McKesson : How Biopharmaceutical Manufacturers Can Commercialize New Gene Therapies

There's that moment when a biopharmaceutical manufacturer looks at the results of its clinical trial and says, 'We did it.' Researchers at your biopharma company created a new gene therapy. It's a new drug that will help patients with a hard-to-treat medical condition.

But now what? How does your company successfully commercialize your new gene therapy to benefit patients with an orphan disease or rare cancer? Our clinical trial commercialization experts say the answer is an action plan featuring high-touch and high-tech strategies.

How gene therapy works

What makes commercializing a new gene therapy difficult is the drug itself. Each therapy is a personalized medication. The FDAⁱ says gene therapy can work one of three ways:

Gene therapies can create commercialization challenges for your biopharma company. The challenges include:

• Executing complex logistical processes
• Maintaining the quality and integrity of your product
• Minimizing the risk of adverse events
• Ensuring patient access to your high-cost drug
• Helping other stakeholders with financial risk
• Enhancing the experience of your customers
• Collecting data to improve clinical outcomes
• Accessing claims and outcomes data for value-based reimbursement

Your company can overcome each challenge with high-touch services backed by state-of-the art technology. Here are the four steps that should be in your action plan:

1. Rethink distribution for these unique drugs

Logistics come first. Your company's normal distribution channels won't work. Your company needs to rethink distribution to avoid treatment delays and give the best care to patients who need your drug most. The following items should be on your distribution checklist:

• Temperature during transportation and storage
• Storage requirements during the drug's lifecycle
• Drug variability from thawing to use
• Drug education for providers and patients

Your biopharma company should tailor distribution to meet the needs of each gene therapy. One choice may be a 'just-in-time' approach that overnights delivery for next-day use by patients and providers.

An online ordering platform and inventory management tools back just-in-time distribution. It's one example of how your company can use technology to replace manual tasks with electronic transactions.

2. Use an electronic service hub for patient access

Your company can use technology to perform other tasks critical to the success of your new gene therapy:

• Automating prior authorization
• Educating patients, payers and providers
• Evaluating and minimizing risk
• Gathering and analyzing outcomes data
• Monitoring patient adherence to your new drug
• Offering copay and financial aid to patients
• Giving adherence support to patients
• Reporting adverse events
• Streamlining claims processing

The use of technology is the first step biopharmaceutical manufacturers can take to create an electronic service hub that improves efficiency, reduces costs and eases patient access to new gene therapies.

3. Support physician evangelists

Let's focus on some of the tasks ahead for your biopharma company as you commercialize your new gene therapy.

One task is to educate physicians whose patients will benefit most from the new therapy. Your company already has a group of doctors who had access to the drug during clinical trials. They are familiar with the drug and its benefits. They can drive faster adoption of your new drug after the FDA approves it.

Your company can help these physicians expand the use of your new drug by providing educational and administrative support to them and financial assistance to their patients. The physicians become champions of your new gene therapy and thought leaders with their peers. With the right high-touch services, they can drive awareness of your company's new drug across both pre- and post-commercialization stages.

4. Gather real-world evidence

Another task critical to your biopharma company's commercialization success is gathering and analyzing outcomes data. Your company knows how your new gene therapy performed during clinical trials. But do you know how it will perform once it goes to market? That's where real-world evidence (RWE) comes in.

RWE is data that tells your company how prescribers and patients are using your new gene therapy after it hits the market. RWE also tells your company how well your new gene therapy is performing clinically. The way to gather RWE is through Health Economics and Outcomes Research (HEOR). Your company can do HEOR in-house, or can choose to use an outside vendor.

HEOR can be done in two ways. The first is through a retrospective analysis of prescriber and patient information from a database like a disease registry. The second is through a prospective analysis of medical records or an observational study.

When you use HEOR to gather REW, your biopharma company can:

• Confirm the immediate effectiveness of your new gene therapy
• Confirm the long-term effectiveness of your new gene therapy
• Spot any long-term medication safety issues
• Find clinical factors that could affect the drug's effectiveness
• Find other patients who could receive help from the new drug
• Justify the cost of the new drug to payers
• Find and address any adherence issues, including cost to patients
• Support reimbursement terms in value-based contracts
• Use the information to improve future clinical trials

RWE also can help your company win approval of future gene therapies to help other patients with rare diseases. RWE can answer questions about the evidence, value and affordability of your new drug.

By incorporating high-tech capabilities and high-touch services in your action plan, your biopharmaceutical company can successfully commercialize your new gene therapy.

Related: Learn more about McKesson's specialty drug distribution services for pharmaceutical manufacturers