Comments on Negotiation Data Elements and Evidence about Alternative Treatments for the Medicare Drug Price Negotiation Program
Dear Dr. Meena Seshamani, M.D., Ph.D.,
Thank you for the opportunity to comment on the negotiation data elements for manufacturer submitted data and evidence about alternative treatments to support implementation of sections 11001 and 11002 of the Inflation Reduction Act (IRA) (P.L. 117–169), which establishes the Medicare Drug Price Negotiation Program to negotiate maximum fair prices for certain high expenditure, single source drugs and biological products.
My comments on the negotiation data elements are reflective of my experiences as a Health Economist in the pharmaceutical industry generating and leveraging evidence for Health Technology Assessments and drug price negotiations with government payers, primarily outside of the United States. I am also a Doctoral student in Health Policy at Johns Hopkins Bloomberg School of Public Health. The comments I have provided below are my own and do not reflect the opinions of any organizations that I am affiliated with other than myself.
In the final version of the Negotiation Data Elements ICR form it is critical that CMS provide more specificity regarding the information that should be generated and provided to assess a drug’s therapeutic impact and comparative effectiveness in Question 41.
My first comment on Question 41 is referring to the instructions. It currently states, “Specify the therapeutic alternative and indication of the selected drug that you are discussing”. This statement requires clarification regarding how the appropriate “therapeutic alternative” will be determined. Dr. Ziouani, Dr. Granados and Dr. Borget assessed the most common approaches to determining therapeutic alternatives for comparative effectiveness assessments in their publication, “How To Select The Best Comparator? An International Economic Evaluation Guidelines Comparison”. It was determined that the most common approach to selecting a therapeutic alternative is to use the standard of care for local practice. Standard of care can be determined by referencing treatment guidelines and should be pre-specified by CMS for each selected drug.
My second comment on Question 41 is also referring to the instructions. It currently states, “When discussing the therapeutic impact of the selected drug, indicate outcome(s) used, the indication(s) to which the evidence applies, and the therapeutic alternative(s) to which the evidence applies.” This statement is ambiguous as currently written and requires more clarity on the outcomes of interest. Potential outcomes of interest could be the primary and secondary endpoints that were included in the clinical trial which was used to achieve FDA approval.
My third comment on Question 41 is referring to the question, “To what extent does the selected drug represent a therapeutic advance as compared to existing therapeutic alternatives? Please discuss for each indication of the selected drug, as applicable.” The definition of a “therapeutic advance” must be explicitly stated. A drug can be considered a “therapeutic advance” for a variety of reasons, which may differ by therapy area. Common attributes associated with “therapeutic advances’’ include improvements in clinical endpoints, improvements in a patient’s quality of life and ability to do daily activities, and a reduction in adverse events associated with currently available therapies.
Dr. Aris Angelis and Dr. Panos Kanavos have identified several attributes generally taken into consideration by payers, clinicians and patients when assessing the value of a medicine in their publication, “Multiple Criteria Decision Analysis (MCDA) for evaluating new medicines in Health Technology Assessment and beyond: The Advance Value Framework.” The attributes highlighted include burden of disease (severity, prevalence, availability of treatments), therapeutic impact (clinical endpoints and quality of life), safety (adverse events, tolerability and contraindications), innovation (nature of treatment and ease of use), and societal impact (improving public health, improving productivity, reducing other healthcare costs).
Some of these attributes may not be appropriate for the Medicare Drug Price Negotiation Program given the program will be focusing on single source drugs that have been approved for at least 7 years or biological products that have been approved for at least 11 years. Additionally a societal perspective may not be appropriate for Medicare given the intention of the Drug Price Negotiation Program is to lower the price of some of the costliest single-source brand-name Medicare Part B and Part D drugs. Therefore, the attributes that CMS should consider when defining a “therapeutic advance” should at minimum include burden of disease, therapeutic impact and safety.
In conclusion, Question 41 of the final Negotiation Data Elements ICR form should include the following clarifications:
- The existing therapeutic alternative should be the standard of care for the disease of interest. Standard of care can be determined by referencing treatment guidelines and should be pre-specified by CMS for each selected drug.
- The outcomes of interest should be the primary and secondary endpoints that were included in the clinical trial that was used to achieve FDA approval.
- A therapeutic advance should be defined as a reduction in burden of disease, a significant improvement in clinical endpoints or an improved safety profile compared to standard of care.
Thank you again for the opportunity to comment on the negotiation data elements for manufacturer submitted data and evidence about alternative treatments for the implementation of the Medicare Drug Price Negotiation Program. If there are any questions regarding my recommendations please do not hesitate to contact me via email at dnbargo@gmail.com.
Sincerely,
Danielle Bargo
