eBook

Bringing your first biopharmaceutical to market

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BRINGING YOUR FIRST BIOPHARMACEUTICAL TO MARKET

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In today’s competitive landscape, the successful launch of a bio-pharmaceutical involves a complex web of changing dynamics across R&D, regulatory, market access and marketing and sales.

An analysis of the market shows a growing number of small and emerging companies are choosing to launch drugs on their own, rather than sell or partner. A recent study from IQVIA indicates biopharma companies with less than $500 million in annual revenue launched 42% of all new drugs in 2018, up 26% from the prior year — and, because these com-panies account for 73% of late-stage clinical research, more product launches from smaller companies are expected over the next few years.

Yet, for companies launching their first biopharma product, the pressure to deliver a successful product launch is extremely high. Nearly 66% of all drug launches fail to meet pre-launch sales expectations, according to a study conducted by McK-insey. With both credibility and funding on the line, the failure to successfully launch a first product could put the viability of the entire company at risk.

With so much at stake, it is important to approach the launch of a first product with a holistic view that covers the entire lifecycle of a product — from discovery, to development and commercialization, all the way through to patient adherence.

It’s never too early to think about where the new launch fits into the commercial landscape. Ideally, there is a confluence of activity happening simultaneously as the product moves through the approval process — especially as the clinical trial moves to Phase III.

“Not only do clinical trials need to be successful,” says Joe DePinto, president of Cardinal Health Specialty Solutions, “but commercialization needs to be successful, especially during those first seven-to-eight quarters, which are so critical to set up the trajectory for the success of the brand.”

The landscape is fraught with roadblocks and uncertainty. Competition is stiff. The regulatory environment is rapidly evolving. Regulators, payers, physicians and patients all de-mand demonstrated value, defined by both cost and outcomes. Patients require increasing support, adherence rates remain notoriously low and, as therapies become more targeted, reaching small populations of providers and patients becomes more challenging.

In this eBook, we’ll explore steps that biopharma companies can take when launching a first product to reduce their risks, avoid pitfalls and increase their chances for success across the entire biopharmaceutical lifecycle.

Reducing risk

THE TARGET PRODUCT PROFILE4

CHANNEL MANAGEMENT AND LOGISTICS

PATIENT/MARKET ACCESS AND SUPPORT

COMMERCIALIZATION& CHOOSING A LAUNCH PARTNER

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THE FDA APPROVAL PROCESS3

CONTENTSJOANN WHITCHER Senior producer,Haymarket Studio

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Here are several recommendations to help streamline the approval process:

• Consult with the FDA early in the process and throughout the application journey, advises Phillips. Not only does the FDA appreciate this effort, but this can also help ensure the application contains all the required information in the appro-priate language.

• Get FDA agreement early on as to exactly what the indication is — as that’s what will drive labeling language, especially if related to disease, treatment or metrics.

• Take advantage of all appropriate regulatory accelerated pathways, such as Fast Track and Breakthrough Therapy.

• Determine how to collect the data needed for approval in the most judicious way. This is particularly critical if you are pursuing an expedited pathway.

• Provide responses to FDA requests in a timely manner.

• Make sure the approval process does not outpace the manufacturing of the prod-uct; the FDA wants to know that once the product is approved, it will be commercially released in a reasonable amount of time.

Phillips also advises that regulators are in-creasingly looking for data based on real-world evidence, informed by patient-reported value- based outcomes describing the impact of drugs on quality of life, daily activities and symptoms.

This data is of interest to both regulators and payers. For regulators, real-world data is used to confirm claims on the drug’s safety and efficacy. Once the product is commercialized, payers will use that post-approval data to de-termine reimbursement.

The FDA is increasingly focused on patient experience data, not just in approvals and label expansions, but for risk benefit assessments and for putting other items in context, explains Phillips. The idea behind the initiative is that patients affected by an illness can — and should — be involved in the FDA’s decisions about the benefits and risks of any drug meant to help them.

To that end, clinical trial data will be sup-plemented with input from patients, submitted by drug companies, caregivers, research teams and others.

