Boston, Mass., September 29, 2017 – The Institute of Clinical and Economic Review (ICER) has issued a Final Evidence Report assessing the comparative clinical effectiveness and value of poly ADP-ribose polymerase (PARP) inhibitors for treatment of ovarian cancer. The report includes a summary of a public meeting of the Midwest Comparative Effectiveness Public Advisory Council(Midwest CEPAC), during which the independent Council deliberated and voted on evidence presented in ICER’s report.

 

“Our report and meeting highlighted the need for further research on the long-term efficacy and safety of PARP inhibitors. In particular, information on improvement in overall survival is not yet available, and the trials of each agent differ in study population, design, and measurement of key outcomes. Some key studies involved no comparison to alternative treatments. Further, the high costs of the drugs do not align with their benefit to patients,” said Dan Ollendorf, PhD, ICER’s Chief Scientific Officer.

Dr. Ollendorf continued, “Despite these shortcomings, PARP inhibitors are the first novel treatments available for ovarian cancer in many years, and do have the potential to improve upon existing treatment options for many patients. Stakeholders must collaborate to conduct additional research that provides more clarity on the appropriate clinical use of these drugs, and to ensure that the high costs do not lead to restricted access for patients with already limited options. Our hope is that the objective analyses provided in our report can serve as a starting point for critical conversations around future research, pricing, and access.”

During the public meeting, the Midwest CEPAC voted on key questions related to the clinical effectiveness and value of the PARP inhibitors in two key populations: women receiving maintenance therapy for recurrent disease that has previously responded to a platinum-based chemotherapy, and women being treated for recurrent disease who have a breast cancer (BRCA) gene mutation. The Council found evidence sufficient to show a greater net health benefit of niraparib (Zejula®, Tesaro, Inc.) in either population, and of olaparib (Lynparza®, AstraZeneca) as maintenance therapy. Votes were split on the net health benefit of olaparib and rucaparib (Rubraca®, Clovis Oncology) in treatment of recurrent disease.

 

With input from patient representatives participating in the meeting, the Council further voted on the importance of other benefits and contextual considerations of the therapies, citing the reduced complexity of the treatment regimens, reduced caregiver burden, and the treatments’ novel mechanism of action as some of the most important added benefits of PARP inhibitors.

Voting concluded with Council deliberation on the value of olaparib in recurrent, BRCA-mutated disease, the only situation identified in ICER’s report in which a drug met commonly-accepted thresholds for cost-effectiveness. The Council’s value votes were split between low and intermediate, with members citing the significant uncertainty in the available data as the reasoning behind their votes.

ICER’s final report includes key policy recommendations aimed at advancing currently available evidence and supporting collaboration among stakeholders to ensure access for patients. Key recommendations include:

 
  • Current pricing of PARP inhibitors has the potential to align with clinical benefit in recurrent disease, but alignment will be more challenging for these drugs when used as maintenance therapy. To facilitate affordability and patient access, prices must be lowered.
  • Payers and manufacturers must work together to establish innovative payment mechanisms to seek affordability for patients, including outcomes based contracting and/or package discounting.
  • Single-arm studies and surrogate endpoints do not provide the type of information that clinicians and patients need to make treatment decisions.  Critical evidence gaps like these must be addressed in the design and execution of clinical research by researchers, manufacturers, and patient groups alike.
  • Current evidence is inadequate to determine which patients would benefit most from maintenance therapy.  Further research should be conducted to identify the patients who might benefit most from a maintenance regimen as well as those for whom surveillance remains a viable option.
  • Manufacturers should broaden eligibility criteria for patient assistance programs and price PARP inhibitors according to dosage strength to counter the financial toxicity that many patients with cancer experience.
A more detailed explanation of key policy recommendations is included in ICER’s full report. A summary of the key findings of the report is available in ICER’s Report-at-a-Glance.