Pharmaceutical Product Launch

Stuart R. Gallant, MD, PhD

Above is a photo from the 1947 launch of the M.V. St. Essylt at J.L. Thompson and Sons was a shipyard at Sunderland in the north of England. She had a gracefully raked funnel and first-class accommodations for 12 passengers in addition to her crew and cargo. Judging from the rapt attention of the crowd, it must have been a majestic sight to see her slide down the ramp into the River Wear.

In today’s post, we are going to discuss product launches. Deloitte has a nice report crunching the numbers on a range of pharmaceutical product launches [1]. It’s well worth the read, and I am not going to try to summarize the entire white paper here. Two facts jumped out at me when I read the report: 1) a third of the drugs analyzed (36%) failed to meet market expectation at launch and 2) drugs launched by large companies tended to be the underperformers. So, it’s not a question of resources. If it was, small companies would be the underperforming class. It’s a question of execution, and any company should be able to execute, provide that it has the right culture.

The Basics: 3 Ps of Product Launch

There are three key elements (“three Ps”) to a successful pharmaceutical product launch. The first is the Product. In a previous post, PharmaTopo used this illustration [2]:

The point of that post was that medications can be valued in different ways—from a healthcare economic point of view, from a scientific point of view, and from the point of view of the patient. As the development team is designing the product, they need to keep each of these spheres in mind, but particularly the sphere of the patient. The development team’s goal is embodied in the Target Product Profile (TPP)—the first pharmaceutical development document [3]. If the team describes a product which provides real value to its patients, the project begins on a solid footing.

The second P is the Patient. The patient travels a journey that it is important to understand:

The patient typically starts outside of the medical care system, unless they are for example an ill person who is already in the hospital and under physician care. So, something has to motivate them to seek care. Once they have sought care from a physician or an urgent care clinic, what is that interaction like for them? How does the diagnostic process operate for their illness? Are their preferred methods of diagnosis, such as analytical tests or recognized diagnostic standards? Once diagnosed, the patients enter treatment, and a brand choice is made. What treatment guidelines exist? How is the choice of medication made? And, once the brand is chosen, who provides the drug to the patient?

At each stage, many results are possible, and the drug development team has to understand entire tree of the possibilities in order to forecast (and maximize) the market for the team’s drug.

The third P is the Payer. Around the world, countries have different methods of paying for medical care. To be included in formularies and compensated correctly, a pharmaceutical development team needs to understand the perspective of the payer. How are drugs regulated? What is required to be approved for sale in a certain region? How are medications justified? What are the best ways to represent the advantages of a particular treatment to the payer.

An Example: BioMarin Launch of Roctavian

With a market cap of 16B USD, about 3,000 employees, and 7 licensed treatments, BioMarin is a California-headquartered biotechnology company. Two things make BioMarin unique:

BioMarin has focused on orphan diseases, illnesses that have small patient populations and which prior to BioMarin’s work had no licensed medical treatment.
Since its founding, BioMarin has remained a self-standing pharmaceutical company in an industry which is almost defined by its tendency to merge and consolidate.

The second of these traits is linked to BioMarin’s history of successful product launches. Shareholders recognize that BioMarin offers a significant value for their investment, and as a result, BioMarin has not felt pressure to merge. (BioMarin has however done 12 early-stage partnerships to enrich its development pipeline.) BioMarin forecasts continued growth in its pipeline and revenue, expecting to be at 4B USB to 5B USD in revenue by 2025.

BioMarin is looking toward a late 2022 launch of its product Roctavian, a gene therapy for hemophilia A. Hemophilia A is an inherited disease, affecting almost exclusively boys and men because of its sex-linked genetics. Patients with hemophilia A depend on regular infusions of Factor VIII to prevent uncontrolled bleeding. PharmaTopo recently did a post on Factor VIII therapy [4]. Even with optimal therapy, patients can expect to have several painful bleeding episodes per year.

Roctavian is a virus (an adeno-associated virus 5) that has been engineered to contain the DNA for a small active portion of the human Factor VIII gene, combined with a promoter gene. After delivery of Roctavian, the DNA is taken into the patient’s liver cells and for a period of about 6 years, the patient’s liver releases functional Factor VIII into the patient’s blood, ensuring hemostasis.

There are about 150,000 hemophilia A patients in markets that BioMarin believes it can reach, and of those, about 60,000 have severe hemophilia A and would benefit most strongly from BioMarin’s gene therapy approach. Let’s take a look at what BioMarin has done so far to set up Roctavian for success:

Product: BioMarin has provided several advantages and benefits to patients.
- Because hemophilia A patients depend on Factor VIII to prevent bleeding, they need to have access to Factor VIII, receiving an infusion once every 3 days. Patients receiving Roctavian could forgo 98% of their infusions while maintaining hemostasis, based on Phase 3 clinical data. In addition, to removing the anxiety of having access to Factor VIII, Roctavian removed the discomfort and inconvenience of the infusions—all big wins for the patients
- Even with optimal Factor VIII treatment, hemophilia A patients have about 5 bleeding episodes a year. These can include painful bleeding and swelling of affected major joints such as the knee. With Roctavian, bleeding episodes were reduced by 85%.
- And, this is a small thing, but important: Here at PharmaTopo, the advantages of strong, distinctive trade names are appreciated. Roctavian is a great brand name, and the BioMarin team did an excellent job selecting the name and getting it approved.
Patient:
- Patient organizations are extremely important in today’s pharmaceutical development environment. They can act as cheerleaders of products they like. They can facilitate patient enrollment in trials they support. And, they interact frequently with researchers and clinicians, sometimes steering research funding to favored programs. BioMarin was founded based on these types of interactions and received its startup funding in part from families of lysosomal storage disease patients. So, BioMarin is an ideal organization to partner with hemophilia A families.
- Because BioMarin has experience with orphan diseases, their team is very good at locating and getting treatment for every eligible patient. This is an invaluable skill in launch of a new and innovative treatment such as Roctavian.
Payer: Roctavian will be an expensive treatment for payers. BioMarin has several aces up its sleeve to make the expense more palatable.
- BioMarin understands the economics of treating Factor VIII deficiency. Factor VIII treatment can cost upwards of $100,000 per year before insurance coverage is factored in. BioMarin plans to price their treatment, which provides 6 years of Factor VIII expression from the patient’s liver before the effect starts to wane, as a superior treatment and worth a premium price. They will offer a guarantee that if their treatment does not function for the full 6 years in a given patient, they will provide a prorated refund.
- BioMarin has historically insulated itself from some of the bad press around drug pricing by pricing its treatments at the same level in the US and in a basket of similar economies around the world. This pricing strategy prevents unfavorable stories in the media comparing the cost of treatment on BioMarin drugs in the US versus the EU, for example. Presumably, it will continue this policy with Roctavian.

With these and other plans and actions, BioMarin hopes to have another successful product launch with Roctavian.

Team

A critical aspect of a successful product launch is a high functioning team. At the start of this post, Deloitte’s observation that large companies tend to underperform in product launches was mentioned. Large companies have the tendency to become siloed. Most people can only function with a certain number of close work relationships, so workers tend to divide the world into “my team” and “over there.”

The solution to this problem is good project management and a strong project team culture. It may not be a surprise then that BioMarin has a reputation for excellent project management. This may be the reason that BioMarin has continued to have successful product launches as it has grown in size.

Successful project management is a complex topic that may be the subject of a future PharmTopo post, but one idea is critical:

To have a successful team, the team members must perceive that they are responsible primarily to each other and to the project—rather than to their line managers who they report to as part of the organization’s structure.

All excellent project management starts from that premise. With that said, here are some thoughts on the members of the launch team:

CMC: CMC (i.e., Manufacturing and Supply) is involved in the project from the start and plays a significant role in, for example, the Target Product Profile. CMC is usually rate limiting on the approach to Phase 1, but as Phase 3 and launch approach, CMC is often out of the critical path. Clear communication of drug product demand is very important. And, CMC needs to always have a backup plan if demand exceeds projections.
Quality: The Quality function ensures that your clinical and manufacturing operations are fully compliance with US, EU, and ROW regulations. This function works closely with CMC.
Legal: Legal counsel is involved in the project from the beginning. PharmaTopo recently posted on patent strategy [5]. Without intellectual property, there is no product.
Regulatory: Regulatory plays a key role in the product label. Without a label, there is no product. The company’s sales team can only make representations about what appears on the label.
Marketing: Marketing bridges the gap between the label and the sales team. The sales team needs a story that they can tell about the product. The marketing team works with patients, doctors, insurers, and others to define what that story will be. Marketing also establishes the metrics by which a launch will be a “success.” Their marketing plan needs to be aggressive (so that the sales team is pushed to maximize revenue) but realistic.
Medical Affairs: Medical Affairs (MA) is a specialty pioneered in the United States as an interface between doctors, researchers, patient organizations and other organization on the one side and the pharmaceutical company on the other. By sponsoring and facilitating research on the drug candidate, MA plays a key role in helping to understand how to use this new medication.
Market Access: Market access has to do with the vagaries of pharmaceutical regulation and reimbursement across all the countries that you plan to sell your product. Particularly, for orphan drugs, market access also involves locating each and every patient possible. This may be a significant challenge and may drive the decision of which countries to launch into first. For example, are there genetic traits that make the incidence of the disease higher in Brazil or in Italy. Reaching those countries first may be important to a successful drug product launch.
Commercial Operations: Modeling the demand and supply of the company’s drug to ensure a smooth functioning supply chain is incredibly important. Ideally, there should be no waste in the supply chain, but that can be a challenge. Worse than waste is inability to provide the medication to a patient who needs it. Commercial operations walks a tight rope.

An excellent, well-functioning, team organized according to the principles of project management allows the drug product to achieve its maximum during commercial launch.

[1] Ford, J. et al. Key Factors to Improve Drug Launches, Deloitte (2020).

[2] pharmatopo.com/index.php/2022/01/07/the-value-of-medications-1-of-2/

[3] pharmatopo.com/index.php/2021/12/31/drug-development-plan/

[4] pharmatopo.com/index.php/2022/03/22/pharmaceutical-design-focus-factor-viii-1-of-2/

[5] pharmatopo.com/index.php/2022/09/20/pharmaceutical-patent-strategy/

Disclaimer: PharmaTopo^TM provides commentary on topics related to drugs. The content on this website does not constitute technical, medical, legal, or financial advice. Consult an appropriately skilled professional, such as an engineer, doctor, lawyer, or investment counselor, prior to undertaking any action related to the topics discussed on PharmaTopo.com.