When developing a first prescription drug product, it often feels as if the future of the company hinges on FDA approval. Companies seeking approval for the first time must thoroughly understand the five-step process they must complete: discovery/concept, pre-clinical research, clinical research, FDA review and FDA post-market safety monitoring.

“For smaller or newly launched pharma manufacturers, the process of filing for FDA approval can be a minefield that must be carefully traversed to avoid delays to the release of the therapy,” says Todd Phillips, director of regulatory affairs with Cardinal Health Regulatory Sciences. “The regulatory approval process, and what the FDA is looking for, continues to evolve, so it’s important to work with experienced partners who can guide you to the fastest path to approval.”

The FDA approval process

“For smaller or newly launched pharma manufacturers, the process of filing for FDA approval can be a minefield that must be carefully traversed to avoid delays to the release of the therapy.”

TODD PHILLIPS CARDINAL HEALTH REGULATORY SCIENCES

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To ensure a successful launch, there are critical elements that need to happen concurrently with the approval process, especially as the clinical data progresses from Phase 2 to Phase 3.

One such element is the Target Product Profile (TPP). The TPP plays a key role throughout the drug discovery and development process, helping to define the characteristics of the new product and its therapeutic area, patient population, standards for safety and clinical efficacy, design of clinical research strategies and constructive communication with regu-latory authorities.

“With the launch of a new biopharmaceutical, there needs to be alignment among all the different stakeholders involved in the launch, including the clinical, regulatory, market access and commercial teams,” says Eli Phillips Jr., Cardinal Health VP for regulatory sciences and insights & engagement. A well-defined TPP will help ensure that alignment.

As clinical and drug development teams partner with commercial coun-terparts, the TPP acts as a reference point to what the key differentiators will be — both therapeutic and within the competitive landscape.

The TPP document is where “everything comes together,” explains Joe DePinto. “As the clinical data progresses, the TPP starts to tease out the key details related to efficacy, safety and value, and then a value proposition is born along with the key differentiators.”

DePinto adds the TPP will ultimately serve as the foundation for com-mercial planning and market positioning.

The target product profile

“With the launch of a new biopharmaceutical, there needs to be alignment among all the different stakeholders involved in the launch, including the clinical, regulatory, market access and commercial teams.”

ELI PHILLIPS JR. CARDINAL HEALTH REGULATORY SCIENCES AND INSIGHTS & ENGAGEMENT

solutions.

So all you see is success.We know that what matters most is a seamless treatment journey for patients. That’s why we’re here, working behind the scenes to help bring your specialty product to market. At Cardinal Health Specialty Solutions, we are an expert team of regulatory consultants, real-world researchers, third-party logistics leaders, and patient support specialists, helping you break through barriers to care.

To see how we can help, go to:cardinalhealth.com/allwesee

© 2019 Cardinal Health. All Rights Reserved. CARDINAL HEALTH, the Cardinal Health LOGO and ESSENTIAL TO CARE are trademarks of Cardinal Health and may be registered in the US and/or in other countries. Lit. No. 1SS19-982314 (7/2019)

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Pharma companies may breathe a sigh of relief after clearing all the FDA hurdles, but other challenges can delay a product launch — particularly when it comes to channel planning.

Channel planning encompasses both big picture strategizing focused on commercial goals, as well as the detailed logistics necessary during fulfillment and distribution to ensure that healthcare providers and pa-tients can access the product as promised.

Channel planning considers the product’s therapeutic area; which patient populations are being targeted; the type of payer coverage (reimbursement) being sought; and logistic requirements, all the way through whether or not the product needs refrigeration and a pack-out. It considers whether the pharma company will distribute the product through the specialty channel, through broader, wholesaler distribution or through a more limited network, direct to specialty pharmacies and providers.

“It drives the model for the pharma company’s business,” says Shantese Marchbanks, director of account management and business implemen-tations at Cardinal Health 3PL Services.

AVOIDING COSTLY DELAYSTo avoid delays, each element within channel planning requires an expert’s understanding of the process. For example, nationwide distribution of a drug product layers complexity upon complexity. At the federal level, drug distribution falls under FDA guidelines, but more than 40 states require pharmaceutical manufacturers to acquire a license to distribute a prescrip-tion drug product within their state, explains Marchbanks.

