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Operational Challenges Payors Face with Rare Disease Limited Distribution Drugs

Often when pharmacy benefit manager (PBM) agreements and specialty pharmacy agreements are put into place, no one considers the 1 or 2 percent of the overall population that is made up of rare disease patients. As a result, some health plans look to non-PBM-owned specialty pharmacies, such as independent rare disease specialty pharmacies, to help with the operational support and effective strategies to care for these rare disease patients.

You have to have a plan for every drug as you want to provide patients with access to care as fast as possible. From a contracting perspective, you want to prevent “holes” in the contract.” – Dino Conti, PharmD, MBA, Chief Pharmacy Officer, Health Plan Pharmacy Consulting, Solid Benefit Guidance, an Arthur J Gallagher Company.

To help identify these “holes” in the contracting process, health plans can ask specific questions to PBM-owned specialty pharmacies, such as:

  • Do you currently have access to X,Y, and Z rare disease drugs?
  • Which drugs have been identified as limited distribution drugs?
  • Do you have a separate contract to access rare disease drugs?
  • Are the aggregate guarantees different for limited distribution drugs you can access versus limited distribution drugs you can’t access?

Answers to these questions can help a health plan add a level of protection by creating a separate contract with the PBM-owned specialty pharmacies for the rare disease limited-distribution drugs they don’t have access to. With a robust rare disease drug pipeline and continued blurred lines between medical and pharmacy benefit designations, it is imperative that coordination with other specialty pharmacies with access to rare disease drugs and their services be seamless to the employer, to the health plan, and most importantly, to the patient.

Rare disease specialty pharmacies can play a more proactive role in providing this seamless coordination by educating payors on the complexities and subtleties of managing a rare disease patient population by focusing on topics like:

  • Therapeutic landscape launches: a novel therapy vs. a new competitor drug
  • Rare disease and drug information
  • Site of care considerations
  • Patient selection processes
  • Rare disease drug delivery nuances

Another way to ensure seamless coordination is through a special handling unit.

We see a lot of value in these special handling teams to help with rare disease patients, especially with unanticipated issues.” – Chandra Wahrgren, MBA, SVP, Corporate Pharmacy Enterprise & Innovation, Moda Partners

As mentioned in recent articles, patients with rare diseases undergo an arduous, time-consuming process to get an accurate diagnosis. The last thing anyone wants in the healthcare delivery system is for this type of patient to fall through the cracks due to operational challenges in accessing a rare disease drug.

These operational challenges require payors to collaborate closely with healthcare providers, pharmaceutical manufacturers, patient advocacy groups, and regulatory bodies to address the unique considerations associated with rare disease limited distribution drugs to ensure equitable access to these therapies. Payors can minimize these operational challenges by having a line of sight on the rare disease pipeline. When a new drug comes to market, there are early adopter physicians who will want to prescribe it, especially if it is a rare disease drug that can help one of their specific patients. One of the best resources for keeping up with the rare disease pipeline is your rare disease specialty pharmacy partner.

Click here to download our whitepaper detailing what payors should consider when evaluating a specialty pharmacy partner.