The Challenges that Impact Patient Perspectives and Priorities for Advanced Therapies in Europe

When it comes to innovation in biosciences and health, advancements in medicine are cultivating new ways to create unique and potentially life-changing solutions for individuals with a range of complicated health conditions, and health overall. Advanced therapies, such as cell and gene therapies have demonstrated life-altering and life-extending outcomes for patients treated. Although advanced therapies are highly complex and not without risk, trials indicated success in the treatment of advanced cancers where other traditional treatments have failed.

In the UK, the NHS is providing NICE-approved CAR-T treatments for children and young people with B-cell acute lymphoblastic leukemia. This type of immunotherapy involves collecting and using the patient’s immune cells to treat their condition. A panel of expert clinicians decides upon the eligibility for patients undergoing CAR-T treatment following a referral from a specialist doctor. Treatment involves several steps over weeks where the patient’s blood is taken, processed at the manufacturer’s laboratory, and modified to target the cancer cells.

Cell and gene therapies have the potential to be curative approaches to a significant number of genetic diseases, however, there are obstacles preventing patient access to gene therapies based on the need for positive market authorization limiting patient access to treatment. Where advanced treatments such as cell and gene therapies can potentially eliminate lifelong treatments, patient access is currently one of its biggest challenges yet to overcome. For this article, I will discuss the areas that may hinder patient perspectives and priorities such as patients’ beliefs and perceptions, healthcare professionals, pricing regulations and briefly, manufacturing.

With Advanced Therapies Europe on the horizon, let’s kick start the conversation into the critical importance of patient perspectives and priorities with cell and gene therapies.

What are the Fundamentals of Patient Beliefs and Patient Perceptions?

In medicine, practicality and ethics are constantly in discussion. How do we dictate who receives advanced treatments while paying careful and considerate attention to a patient’s quality of life?

The patient perspective is the patient’s experience of the disease, treatment and the impacts on their caregivers, psychosocial, spiritual and future prospects in the outcome of their illness.

Having a serious illness may lead to false hope in the appropriate applicability of novel treatments under clinical trials, especially where other treatments may not have been successful. Advanced therapies, such as cell and gene therapies, have a promising ideological effect on treating disease, especially where the narrative may suggest curative potential. Still, there is so much that goes into how accessible, successful and safe these new treatments really are.

Many treatments are immediately inaccessible to those who cannot afford them. In considering advanced treatments, there is more demand – in theory, than there is supply, and the cost of these treatments is beyond what the majority of people affected can afford. Healthcare funding opportunities vary between countries, and for most, healthcare and reimbursement structures are not publicly funded. However, even in countries where public funding is an option – such as with the UK’s NHS – healthcare systems still are not in a position to afford these treatments for very many patients, and currently remain under pressure due to insufficient funding in an era of increased cost of living.

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The Role of Healthcare Professionals in the Treatment Process (UK)

A major challenge in the UK is the system by which the NHS pays for treatments, such as medicines and medical devices.

There has been hesitancy towards the use of advanced therapies where there remains uncertainty into  such as the overall long-term effect and the redaction in approved medication.

Clinical trials are vital in drug production and we are all aware that just because something works doesn’t equate to the efficacy on human patients. As some advanced therapies are using individually modified cells, the risk seems to outweigh the potential success. Currently, there are more than 20 FDA-approved cell and gene therapy products available for a range of diseases. There has been rapid growth in clinical trials with more than 2000 active and upcoming trials registered on ClinicalTrials.gov. This is good news, however, the rate at which clinical trials get approved and funded does not align with the ideal timeline pace most companies fundamentally need.

Though every step in each process is crucial, overseen to the highest standard within guidance and regulations, it does delay the process of production and scalability to mass produce – thus increasing the theoretical demand over the supply available. As a result, the cost of vital treatment remains high.

How Pricing Regulation Hinders the Access to Treatment

The cost of effective treatment for diseases deemed incurable by nature and aggression means that an advanced solution is not the most logical and cost-effective for institutes such as the NHS. Health equity and access to cell and gene therapy are no doubt inaccessible to those in need when first-in-line treatment, such as chemoimmunotherapy, does not work for the individual.

The high up-front costs and uncertainty with regard to the long-term outcome are not ideal for patients, insurance companies and hospitals. Payment models are being explored, but because cell and gene therapies are high, complex, expensive, specialized treatments, either experimental or available, they are only specific to certain patient populations.

As cell and gene therapies becomes more available, awareness has been raised to inspire others to take action to help reduce the limitation on treatments available for those in need. Some organizations are developing individualized experimental therapeutics for patients regardless of their ability to pay. The BIA analysis of Pitchbook, July 2021 , stated that there were over 100 UK companies working on new ways to design, manufacture and safely administer cell and gene therapies, driving next-generation approaches

The Setbacks in the Manufacturing Process

Manufacturing is one of the biggest issues for cell and gene therapy contributing to the high cost of goods and the cost of the drug. Manufacturing a scalable product that is just as effective on a small, personalized scale is an issue yet to be solved in a reasonable, effective way. The shortage of experienced and knowledgeable experts within the field, which is rapidly growing, is yet to be filled in surplus to fulfill the growing need for experts to manage the scalability. There have been a variety of solutions implemented such as automation, digitalization and standardization.

Conclusion

The EU legislative review needs to be revised to allow more advanced therapy medicinal products to market. The regulatory framework for ATMPs is to ensure the free movement of medicines with the EU to facilitate their access to the EU market, and to oversee the competitiveness of European pharmaceutical companies in the field while guaranteeing the highest level of health protection for patients. As more advanced therapy medicinal products emerge into the innovations of medicine, tackling these challenges on a continual step to creating better and quality access to these services, strengthening the fight against life-altering or threatening diseases.

There are many foundations that bring awareness and support to patients and information to other stakeholders. An important step in the development of drugs and advanced therapies is to educate those involved. By bringing the conversation of advanced therapies into the stream of cancer treatment and with the emerging treatment of other diseases, we can tackle one of the few challenges.

At Advanced Therapies Europe, the conversation for these exact issues is just the starting point of a wider conversation. There is so much more to come from advanced therapies through thought leadership, innovative ideas and community. As we process in this innovative field of medicine, the future looks promising. Will you be joining us at Advanced Therapy Europe this Autumn to continue the conversation?

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