatment within 24 hours. This will be especially important in urgent cases where a condition is serious or life-threatening.

The payer landscape is important, at a time when healthcare and drug budgets continue to tighten. In managed care, price is very important, and one of the challenges is to provide payers with the right clinical information, including safety, efficacy and costs, in comparison with current therapy.

Communication is key

Communication is vitally important in the time leading up to launch, and has to be tailored to audiences, taking into account the amount of information that is allowed to be imparted according to regulations.

It is important to create stakeholder networks. These can be used to educate payers and healthcare professionals about the disease and its prevalence, and discuss how they are using existing therapy. Provided this does not include specific product mentions, communication can be started before approval and launch. Typical activities include discussions at conferences, engaging patient advocates, and educating the stakeholders, from payers to parents and caregivers.

For new entrants to the U.S market the communications process also needs to establish the company and its product pipeline with payers. This communication must be relationship-driven through national account managers, medical science liaisons and professional sales representatives, particularly as payers and physician’s time is limited.

Setting objectives

Part of the planning process is meeting client and product objectives. These may be based on:

  • communications, for example visiting a specific number of payers, creating a KOL network, setting up a certain number of seminars at congresses, or collaborating with patient advocacy groups
  • pricing and reimbursement
  • prescribing levels
  • having products loaded correctly in compendia and payer account systems.

Launching a drug does not always go to plan, and so the process needs to incorporate not just plan A, but plan B, C and D. Don’t forget to expect the unexpected.


To gain more insight into how Quintiles helped support a European company with its orphan drug launch into the U.S., watch our webinar to get our key learnings.

Topics in this blog post: Evidence, Market Access, Orphan Drugs, Payers