The Value of Care Coalition previously asked: Who Do Prescription Drug Affordability Boards Actually Listen To? They don’t seem to be listening to the stakeholders providing comments or, in some cases, even their own analysts.
Now, a new fact sheet from VCC examines the guidance being given to PDABs and why the suggestions are concerning for patients, providers, and caregivers.
PORTAL Guides PDAB Processes
The Program on Regulation, Therapeutics, And Law (PORTAL) published white papers that, taken together, inform a toolkit for PDAB operations. The papers offer suggestions to PDABs regarding methodologies and data usage on topics like drug comparisons, cost reviews, and setting upper payment limits.
Unfortunately, PORTAL’s suggestions raise concerns for patients and clinicians.
Broad Drug Categorization
PORTAL suggests that prescription drug affordability boards assess drug affordability at the “active moiety” level. That is, they’re comparing drugs based on their active ingredients.
This means that variations in dosage and delivery method aren’t considered. For example:
- Liquid formulation is essential for children or those who have trouble swallowing pills.
- Extended-release versions may improve adherence for some patients.
- Specialized formulations exist because they provide unique benefits that other versions of the same drug can’t.
By grouping all drug variations together, PORTAL recommends PDABs make decisions that may ignore medical necessity.
Therapeutic Alternatives are not Therapeutic Equivalents
To evaluate the affordability of drugs and arrive at an upper payment limit, PDABs may compare targeted drugs to their therapeutic alternatives.
To select therapeutic alternatives, PORTAL recommends PDABs rely on clinical practice guidelines and unpublished clinical trials data. This could be problematic because:
- Clinical guidelines may not be consistent and provide generalized recommendations while not accounting for individual patient needs.
- Updates to guidelines can take years, which means they may not reflect innovations and medical breakthroughs, common off-label usages, or typical clinical practice.
- Data from unpublished studies may be unverified or incomplete, with outcomes for safety and efficacy that are not yet fully known.
Without robust, meaningful input from clinicians, PORTAL’s recommendations result in PDABs defaulting to one-size-fits-all models that lack real-world nuance. This could lead to forced medication switching not recommended by clinicians for their patients.
Costs Over Clinical Considerations
PORTAL recommends the integration of clinical and economic evaluations into one cost review process.
Common economic models, such as the QALY, are widely acknowledged to discriminate against patients with chronic illnesses, rare diseases, or those with disabilities. Plugged into calculations, these value considerations further devalue struggling patient populations.
Further, overreliance on current cost economic models may undervalue future treatment benefits. For example, timely access to treatment for a complex condition could prevent future spending on hospitalization, emergency care, and surgeries. These models may also fail to consider non-medical costs related to caregiving and lost productivity.
Without focus on the benefits of improved health outcomes, PORTAL recommendations may lead to short-sighted decisions with negative long-term consequences.
What Patients Need
PORTAL’s one-size-fits-all recommendations fail to allow for individual patient consideration.
No conversation about the value of a treatment can be complete without robust input from patients and clinicians and a true understanding by decision-makers – two recommendations not included in PORTAL’s guidance.