Patients Rising is a Washington, DC-based non-profit organization with a very specific mission: we fight for access to vital therapies and services for all patients with life-threatening and chronic diseases. That is why Patients Rising is committed to speaking for patients as part of a balanced dialogue with providers, payers, policymakers and the advocacy community to address the complement of cost and access challenges Americans with serious diseases face today.
Clearly, one of the most pressing issues is improving access to, and reimbursement for, novel medicines that improve and extend people’s lives. This is especially the case for patients with chronic liver diseases, such as primary biliary cholangitis (PBC) and non-alcoholic steatohepatitis (NASH), which can lead to liver failure and death if not adequately treated. Thus, Patients Rising signed onto the joint letter from the Global Liver Institute and other liver disease organizations that raises serious questions about the Institute for Clinical and Economic Review’s draft reports on obeticholic acid (OCA) for the treatment of PBC and NASH.
Additionally, Patients Rising plans to join the Global Liver Institute in attending the CEPAC meeting in Portland on July 15th to raise our concerns that ICER’s draft reports on OCA, which could have detrimental consequences for the estimated 130,000 Americans living with PBC and the approximately 30 million with NASH. Our specific concerns are summarized below.
1.) Many Americans with PBC and NASH Have Few Treatment Options
In announcing accelerated approval of OCA on May 31, 2016 – five days after ICER announced its draft reports – the Food and Drug Administration’s (FDA) presented a compelling argument for why this new therapy addresses an unmet need for patients. Although the only other treatment for PBC, ursodeoxycholic acid (UDCA), is effective in about 50 percent of patients, up to 40 percent do not achieve an adequate reduction in blood chemistries with UDCA, while 5-10 percent are unable to tolerate the therapy.
Of equal significance, FDA granted OCA orphan drug status due to the very small patient population for PBC and the fact that since UCDA was approved more than 20 years ago, there had been no other treatment option available. When FDA’s Gastrointestinal Drugs Advisory Committee voted unanimously in April 2016 to recommend accelerated approval of obeticholic acid, committee members agreed that OCA fills an unmet need for patients with rare and difficult liver diseases.
2.) ICER Has Not Consulted with PBC and NASH Experts and Advocates Who Best Understand the Science and Limitations of UCDA
As documented at the Gastrointestinal Drugs Advisory Committee meeting in April, PBC is a rare and potentially fatal disease leading to liver failure and the need for a liver transplant in the 40-50 percent of patients who do not respond to UDCA. Yet, ICER conducted its assessments without appropriate patient participation in the clinical evaluation process and none of ICER’s panel members has treated a PBC or NASH patient.
It is because the patient experience is absent that Patients Rising considers value frameworks – as they are currently being used in healthcare decision-making – as soulless backroom number crunching exercises. Obviously much can be garnered from study findings, and it appears ICER has gone to great lengths to qualify and quantify data for its evaluation. However, without considering value at the individual patient level, treatment options for rare diseases will be deemed too costly and access will be limited.
3.) ICER’s Clinical Effectiveness and Value Assessment of OCA Is Premature
When it comes to assessing the “value” of new drugs for orphan diseases like PBC, it is premature to even focus on a therapeutic category where there have been no new treatments in more than 20 years. Further, the only available evidence on OCA involves patients treated in clinical trials, which does not reflect the potential use of this new therapy in real-world practice.
This rush to evaluate a new medication that has yet to be prescribed and taken by eligible PBC patients raises the question of why ICER is moving forward quickly to estimate OCA’s potential off-label use in treating NASH? The only conclusion is that ICER’s report will be used by health plans to keep NASH patients from receiving this form of treatment, even though the disease can lead to cirrhosis, in which the liver is permanently damaged and scarred.
4.) ICER’s Model Will Impede Physicians’ Clinical Judgement
No matter how much data is collected and analyzed, value assessments should not replace the clinical judgement of physicians treating the patient in front of them. Especially in the case where meaningful Phase III evidence is lacking, we must rely on the doctor’s understanding of the differences among each patient and whether specific patients have co-morbidities or prior toxicities that must be taken into consideration, which no value framework can ever really assess.
5.) Concerns About ICER’s Cost Effectiveness Model
In both of ICER’s draft reports, the organization makes an assumption about the cost for a year’s worth of OCA treatment absent any list price at the time of this report. A $65,000 list price is used for analysis, which ICER has deemed too high and has set a $15,000 amount for overall fiscal sustainability. Yet, from our discussions with liver disease organizations, it is apparent ICER uses a different model for liver transplants where the average negotiated rate for insurers is used instead of an arbitrary list price off of a hospital charge master. Thus, if ICER is willing to use this real-world approach when analyzing the true estimated cost of a hospital procedure, the same parameters should apply to new drug treatments because insurance companies and pharmacy benefit manager rarely, if ever, pay a list price.
Additionally, we are aware that ICER has not taken into consideration the following factors when determining the cost of OCA:
- Additional competitive treatments entering the market
- Rebates provided by pharmaceutical companies to pharmacy benefit managers
- The drug going off-patent
- The cost to develop the medicine, which in this case is 14 years
Beyond these specific issues, the burning question for Patients Rising is why is ICER putting OCA under the microscope now? Is the goal to question the value for this new therapy before it even reaches patients so as to narrow utilization and therefore, limit costs to payers?
Like other advocates and healthcare stakeholders, we share ICER’s concerns about the rising costs of care. Yet, what is in the best interest of all Americans is better ways to treat diseases so patients can live long and prosper – and not methods that devalue patients’ needs for the sake of overall national sustainability.
Thank you in advance for considering our views.
Co-Founder & Executive Director, Patients Rising