Five marketing strategies for novel therapeutics

Novel therapies, known as advanced therapy medicinal products (ATMPs) in Europe and as cell and gene therapy products (CGTPs) in the US, are game changers for the treatment of severe conditions that currently have few or no therapies or treatment options. Driven by scientific innovations, impressive clinical outcomes and a succession of new product approvals, the market for advanced therapies is projected to be worth almost $21.2 billion by 2028.¹

With this kind of momentum in the sector, we are at the threshold of a new era in how patients are treated – and how disease and illness can be prevented and managed. The ATMP sector is now considered as being at an “adolescent stage” by many analysts; holding out the promise of making personalised medicine a reality and improving global health through the wider accessibility of innovative and personalised medicines and devices.

Risk Mitigation

Promise, however, brings challenges with it. Innovative therapies employ a model that is markedly different from conventional development paradigms — and one for which more tailored approaches are needed. At heart, the “traditional” clinical development paradigm is often simply not applicable to these organisations. That is because ATMPs are dealing with smaller patient populations; special requirements for manufacturing where patients’ lives can depend on the speed at which a therapy can move from bedside to manufacturing and back again; and pricing models that can make the therapy prohibitive for many payers. 

To obviate risks along the way, companies should 1) plan early, building bridges between quality (chemistry, manufacturing and controls [CMC]), non-clinical and clinical disciplines; 2) develop a regulatory strategy as soon as drug development begins; and 3) analyse the healthcare landscape to determine the market access model that will provide a strong value proposition for decision-makers and payers. 

Most importantly, organisations should initiate interactions with regulatory authorities early in development planning. Because ATMPs are complex biological entities, current regulations around them are also complex — and constantly evolving. Sometimes, regulatory scientific guidance can be too general for a developer to know how exactly to apply it to a given novel product. This is because regulators are unable to anticipate future developments when they draft their guidance documents.

Regulatory agencies should be involved throughout a development programme so they stay in agreement, and so that organisations can incorporate their insights into the programme. Regulators are increasingly open to dialogue for immature and early programmes, and they see their roles as enablers in addition to their more traditional roles as gatekeepers.

Ultimately, the goal is to build an agile approach to planning that minimises delays or risks of failure.

Five Risk-Reducing Strategies

From day one, development planning should aim to build bridges between quality, non-clinical and clinical disciplines.

There are many complexities to consider in the commercialisation process for advanced therapies. Patient populations are very often smaller and more targeted and even though that means that product quantities can be low, they also have very specific logistical requirements. For example, 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 in cases where shelf-life is very short.

No matter how potentially transformative advanced therapies might be, pricing for them may ultimately prove prohibitive for some payers. At the same time, the underlying quality, regulatory and manufacturing guidelines that apply to traditional drug development must still be considered – and those guidelines can be nuanced depending on country or region, which can make them challenging to navigate.

1. Undertake a risk/benefit assessment: ATMPs have considerable potential to provide significant treatment options, perhaps even with curative intent, but they also come with significant known and unknown risks, many of which are unique to this product class. Therefore, risk too needs to be considered from an early stage – with a primary focus on safeguarding the patient but also minimising risks to 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 toward risk identification, evaluation and mitigation. That agile approach should apply not only to the biological activity of the ATMP, but also the quality attributes, the manufacturing process steps and the therapeutic administration procedures.
2. Set up an integrated product development plan (IPDP): For a holistic approach to the creation of an 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 as well as pricing /reimbursement aspects all need to be considered. The IPDP is a living document that will be continually updated as development progresses, promoting organisational prioritisation and decreasing time-to-decision. Defining the patient population and the target stage for a given disease, for example, are important considerations that could have an impact on the design of non-clinical studies etc.
3. Consider models to scale manufacturing: The path to commercialisation right from day one. Moving the therapy from the lab to scaling it for supply to patients, which means producing a sterile drug product in sufficient quantities, can be challenging. To ensure scalability without burning cash as you go, organisations must align manufacturing readiness with the regulatory pathway, the patient population and the dosing that is being pursued.
4. Speed up commercialisation with an effective regulatory strategy: There are distinct aspects to the regulatory plan – all happening 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 along with development and as new information about the competitive environment, study results and interactions with regulatory agencies come in.
5. Ensure that you have a market access strategy: To gain market access, developers must be able to demonstrate clinical and economic evidence to providers, healthcare decision-makers and importantly, payers. Given the complexities of the way healthcare is paid for, it is crucial to understand who will finance the therapy and the mechanisms by which the care will be reimbursed. Developers must consider strategies that take a more holistic view of patient treatment and provide better real-world evidence, therefore offering a stronger value proposition for decision-makers. This planning must begin at the onset of an idea, during the proof-of-concept phase, so that later considerations on risk-benefit and cost-benefit converge and can be derived from overlapping evidence generated throughout the development.

A starting point only

An IPDP is crucial when moving from research to development. Organisations must go through the planning process with the understanding that this will be a starting point only and that the plan will adapt as the science evolves. More importantly, through upfront structured planning – even while acknowledging things will change – the company will avoid road bumps and move faster as it progresses toward commercialisation.

Developers of innovative therapies are charting new waters and have complex considerations to navigate, but proper strategic planning will help organisations to clear the obstacles that lie ahead and produce the ground-breaking new therapies that patients need.

About the Author

Christian K Schneider, MD, is Head of Biopharma Excellence and Chief Medical Officer (Biopharma) at PharmaLex. He was previously interim Chief Scientific Officer at the UK’s Medicines and Healthcare products Regulatory Authority (MHRA), where he was also Director of the National Institute for Biological Standards and Control (NIBSC) for five years. He has also held leading positions at the Danish Medicines Agency and at the Paul-Ehrlich-Institut, Germany’s Federal Agency for Vaccines and Biomedicines.

At the European Medicines Agency (EMA), he has chaired the Committee for Advanced Therapies (CAT) as well as the Biosimilar Medicinal Products Working Party (BMWP) – and served as a member of the Committee for Medicinal Products for Human Use (CHMP). He is one of the key architects of EMA’s advanced therapies and biosimilars framework. As a regulatory scientist, Christian has published 50+ articles in international, peer-reviewed journals.

Christian is contactable at: [email protected]

References

1. Advanced Therapy Medicinal Products Market Worth $21.2 Billion By 2028 [Internet]. Grandviewresearch.com. 2021 [cited October 2021]. Available from: https://www.grandviewresearch.com/press-release/global-advanced-therapy-medicinal-products-market
2. Verdin P, Mon Tsang T. Next-generation therapeutics thrust into the spotlight. Biopharma Dealmakers [Internet]. 2021 [cited October 2021]. Available from: https://doi.org/10.1038/d43747-021-00105-y