Tailored, multidisciplinary strategies for marketing these advanced treatments must be implemented as early as possible in the development process

By Christian K Schneider

Nevertheless, there are challenges as well as opportunities within the sector, particularly when it comes to commercialisation of products. Innovative therapies employ a model that differs considerably from conventional development paradigms – one which requires a more tailored approach. At heart, the ‘traditional’ clinical development paradigm is often not applicable because ATMPs deal with smaller patient populations; they have special requirements for manufacturing, where patients’ lives can depend on the speed in which a therapy can be made available, and they offer pricing models that payers may see as prohibitive.

Companies need to do three things to plan for – and mitigate – the risks along the way:

• Plan early, building bridges between quality (chemistry, manufacturing and controls), non-clinical and clinical disciplines
• Develop a regulatory strategy as soon as drug development begins
• Analyse the healthcare landscape to determine a market access model that appeals to decision makers and payers

With advanced therapies, there are many complexities to consider in the commercialisation process. The smaller and more targeted patient populations typical of this sector also have specific logistical requirements. For instance, manufacturing considerations and patients’ lives can depend on the speed at which a product moves from the bedside to the facility and back again, especially if shelf life is short.

Advanced therapies may be potentially transformative, but pricing for them could ultimately put off some payers. Meanwhile, the underlying quality, regulatory and manufacturing guidelines that apply to traditional drug development must still be considered. Companies should therefore:

1. Undertake a risk/benefit assessment: Advanced therapies offer considerable benefits, but they also come with risks, many of which are unique to this product class. Therefore, risk needs to be considered from an early stage, for patients as well as for healthcare professionals and caregivers. The risk/benefit assessment should be designed as a gate to go/no-go decisions at each stage of development. Sometimes, the ‘go’ will require a change in direction, so the process should be agile, with an eye towards risk identification, evaluation and mitigation. That agile approach should apply not only to the biological activity of the ATMP, but also to its quality attributes, manufacturing process steps and therapeutic administration procedures.
2. Develop an Integrated Product Development Plan (IPDP): All development disciplines, such as manufacturing, non-clinical and clinical development, as well as regulatory affairs, need to be involved. Even for early-stage programmes, commercial aspects, such as targeting specific countries for commercialisation, the competitive environment and pricing/reimbursement aspects should all be considered. The IPDP will be updated as development progresses, promoting organisational prioritisation and speeding decision-making. For example, defining the patient population and the target stage for a given disease could have an impact on the design of non-clinical studies.
3. Consider models to scale manufacturing: It can be a challenge to move a therapy from the lab to scaling it for supply to patients and producing a sterile drug product in sufficient quantities. To ensure scalability without needless expense, organisations need to align manufacturing readiness with the regulatory pathway, the patient population and the planned dosing regimen.
4. Accelerate commercialisation with an effective regulatory strategy: There are distinct aspects to the regulatory plan – all of which take place in parallel – which should evolve as development progresses: 1) documenting the goal, which can be visualised via the Target Product Profile (TPP); 2) keeping pace with competitive therapies; 3) maintaining regular checkpoints with regulatory agencies; and 4) considering regulatory pathways, depending on markets or regions, indication areas and classification of the therapy. The regulatory strategy should evolve alongside development and as new information arrives regarding the competitive environment, study results and interactions with regulatory agencies.
5. Initiate a market access strategy: For developers to gain market access, they must be able to demonstrate clinical and economic evidence to providers, healthcare decision-makers and payers. Given the complexities of healthcare payment systems, it is important to understand who will finance the therapy and how care will be reimbursed. Developers must consider strategies that offer a strong value proposition for decision makers, ideally at the proof-of-concept phase, so that later considerations on risk-benefit and cost-benefit converge.

Rushing from research to development without an integrated product development plan is risky. Organisations must go through the planning process with the understanding that this will just be a starting point and that the plan will adapt as the science evolves. More importantly, through upfront structured planning, the company will avoid road bumps and move faster as it progresses towards commercialisation of the product.

Developers of innovative therapies face challenging and complex considerations. However, with proper strategic planning, organisations can clear the obstacles that lie ahead and move closer to commercialising the novel – even curative – therapies that patients require.

Christian K Schneider, MD, is head of biopharma excellence and chief medical officer (biopharma) at PharmaLex