Collaboration Needed To Ensure Access To Orphan Drugs – Express Scripts Exec

In an effort to ensure that there is access to high-priced drugs that treat rare diseases, relevant stakeholders, including manufacturers, payers, patients and physicians, need to come together and collaborate on access solutions that meet the needs of all parties, Express Scripts VP of Pharma Strategies and Account Management Bill Martin tells NORD/DIA conference.

Ensuring that high-priced specialty drugs for orphan conditions remain accessible to patients requires a collaborative effort among stakeholders, Express Scripts Holding Co. VP of Pharma Strategies and Account Management Bill Martin said.

Speaking Oct. 22 at the “U.S. Conference on Rare Diseases and Orphan Products: Shaping the Future Now” in Washington, D.C., Martin noted that all the key stakeholders in this conversation – patients, physicians, manufacturers and payers – all have a slightly different perspective when it comes to orphan drugs. “If we get the parties to talk, maybe that’s where we find the resolution in this.” The conference was co-sponsored by the National Organization for Rare Disorders and the Drug Information Association.

Martin framed the conversation around the different needs each stakeholder has: patients are seeking affordable access; physicians want to be able to make appropriate prescribing decisions; and biopharmaceutical firms are seeking to roll out new treatments to help cover losses from blockbusters going off-patent. Many treatments targeted at smaller populations are coming out with high price tags. From a regulatory perspective, orphan drugs typically have a lower bar to meet regarding clinical trials needed for approval, but the flip side of that is that payers may limit coverage to patients meeting the same parameters that were used for enrollment in those trials.

The payer perspective in some ways becomes the lynch pin to the access question, since they provide the coverage, and in many cases set tight restrictions on orphan drugs’ use. A specific example presented during the conference session was with patients suffering from paroxysmal nocturnal hemoglobinuria (PNH).

Tamir Orbach, former president of the PNH Research and Support Foundation, talked about the only FDA-approved drug to treat PNH, Alexion Pharmaceuticals Inc.’s Soliris (eculizumab). Coverage of the drug is typically limited to the approved indication that was based on how participants in the pivotal clinical trial were screened. It received regulatory approval March 16, 2007, and remains one of the highest-priced orphan drugs (Also see "Alexion’s Soliris Blood Treatment Will Cost Almost $400,000 Per Year" - Pink Sheet, 2 Apr, 2007.). In general, higher price tags for orphan drugs have brought an increase in payer scrutiny of the value of these drugs (Also see "Pricing Rare Disease Drugs Requires Care As Payers Increase Scrutiny" - Pink Sheet, 29 Aug, 2011.).

Since then, retrospective studies from the past seven years suggest that more people suffering from PNH could benefit from treatment beyond those meeting the standards used in the pivotal trial. However, Orbach noted that insurance companies “have based their approval for Soliris on these seven-year-old initial clinical trial data” and are not taking into consideration retrospective studies that suggest a larger population of PNH patients could derive benefit from this product.

“What’s happening is these insurance companies are refusing to change their coverage policies based on retroactive or retrospective studies,” Orbach said. “They want new studies” to justify broadening the coverage. “The problem is because PNH is so rare, those clinical trials are probably not going to happen. There aren’t enough patients to go on to new trials.”

Express Scripts’ Martin agreed, saying the conundrum applies to orphan drugs in general. “The evidence to hurdle is lower for orphan approval, but the evidence to support the broader coverage” is greater, he observed. But he was not necessarily faulting payers for wanting to see more evidence to support broader coverage. “The evidence should support the coverage, and it should be appropriate research to support coverage. But when that evidence comes, it is fair that coverage should follow.”

Later at the same conference, former CMS Administrator Mark McClellan said FDA’s Sentinel project and existing innovative clinical networks could be good places to start on collaboration in the orphan drug area. He called Sentinel “a model that would be helpful in rare diseases in polling their efforts on registry building and not thinking you have to get all of the data individually, disease by disease, in one place” (Also see "Rare Disease Drugs Deserve Lower Cost Sharing In Part D, McClellan Says" - Pink Sheet, 29 Oct, 2012.).

As for manufacturers, Martin said they must do their part to help ensure continued access to these high-priced drugs, through things such as patient assistance programs and working with foundations to help get their products out.

For patients, mobilization and engagement is equally important. Panelist Dennis Jackman, senior VP of public affairs at CSL Behring recalled efforts by hemophilia patients in Delaware who mobilized to ensure access to products and services will be provided through plans in the state’s health insurance exchange being set up under the provisions of the Affordable Care Act.

Jackman noted that plans will cover things that are most important to hemophiliacs – access to specialists and federally recognized hemophilia treatment centers; a full range of FDA-approved products, including access to all the brands; access to a range of specialty pharmacy providers, so there is competition and choice; and ensuring medical necessity is defined by physicians in conjunction with the patients.

“This is right there in code, now, for the health exchanges,” Jackman said. “It didn’t happen by accident. I think it’s essential to replicate that amongst rare conditions as well.”