Solving One of the Hardest Questions in Cancer: How Do We Ensure Patient Access to Medicines?

Liz Lewis
Head of global
oncology pricing,
value, and access
Takeda

An article published earlier this year in Nature¹ by Dr. Fabrice André, Director of Research at Gustave Roussy and President-elect of the European Society of Medical Oncology, remains fresh in my mind. In the piece, Dr. André emphasized the need to reclassify the way we name cancers–shifting the lexicon from organ of origination to molecular characteristics.

His argument was compelling–with all that we know today about cancer, why has the industry not adopted mutation-based clinical trials, for example, to be able to treat the same type of cancer in different types of organs? While this is certainly thought provoking, it would require a major shift across manufacturers, researchers, payers, physicians, patients, and many other constituents to bring to fruition. With that in mind, what really struck me about Dr. André’s commentary was the idea that classifying cancers according to their molecular characteristics versus organ of origination could ultimately speed patient access to medicines.

Naming conventions aside, pharmaceutical companies are facing new guidelines, laws, and regulations requiring a reconsideration of how we prioritize, develop, commercialize, and ultimately, secure access to medicines. We are tasked with one of the biggest obstacles of our industry in decades: how do we effectively bring innovative medicines to patients in need and avoid leaving anyone behind?

In oncology, this is particularly difficult, and I would go as far as to say this field is faced with pressures that may not exist to the same extent in other disease states. For example, the development of a cancer medicine is often not “one and done.” Research and development of a compound often involves evaluating several different types of indications across several different types of cancers, over several years. Notably, more than half of new approvals for orphan oncology conditions² from 2013 to 2017 were subsequent indications.

New regulations add further complexity to this. In Europe, the introduction of joint health technology assessments (HTA) provides the opportunity to receive comprehensive scientific advice on drug development and access. On the other hand, it also creates challenges, as a pharma company cannot feasibly undertake all the advice it receives. One example of this is when companies are directed to run additional clinical trials against comparators that are not the standard of care in other countries. In cancer, especially, standards of care and data requirements can vary greatly across different countries, which can make this process incredibly challenging. What’s more, in 2022, U.S. President Biden signed the Inflation Reduction Act (IRA) into law, with sweeping implications across many different industries. In pharma, the law penalizes manufacturers whose drugs’ prices surpass the inflation rate and creates price controls that can threaten innovation, especially for small molecules–a significant provision for drugs that could be developed to treat several types of cancers.

These factors and regulations have caused pharma to reassess how it’s doing business. Now more than ever, we need to think strategically about our approach to drug development–from the pursuit of potential follow-on indications that can arise when we assess the molecular characteristics of a therapy to patient access. While we’re all still learning and adapting to new policies that affect the pharma industry on a global scale, it is also critical that we figure out how to work within these new parameters–and do so quickly.

In pharma, we need to keep a constant pulse on these kinds of regulatory changes, working swiftly to develop tools, resources, and insights that support ongoing innovation and doing so in close collaboration with both internal and external partners.

I work with a dedicated group of individuals at Takeda to do just this. My team developed a framework to guide global product teams on how to think about pivotal clinical trial designs and go-to-market considerations so that we are better positioned to make informed decisions around drug development and delivery. To my point earlier, the pursuit of multiple indications is a key aspect of cancer care because we know that small indications are often building blocks to other indications, and this framework empowers product teams to think about how to attain follow-on indications as soon as possible and ensure long-term viability of our medicines.

But this effort–and the work of one team–cannot succeed in a vacuum; ensuring medicines reach the patients who need them requires ongoing collaboration. Engaging with external agencies, organizations, and working groups that are shaping global policy and regulatory landscapes is critical to ensure the perspectives of pharma companies are considered. Additionally, it is our responsibility to embed pricing and access considerations in the R&D process from the beginning to foster meaningful improvements in care and benefit the lives of patients around the world.

As I think back to Dr. André’s article and reflect on these ever-changing policies and regulations, it’s clear to me that the oncology industry is in need of creative solutions to enable and accelerate patient access. We know that change is and will remain a constant, and it takes a village to find ways to adapt, regardless of the obstacle we may face, for the benefit of patients. Let us approach each challenge with a solutions-oriented mindset and never forget who we’re fighting for.

Sources

1. Andre, Fabrice; Rassy Elie; Marabelle, Aurelien; Michiels, Stefan; Besse, Benjamin. Forget Lung, Breast or Prostate Cancer: Why Tumour Naming Needs to Change. Nature. January 31, 2024. https://www.nature.com/articles/d41586-024-00216-3
2. Patterson, Julie; Motyka, James. Unitended Consequences of the Inflation Reduction Act: Clinical Development Toward Subsequent Indications. AJMC. February 2, 2024. https://www.ajmc.com/view/unintended-consequences-of-the-inflation-reduction-act-clinical-development-toward-subsequent-indications