Additionally, in several states the application process cannot begin until after the product is approved by the FDA. The time required to acquire all the necessary licenses could take anywhere from three to nine months, delaying the launch and negatively impacting revenue and market share.

Channel managementand logistics

Adding to the challenge is that each state has its own licensing requirements, many of which are continually evolving.

“Pharma companies entering the market for the first time often haven’t given thought to, or built in, adequate planning for things like licensures,” says Marchbanks.

The Cardinal Health 3PL Title Model exclusive distribution solution mitigates this problem by taking the title of the product and handling all warehousing, distribution and order-to-cash management on behalf of the manufacturer until the manufacturer can get its own license, says Marchbanks. This allows the manufacturer to leverage the existing state licenses used by Cardinal Health. The 3PL Title Model enables Cardinal Health to take owner-ship of the product, as well as the financial risk on the back-end, so the pharma company can stay focused on the product launch.

At its core, the Title Model is very similar to a traditional 3PL distribution model. In both, the 3PL provider partners with the manufacturer to distribute to their customers, which can include physician offices, hospitals, specialty pharmacies, specialty distributors and wholesalers. The 3PL provider handles all aspects of order-to-cash management, and manages the distribution and storage of all the pharma company’s products, eliminating the cost of brick and mortar and having to hire a lot of back operations personnel.

“Our case studies have demonstrated that leveraging the Title Model gets the product to market five months faster than if they did it on their own,” says Marchbanks. “This makes a big difference at a launch when every moment is precious.”

Marchbanks adds that Title Model can also be an effective tool when launching cell and gene therapies, which may have a unique supply chain and more complex order-to-cash requirements.

“Pharma companies entering the market for the first time often haven’t given thought to, or built in, adequate planning for things like licensures.”

SHANTESE MARCHBANKS CARDINAL HEALTH 3PL SERVICES

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The success of a biopharma launch is directly dependent on patients having access to the new therapy. In the rare and orphan drug space, where the patient population is small, helping facilitate access is even more critical.

Unfortunately, barriers to access are often part of the patient journey — particularly for specialty products, which often require a benefits investi-gation and prior authorization before a payer will approve reimbursement. Navigating these access hurdles can be overwhelming for patients, which is why patient support services and access programs need to be primed and ready when a launch is imminent.

Some pharma companies manage patient access and support in-house, but more and more companies are turning to external hub providers to manage all or part of these services, because outsourcing enables them to add patient support expertise without building significant infrastructure. Also, from the compliance perspective, it may be helpful to have the patient support team separate from the commercial team.

Hub support programs are designed to help remove the barriers standing in the way of patients accessing and staying on therapy, says Josh Marsh, program director at Cardinal Health Sonexus Access & Patient Support.

“There are three pillars to patient support services: reimbursement, financial assistance and adherence support,’” says Marsh. Within each pillar are a multitude of programs that support the patient. How, or if, the manufacturer — or its hub partner — ramps up these services depends on the profile of the product and the needs of the patient.

If there are significant access barriers for a product, for example, the Sonexus team can help patients overcome hurdles by assisting with prior authorizations and appeals. Under the financial assistance pillar, the team can identify resources, assistance programs and foundation support to help patients afford their medication. The Sonexus team can

Patient/market access and support

also manage free drug distribution to eligible patients through its non-commercial specialty pharmacy, which the OIG has stated reduces certain compliance risks.

DRIVING ADHERENCE & COMPLIANCETo drive adherence and compliance, the Son-exus team can evaluate patient demographics and segmentation to design a tailored program to support patients during their treatment and help keep them on therapy, explains Cathy Pat-ten, director of clinical operations at Sonexus. For some patients, a phone call might be the best way to connect, but others may prefer a text message, Patten explains. Frequency of outreach can be tailored as well.

Particularly within the rare disease and oncology environment, there are more touch-points to ensure adherence and compliance, adds Patten. “Patient contact is required at nearly every step of the journey. More and more, we are getting involved at the point of diagnosis, when the patient is filling out their enrollment form to start on the therapy. Our role is to not only eliminate the barriers, but to make it a good experience for the patient.”

To further build engagement, Sonexus has started using artificial intelligence and natural language processing to proactively identify patient sentiment on patient support calls focused on adherence or compliance.

