PharmaVOICE Blog Post

Prescribe Robust Distribution to Commercialize Your Orphan Drug

Posted By: Dan Limbach
September 17, 2018

Finding a home for your biopharmaceutical company’s new orphan drug doesn’t just happen after it is FDA approved. The successful commercialization of your promising new drug requires a focused and well-planned distribution model.

But first, some background. Orphan drugs treat rare diseases or medical conditions that affect fewer than 200,000 people a year in the U.S. Since 1983, when Congress passed the Orphan Drug Act, the FDA has approved more than 600 orphan drugs—and the pace is accelerating. From January through mid-September of 2017, the FDA approved 50 new orphan drugs alone.1 That’s a record for an annual total.

If your biopharma company is one of the participants in this rapidly growing field, you want to get patients on your therapy as soon as possible. You also want to keep them on your therapy for as long as necessary. When you have a distribution model that supports these goals, patients can enjoy their best possible health outcomes. And you can enjoy your best possible business outcomes.

Let’s look at how a robust distribution model can help your patients access your new orphan drug.

The right logistics

When your orphan drug enters the market, it targets a specific patient population. So, you need a distribution plan that matches the unique needs of those patients.

That process starts with logistics. Your biopharma company needs a plan that considers these factors:

  1. You need to know how many patients can benefit from your orphan drug. It’s less than 200,000, but is it 50,000 or 100,000? With such a small market, missing even 5,000 patients can make a big difference in your orphan drug’s clinical and commercial success.
  2. Setting. You need to know where your patients are treated. Is it in a hospital setting? Is it at a specialty practice? Is it at home? Getting patients on your drug as quickly as possible is critical. That means getting your drug to the right setting the first time.
  3. Many orphan drugs—like some immunotherapies—are fragile in their composition. They have specific packaging, shipping and storage requirements. Your distribution plan must include details on how to meet those requirements to ensure the safe delivery of your drug.
  4. If you’re an orphan drug manufacturer with a limited patient population, you want to keep tight control on your inventory. Given your drug’s high cost, you don’t want to make too much and have excess inventory. On the other hand, you don’t want to make too little and have an unexpected shortage put patients’ health at risk.
  5. Time is of the essence for patients who need your orphan drug. You need to make access to your drug as easy as possible for those who administer your drug. Hospitals, specialty practices and other prescribers should be able to order your drug easily and quickly.

Added-value distribution

What I described above are the basic ingredients of a distribution model to commercialize your orphan drug. You need to know how many patients you have, where they receive care, how to deliver their drugs safely to them, how much to manufacture and how to accept and fill orders.

On top of those fundamentals should be services that add more value to your distribution model. You need more than just the nuts and bolts. You also need the following:

  1. Patient education. By definition, your orphan drug isn’t like any other drug. It certainly will require specific instructions to patients on how to take it properly to be most effective. Your distribution plan should include a patient education program.
  2. Provider education. You’ll need to educate providers on when and how they should prescribe your new drug. Your distribution plan should include a provider education program.
  3. Patient assistance. Orphan drugs can be expensive. One of the things that can stop a patient from getting and staying on your therapy is the cost. Your distribution model should include a program to help patients navigate reimbursement with their health plans. It should also include a patient assistance programthat can offer co-pay help when needed.
  4. Analytics. Your distribution model should have a way to collect and report transactional data. How much you produced, inventory, sales and purchasers. Your system should also be able to slice and dice that data to get more meaning out of it. You can look at purchasing trends, inventory trends, return rates and more. That information will help you focus on the things you should be focusing on to improve your clinical and business results.

The right distribution partner

Without mastering the basics, you won’t have a successful launch. Without the value-added services, you may prevent patients from getting and staying on your therapy. And without what I call cultural alignment with your distribution partner, you may not have long-term commercialization success for your orphan drug.

What do I mean by cultural alignment? It means that you share the same values and objectives which will help to forge a close relationship between you and your partner for long-term success.

How do you find the right partner? I would argue that the courtship should start 24 months before your drug’s launch. That’s when you should start putting together your commercialization strategy. Many manufacturers are so heads down in getting FDA approval that they wait too long. When approval does come, they’re not ready.

You have to assume that the FDA will approve your drug. Two years before your approval date is the right time to build your commercialization strategy and narrow your list of distribution partners. Then, when FDA approval comes, you can start shipping your drug out that day to help patients.

Waiting until you get FDA approval to vet a distribution partner is too late. Ideally, you will have been working with the same partner for two years before launch and have crafted your commercialization strategy together. Your distribution partner should:

  • Have the distribution channel footprint to get your drug to the market quickly
  • Have the market insights and resources to reduce the risk of future supply issues
  • Have the flexibility to grow with you as your product matures

Orphan drugs offer hope to patients suffering from rare diseases or medical conditions. Researchers at your biopharma company gave them that hope. Now it’s time for you to deliver on that promise with a successful commercialization strategy backed by an effective drug distribution model.

Related: Learn more about McKesson’s third-party logistics (3PL) services for drug manufacturers

1 “Insights into Rare Disease Drug Approval: Trends and Recent Developments.” FDA, 2017

 

ABOUT THE AUTHOR

Kevin Kissling is Vice President, 3PL Services for McKesson Specialty Health.  In this role, he leads a team that is focused on growing our manufacturer relationships by providing comprehensive 3PL services and commercialization strategy guidance to our manufacturer partners.  Kevin joined McKesson in 2004 and has held a number of different roles in both the Specialty and Corporate divisions of the company.  Before joining McKesson, Kevin worked in various financial and IT capacities in the consulting, retail and agricultural industries.  He holds a bachelor’s degree in business economics from the University of Delaware.

About the Blog Poster: Dan Limbach

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