American Pharmacy Review: Five Strategies for Bringing Innovative Therapies to Market

So-called Advanced Therapeutic Medicines (ATMPs) in the EU and UK, or so-called Cell or Gene Therapy Products (CGTPs) in the US, have long been considered game-changers in the treatment of serious diseases
.

The market for such therapies is expected to be worth nearly $21.

While innovative treatments hold great potential, they employ a model that differs markedly from traditional development models - and requires a tailored approach
.

For example, conventional clinical development models often simply do not work for ATMP developers

To address and overcome possible challenges, companies need to: (1) plan early to build bridges between quality (CMC; chemistry, manufacturing and controls), non-clinical and clinical disciplines; (2) develop a regulatory strategy from the very beginning of drug development (3) Analyze the healthcare landscape and have identified market access models that will provide strong value to policymakers and payers
.

Overall, the goal should be to establish an agile planning approach that minimizes the risk of delays or failures

Five strategies to reduce risk and accelerate development

A strong strategy is rooted in proper planning
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This requires an integrated approach that combines therapeutic, non-clinical and clinical disciplines from day one

For advanced therapies, there are many complexities to consider in the commercialization process
.

Patient populations are generally smaller and more targeted, and while this means that the number of products may be small, there are also very specific needs

While advanced therapies can be potentially transformative, pricing may be prohibitive for some patients
.

At the same time, basic quality, regulatory and manufacturing guidelines that apply to traditional drug development must still be considered - these guidelines can vary from country to country, which can make them difficult to navigate

1.
Conduct a risk/benefit assessment
.

1.
Conduct a risk/benefit assessment
.

New treatments have great potential to offer important treatment options, perhaps even curative, but also come with significant known and unknown risks, many of which are unique to the class
.

Therefore, risks also need to be considered from an early stage - the main focus is not only on protecting patients, but also on minimising risk to healthcare professionals and caregivers

2.
Develop an Integrated Product Development Plan (IPDP)

2.
Develop an Integrated Product Development Plan (IPDP)

For a holistic approach to creating an IPDP, all development disciplines such as manufacturing, non-clinical and clinical development, and regulatory affairs need to be involved
.

Even early-stage projects should consider commercial aspects such as country-specific commercialization, the competitive environment, and pricing/reimbursement
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The IPDP is a dynamic document that will be continuously updated as development progresses, facilitating organizational prioritization and reducing decision-making time
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For example: Defining patient populations and disease-specific target stages are important considerations that may have implications for the design of nonclinical studies,
etc.

3.
Models that consider mass production

3.
Models that consider mass production

The path to commercialization should be considered from day one
.
Moving therapies from the lab to scale-up for patient administration means producing enough sterile medicines can be challenging
.
To ensure scalability without burning money, companies must align manufacturing readiness with regulatory pathways, patient populations and the doses being pursued
.

4.
Accelerate commercialization through effective regulatory strategies

4.
Accelerate commercialization through effective regulatory strategies

There are several different aspects of the regulatory program -- all of which happen in parallel -- that should evolve as development progresses
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These include: (1) documenting targets, which can be visualized through target product profiles; (2) keeping pace with competing therapies; (3) maintaining regular reviews with regulators; (4) by market or region, indication area, and treatment Classification considers regulatory pathways
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Regulatory strategies should evolve as they evolve and change as new information becomes available about the competitive environment, research findings, and progress in interactions with regulators
.

5.
Initiate a market entry strategy

5.
Initiate a market entry strategy

To gain market access, developers must be able to present clinical and economic evidence to providers, healthcare decision makers, and key patients
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Given the complexity of how health care is paid, it is critical to understand who will fund treatment and how medical reimbursement will work
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Developers must consider strategies that take a holistic view of patient care and provide better real-world evidence, resulting in a stronger value proposition for policymakers
.

don't cut corners

Jumping from research to development without a full product development plan is a risky move
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Companies must go through the planning process with the understanding that this is just a starting point and that the plan will be adjusted as the science develops
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What's more, with structured planning up front -- even when acknowledging that things will change -- companies will avoid bumpy roads and move faster on the road toward commercialization
.
Developers of innovative therapies may be breaking new ground, but with proper strategic planning, the strategic horizons can be limitless
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