“The technology will alert us if a patient seems frustrated or if they complain about financial toxicity,” says Marsh. “Then, we share the data about the pain points with the manufacturer — enabling them to deepen their understanding of the patient journey. The data is rich, as we are talking to tens of thousands of patients monthly.”

Data and the accompanying insights are an integral part of what the Sonexus team deliv-ers to the manufacturer. Data from patient- reported outcomes helps the manufacturer demonstrate the drug’s efficacy to the payer, to remove reimbursement barriers and help with getting on formulary. The robust data gathered from electronics benefits verifica-tion and prior authorization provides deeper insights into the patient journey, helping to increase engagement and boost adherence and compliance.

When it comes to launching a new product, Patten recommends developing the patient strategy early and engaging a patient hub well before FDA approval. “It’s never too soon to start planning how you will meet patient needs. Having a well-defined program to help patients get access to therapy and to support their needs throughout treatment can be critical to driving product adoption.”

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All of the previously discussed elements are key for a suc-cessful commercialization of a launch: a solid target product profile, a sharply defined channel planning strategy and far-reaching patient support services and market access hub programs.

“It’s critical that all of these components mesh together in a story to demonstrate the value and benefit to patients, healthcare providers, payers and health systems,” says Joe DePinto.

Additionally, you have to be able to differentiate your product in at least one of these three areas: quality, cost and/or access, says Eli Phillips Jr.

Commercialization of a new product will become even more complex as targeted and precision therapies become more available.

Targeted therapies may deliver improved outcomes or better quality of life, but their efficacy may be limited to smaller patient populations, says DePinto. “It’s important that the commercial team understands that these types of outcomes are the levers that you can pull to optimize your brand,” he adds.

Messaging and engagement solutions directed to the physician will have to adapt to these smaller patient pop-ulations. Broad, blast marketing campaigns will no longer be effective, says Phillips. Marketing will need to take on the role as educator and help instruct healthcare providers as to how the therapy impacts care and how to determine if a patient is a good fit.

Precision medicine requires a precision campaign, en-riched by data, which will drive the channels used to market the product. “We recommend selecting the channel based on how the HCP likes to receive information, to drive the most impact,” says Phillips. “Tailoring the content to the audience is important, too. Marketers will need to rely on data and insights about prescribers to ensure the message is relevant.”

Commercial teams will have to understand the overall marketplace, as well as the target audience for the bio-pharmaceutical in order to deliver a focused and consistent message. External partners may be able to support these efforts by providing expertise in reaching key prescribers and influencers.

Given today’s dynamic healthcare landscape and the significant investments that have been poured into developing new therapies, it’s critical that pharma companies engage the right partner or partners to support their launch success. Below are some key considerations when evaluating potential partners:

• Therapeutic category knowledge. Does the partner know your disease area and have they worked other products like yours? Relevant prior experience will help to reduce ramp-up time.

• Expertise in managing complex launches. No two product launches are exactly alike, and some have unique complexities. If you are a launching an orphan product, a cell and gene therapy or a drug with a novel mechanism of action, you may face different challenges, or need to make different choices, on your path to commercialization. A partner with experi-ence in complex launches can help you identify opportunities and avoid pitfalls along the way.

• Ability to collaborate across functional ar-eas. For pharma companies launching their first biopharma product, managing many different suppliers can result in administrative overload. Identifying one partner that can support your launch over many functional areas may not only save you time and money, it may lead to a more streamlined commercial approach.

This is an exciting time for emerging biotech com-panies, says Joe DePinto. “Clinical advances are changing the landscape of healthcare, and emerging biotechs are at the center of many of those break-throughs. At Cardinal Health Specialty Solutions, we are committed to working with manufacturers to ensure that their products not only get to market, but are positioned for success with optimal regulatory, logistics, commercial and patient support strategies.”

Commercialization Choosing a launch partner

“At Cardinal Health Specialty Solutions, we are committed to working with manufacturers to ensure that their products not only get to market, but are positioned for success with optimal regulatory, logistics, commercial and patient support strategies.”

JOE DEPINTO CARDINAL HEALTH SPECIALTY SOLUTIONS