Dr Emma Roffe, Oncology Country Head, UK & Ireland, Takeda UK, illuminates the role of combination treatments in the realm of rare diseases, and discusses the potential solutions to make them more accessible
Pharmafile: What has fuelled the high demand for combination treatments in the rare disease landscape?
Emma Roffe: Combination treatments are becoming more common as the understanding of complex diseases increases, especially in areas such as cancer, HIV, rheumatoid arthritis, hepatitis C, and rare disease. This is because using multiple medicines in combination helps to simultaneously target different pathways that can drive a disease.
These treatments have the potential to offer significant clinical benefits to patients, including improving their health outcomes and quality of life. For example, in cancer, there is broad consensus that combining different treatments, with different modes of action, may improve upon the efficacy of just using a single medicine.
Combination treatments have also been revolutionary in infectious diseases, such as HIV and Hepatitis C, where viruses can quickly adapt and become resistant to single treatments.
Rare cancers, like multiple myeloma, are currently treated through daily use of corticosteroids. What potential do combination treatments have for patients with rare disease?
Rare diseases are particularly complex, and we are continuing to discover diverse ways to tackle them, particularly rarer cancers. Due to the nature of many rare diseases, there is a need to use a combination of treatments to help improve patient survival and quality of life. This is particularly evident in the treatment of rare cancers, such as multiple myeloma, where doublets, triplets, and even quintuplets are used to tackle different biological pathways of the disease. Many of these combinations include corticosteroids administered in combination with other treatments that have different modes of action.
What are the main challenges in making combination treatments available for the wider population in the UK?
Despite potentially offering significant clinical benefits to patients, the availability of combination treatments remains a significant challenge as they often face cost-effectiveness barriers even if one of the medicines were to be given away at zero price.
Combinations are usually made up of two or more parts: a ‘backbone treatment’ which is often the current standard of care, and a new ‘add-on’ treatment which is given in combination with the backbone. As the use of combination treatments extends the time before a patient’s disease worsens or progresses, the backbone treatment is often used for longer. This alone can increase the cost of the combination treatment to the healthcare system, even before the cost of the add-on treatment is considered. This is made even more challenging due to strict competition law, which prohibits and restricts discussions between pharmaceutical companies on commercially sensitive topics, such as pricing and reimbursement of their treatments.
There is also no mechanism to apportion value to the component parts of a combination treatment, and the full responsibility falls on the ‘add-on’ treatment manufacturer to bear all the costeffectiveness burden. It is also the case in the UK that we do not have indicationspecific pricing. This means that any price negotiated for the treatments used in combination would also apply across all indications for those treatments, whether prescribed as a single (mono) therapy or in another combination.
Until now, the challenge of combination treatments has firmly sat in the ‘too hard to fix’ box, despite there being consensus within key national and international stakeholder groups that solutions for accessing combination treatments need to be found quickly for the benefit of patient outcomes.
Takeda’s proposed solution suggests ways to tackle the primary barriers in making combination treatments accessible, such as improving cost effectiveness and encouraging dialogue between companies. What does this proposal entail?
In partnership with experts from the patient, academia, clinical, and competition law communities, and with input on key challenges from experts from the NHS and NICE (National Institute for Health and Care Excellence), Takeda has developed a proposed solution for improving access to combination treatments. This solution is proposed in two Whitepapers: An Attribution of Value Framework and The Voluntary Arbitration Framework.
The Attribution of Value Framework proposes an economic methodology that aims to define a fair division of value across the treatments in a combination. It does this by assigning a relative value to each treatment, based on its health benefit.
The Voluntary Arbitration Framework proposes a standard operating procedure to support compliant dialogue and agreement between pharmaceutical companies, on the value attributed to each treatment within a combination.
Takeda’s solution takes into consideration the current processes used by NICE and NHS England in making decisions on access to medicines and competition law. It aims to contribute to the options being explored by other stakeholders, to find effective and implementable solutions that align with current health technology appraisal methods.
We also propose that any solutions that are implemented to address the combination treatment challenge, become embedded into the existing ‘voluntary scheme for branded medicines’ to encourage universal participation of the pharma industry, the NHS, and the wider healthcare community.
Takeda welcomes feedback, critique, and debate of the two whitepapers so that we can contribute to finding a solution that, not only ensures patients can benefit from the scientific innovation and promise of combination treatments, but is also accepted by all stakeholders.
What are Takeda’s hopes and visions for the future, in improving patient outcomes for those with rare disease?
Takeda is guided by an unwavering commitment to put the patient first in everything we do and in the decisions that we make. As part of this, we have a long history of collaborating with healthcare systems, regulatory agencies, health technology appraisal bodies, payers, and the clinical and patient communities, to find solutions to complex challenges that enhance patient access to effective and well-tolerated treatments.
The cost-effectiveness challenge faced by combination treatments is a significant and growing issue that has for too long sat in the ‘too hard to fix’ box. As a trusted partner, we were well positioned to bring together numerous stakeholders to develop a proposed solution that aims to ensure patients benefit from the potential of combination treatments as quickly as possible.
We hope that a solution, whatever that may look like, is quickly embedded into the healthcare system for the benefit of patients now and in the future.
Dr Emma Roffewas appointed Oncology Country Head for Takeda UK and Ireland in 2018, joining the company’s Senior Leadership Team. Her appointment followed a 15 year career at Takeda, working across a number of its portfolios, having joined in 2003 as a Scientific Adviser.