Instilling Value Early in Product Development: A Pharma Perspective

Provider organizations, health policy researchers, and patient advocacy groups have been working hard toward developing a well-rounded value framework, and the pharmaceutical industry is taking note and working to ensure these frameworks are fair on their product. At the 8th Annual Oncology Market Access & Pricing USA 2017 meeting hosted by eyeforpharma, pharmaceutical developers shared strategies on how a “value” culture can be infused right from the preclinical stage of product development.

“The current value frameworks do not do a perfect job of assessing value,” said Matthew Monberg, PhD, director, Outcomes Research Immuno-Oncology/PD-1 at Merck. Monberg noted that while value can be defined in several different ways, the existing frameworks should not be dismissed, “because they are not going away.” He also emphasized that everyone across a company that is involved in the drug development process should be aware of the existence of these frameworks.

Monberg noted that cost-effectiveness models are a complex undertaking and simplifying them could lead to important parts being missed within the assessment. He explained that framework designers would say that if there are issues then there are also possible solutions, “and in some cases, we should assume that framework designers mean the best.” Monberg said.

“In the United States, value frameworks are influential as far as they are credible,” Monberg said. Citing “utility” as a example of an outcome that is measured in a standardized way, Monberg said that drug developers should collaborate with and educate product assessors to help them make informed decisions.

The bottom line though is that patients are paramount and they should be at the forefront of all our decisions, Monberg emphasized. “Patients are not monolithic and it’s an important thing to consider with trial design and other research decisions.”

Keeping patients front and center was also at the core of the presentation by Amanda Bruno, PhD, senior director, value evidence leader, Cross Therapy Area, GlaxoSmithKline, who spoke about establishing a future-proof value creation model.

Value assessment, per Bruno, should,

• Capture the basic metrics
• Have the patient perspective at the heart
• Ensure flexibility and be tangible in the future

This kind of an assessment necessitates asking the right questions to achieve the end goal, which Bruno noted, should be:

• Develop medicines that are valuable to patients, physicians, and payers
• Generate value evidence to allow timely access to medicine, using real-world evidence, randomized clinical trials, and sponsored trials
• Consistently embed value throughout strategy, from discovery through commercialization
• Have a strategic evidence generation plan to ensure patients have access to and use the medicine

“To get there, we need to understand the value drivers for oncology medicines and lead your organization to consider all aspects of value in their decision making,” Bruno said, adding that while there are no “magic bullets” for generating the desired value evidence, the concept should be instilled early on in drug development.

Incorporating patient-reported outcomes measures is important, Bruno said, and patient input should be utilized to fill evidence-gap analysis, data collection tools, and randomized clinical trial design.

Monberg said that the P.I.C.O. model is important to address value in the clinical context:

• Patient, Population or Problem: Ensure that the scope of assessment is broad and detailed
• Intervention: Does the intervention incorporate value in trial design? Maybe subdividing the population based on biomarkers could be an answer.
• Comparison: What are the relevant comparators? Are they a single agent, a therapeutic class, or a meta-analysis?
• Outcomes: Timing of the assessment is important, especially in the context of understanding the full value of the product. “There is conflict between outcomes of interest for regulatory approval versus reimbursement approval,” Monberg said. This raises the value of endpoints, including quality-of-life endpoints. He also indicated that real-world evidence has a significant bearing on outcomes evaluation.

A persuasive and decisive leader can help instill this value goal, Bruno said, including getting buy-in from stakeholders, ensuring business results are delivered, aligning everyone around value creation, and communicating effectively.