Panelists for a session at the Asembia 2021 Specialty Pharmacy Summit in Las Vegas discussed considerations for manufacturers, specialty pharmacies and payers entering the rare disease and gene therapy market.
Panelists at the session “Rare Diseases and Gene Therapies: Considerations for Specialty Pharmacy”, presented at the 2021 Asembia Specialty Pharmacy Summit in Las Vegas, discussed considerations to take into account when entering the rare disease market and gene therapy from a manufacturer’s point of view in partnership with specialist pharmacies.
Nick Calla, Senior Vice President, Industrial Relations, Orsini Specialty Pharmacy, opened the discussion by highlighting the treatment journey experienced by rare disease patients, in which only 10% of the 7,000 rare diseases known today have a identified treatment. In particular, children represent 50% of cases of rare diseases.
“The focus on how gene therapy can help patients with rare diseases is increasing over time,” he said. “You see it both from the point of view of the smaller biotechs and the larger pharmaceutical manufacturers that are going into the mix. “
With many large manufacturers partnering or acquiring smaller companies to access these types of products, there are several unique challenges for these manufacturers entering the market, Calla noted. In particular, it is about managing the large investment required to develop these therapies, reach these very small patient populations and prove their effectiveness to the FDA for approval.
“The manufacturers are working very close to the patient through their field reimbursement teams, through their case managers,” Calla said. “And the specialty pharmacies that then work with the manufacturers have to have a very strong communication, a very close connection… It’s a different dynamic than what we’ve seen in the traditional pharmacy, manufacturer relationship.
As communication plays a key role in improving market access for new rare disease treatments, panelist Dave Frobel, Senior Vice President, Commercial Relations, Orsini Specialty Pharmacy, explained in more detail what involves an effective relationship between a manufacturer and a specialty pharmacy.
A 2018 survey that asked manufacturers for their top reasons for choosing their particular specialty pharmacy found that one in three people identified access to payers and relationships with payers as the top reason.
In addition, more than half of the manufacturers indicated that they wanted improved services at all levels, especially with regard to communication with patients, better responsiveness in the delivery of their products to patients and more data on trends in the patient populations they serve.
“Another part of the survey was about hub services… What the survey found was that 83% of manufacturers said there was room for improvement and 17% said that they were not at all satisfied, ”Frobel added. “So what that tells me is that this kind of standard model for hub services may not fit well with rare diseases, and we may have to customize it in some way. “
Seeing this as an opportunity for specialist pharmacies to adapt their services to these needs, Frobel underlined several considerations for these entities:
- Establish customizable agreements and close working relationships with payers
- Offer a Letter of Understanding or Single Case Agreement
- Be able to build both the medical and pharmaceutical advantage
- Be able to help healthcare providers navigate prior authorizations, appeals and letters of medical necessity
- Have registered nurses trained and experienced in the administration of home infusions, in pediatric populations, and with specific training for the particular drug
Since these cell and gene therapies require multiple storage capacities, such as ultra-low temperature freezer storage, Frobel said it was also critical to meet these needs by delivering to doctors’ offices, outpatient hospitals and in patients’ homes.
“If it’s a weekly infusion, it’s pretty important to have a quick turnaround time to make sure everything is in place by the time they get their last clinical infusion, and you can fill it out. ‘nurse for the next week without missing any infusions,’ he said.
Although cell and gene therapies are often grouped together, the distinction between these 2 options is important in understanding the safety and risk posed to these patient populations. Frobel explained that gene therapy involves altering a person’s genes by replacing the missing gene, inactivating a gene that is not working properly, or by regulating a dysfunctional gene.
“There have been a lot of security concerns about this – probably a big part is that we don’t know what we know, but there are a few key considerations to keep in mind,” he said. . “One is that patients can have an immune response to the viral vector… The other thing that can be worrying is whether this gene therapy is being given to non-target cells and what it might cause, and I think that that’s where there is a lot of investigation and close scrutiny by the FDA. “
Involving the transfer of living cells that perform a desired function for the patient, cell therapies such as chimeric antigen receptor T cell therapy have shown promise in several disease areas, but have been shown to have significant toxicity for patients, which may justify an increase in research on the patients who best tolerate these treatments.
Speaking about the future of cell and gene therapies, Calla said that there are currently thousands of gene therapies in development for oncology (n = 1048) and rare diseases (n = 869) than FDA is watching closely.
The role of the payer in managing the high initial cost of these therapies was also cited as important factors to watch out for, especially with regard to special contractual arrangements, value-based contracts with manufacturers and share of costs. higher for patients.
“At the end of the day, what we’re really looking at is the patient,” Calla concluded. “We want to give the patient hope that there is treatment potential for one of his rare diseases. We want to be able to collaborate with the patient and his family. And we obviously want to continue the advocacy for early access to these products and for the FDA to approve more of these products. “