The Promise of Personalized Medicine

An increasing number of cell & gene therapies are in clinical trials providing the hope of treatment for patients who have exhausted their options or continue to suffer despite the best efforts of healthcare professionals.

The progress made in the area of personalized medicine in recent years has opened up the field and garnered the attention of investors. However, many commercial challenges remain, particularly for autologous therapies.

Here are some of the key areas that need to be tackled:

Pricing

Pricing personalized therapy is a new challenge for the healthcare industry. Extremely high development costs, the need to invest in new processes and technologies, as well as long timelines from early-stage research & development to approval, add to the challenge. Patient profiles are also critical to consider. For example, what are the implications if lymphoma patients fall primarily in the Medicare age-range versus other age groups or covered populations. What are the critical factors that should inform pricing in this new era of personalized medicine? 

Approving Reimbursement

In addition to unprecedented pricing, the reimbursement/authorization process is cumbersome. Until these therapies are approved for first or second-line treatment, the extra burden is shouldered by patients, their caregivers, health systems, and healthcare providers. Currently, apheresis and CAR T therapy are coded together in payer systems, requiring approval before collection. During this period, if bridging chemotherapy treatment is required, it can compromise the likelihood of a successful (timely) apheresis collection, a critical first step to CAR T therapy delivery. How do we ease the burden and better operationalize authorization and reimbursement for personalized therapies? 

Standardizing Manufacturing & Supply

Now that there are a few approved therapies, how do we provide them to patients better, cheaper, faster? A lack of infrastructure and standards makes this particularly challenging. For example, is a product release standard specification of 70% viability appropriate? Ultimately, we need to determine what needs to be measured and what should be tested. Can we create shared protocols? How do we harmonize standards across multiple care centers? Improve logistics and transport? Use data collection and AI to optimize the supply chain? As we better understand the patient pathway, what guidelines will support safe, efficacious, and expedient production & delivery? 

Delivering Treatment

Each patient is unique. Particularly when it comes to these unique, personalized therapies, not everything works for everyone. It can take several iterations of drawing blood, re-visiting protocols, and re-administering to deliver therapy successfully. Yet, the source material is limited. As one healthcare provider said, “[in autologous therapy] you just can’t afford a failure”. From a clinical process and efficacy point-of-view, there is a lot to learn, and much is still unknown. How can we more quickly characterize diagnoses to identify potential patients earlier, improve the patient journey, and facilitate clinical efficacy?

Expanding Access

For these novel therapies to make a meaningful difference for more patients, the standard of care will need to change. Access needs to move beyond leading health centers in major cities, into community settings. What kind of education, infrastructure, and payment/reimbursement models are needed to increase access?

Healthcare stakeholders including regulators, payers, healthcare providers, and manufacturers must come together to address these issues. The solutions will come from insights about needs and challenges across the supply chain, down to the individualized patient experience.

By putting the Spotlight on patients, Luminous can map the patient journey and help biotech, pharma, medical device companies, cell collection and processing organizations, logistics businesses and payers navigate the complexity of personalized medicine.