The Multiple Myeloma Treatment Pipeline and Specialty Pharmacy

Pharmacy Practice in Focus: OncologyJune 2021
Volume 3
Issue 3
Pages: 12

The American Cancer Society predicts nearly 35,000 Americans will receive a diagnosis of multiple myeloma (MM) in 2021.1 Dealing with a cancer diagnosis is difficult, but patients with MM have reason to be hopeful. Survival rates are on the rise, and a robust pipeline of promising immunotherapies and novel treatment options are in development.2 Specialty pharmacies will play a critical role as new treatments for MM are approved.

Facilitating Medication Access

Specialty pharmacies help patients navigate access to medications that may not be carried by community pharmacies, such as high-cost therapies and those indicated for rare conditions. Although a host of new therapeutics for MM are approaching regulatory approval, this does not guarantee broad access or immediate health plan coverage. One study found that 3 years after prescription coverage for novel therapeutics had been incorporated into health plans, access remained restricted by prior authorization, step therapy, or other measures.3

Another recent report describes how patients were left to coordinate access to specialty medication on their own, spending 3 hours or more on the phone trying to obtain their prescribed therapy. Over half waited more than a week to receive their first dose of medication, while many also faced financial barriers that contributed to high prescription abandonment rates.4

“Today we are fortunate to have groundbreaking MM treatments with fantastic success rates and fewer [adverse] effects. These modern advancements often come with a high price tag and a complicated path to therapy initiation. Before granting approval, payers require navigating some combination of prior authorization and step therapy, as well as potential denials of authorization and appeal processes. Even after insurance approval, increased cost-sharing can create substantial financial burden, while limited distribution arrangements may confuse patients accustomed to the traditional retail prescription process,” said Joshua Stoneking, PharmD, vice president, oncology, at BioMatrix Specialty Pharmacy.

Access challenges are often compounded in MM because of treatment protocols requiring multiple lines of ongoing therapy.5 Timely medication access is especially important for patients receiving oncologic therapies, as delays have been linked to worsened survival rates.6

Results from a recent study revealed several factors influencing timeliness of therapy initiation for patients with MM, including age, insurance coverage, type of cancer center providing care, geographic location, and Charlson Comorbidity Index score. In some cases, uninsured patients were able to initiate therapy faster than their insured counterparts. Additionally, the study report described disparities affecting therapy initiation for women and Black patients.7

Specialty pharmacies are addressing medication access challenges. Understanding the importance of prompt therapy initiation, specialty pharmacies are increasingly focused on time-to-first and -subsequent fill metrics. Some specialty pharmacies are deploying additional support staff and resources to provide dedicated, concierge-level services to help patients understand their coverage and initiate prescribed therapy quickly. These staff are often highly experienced with specific categories of specialty drugs and focus on providing prior authorization support, connection to financial assistance programs, and coordination of resources aimed at reducing barriers to care.

“Specialty pharmacies are designed to facilitate timely access to prescribed therapy,” Stoneking said. “A good specialty pharmacy will offer a dedicated team experienced with the prior authorization and [patient] onboarding processes for specific therapies. Additionally, specialty pharmacies with strong manufacturer relationships and [limited distribution drug] partnerships can promote continuity by treating patients through multiple lines of MM therapy. As new therapies are approved, specialty pharmacies offering comprehensive, therapy-specific support will speed access to therapy and promote optimal outcomes.”

Coordinating Clinical Support

Effective treatment of MM requires significant clinical coordination, as MM treatment plans are highly individualized. Treatment is influenced by many factors, including patient demographics, biomarkers, level of disease progression, response to previous treatments, individual treatment goals, and the preferred treatment paradigms of individual practitioners.8 Treatment often includes a combination of drugs that vary throughout the patient’s therapeutic journey. Many of these drugs produce significant adverse effects that require ancillary therapies to manage.

Results from a recent study showed oncology-focused pharmacists improved clinical outcomes for patients and financial outcomes for payers. These outcomes were directly influenced by individual patient consults and pharmacist-led interventions focused on educating patients about their regimen, recommending dose adjustments, and conducting medication reconciliation.9 Effective specialty pharmacists help patients anticipate and mitigate treatment challenges while providing ongoing resources and support to promote optimal therapeutic outcomes. They also provide resources and tools to help patients adhere to therapy and monitor their treatment progress.

“Many of the MM products in development are improvements to current therapies. Our role as pharmacists will continue to be helping patients initiate therapy quickly, safely, and with resources to maintain adherence, identify adverse events, and minimize [adverse] effects. Clinical pharmacists familiar with both established and enhanced lines of therapy will be well prepared to provide the highly individualized care supporting positive outcomes,” said Kionna Oleru, PharmD, a pharmacy manager at BioMatrix Specialty Pharmacy and Next-Generation Pharmacist® award winner who has focused much of her career in the oncology space.

Advancing Research

Specialty pharmacies are using data to contribute to research advancing the understanding of new therapeutics. In 2016, Congress passed the 21st Century Cures Act, placing additional focus on real-world data (RWD) and real-world evidence (RWE) to support regulatory decision-making for drugs following their FDA approval.10 Specialty pharmacies track a large amount of RWD related to therapy, including utilization, adverse events, efficacy, quality of life, overall treatment outcomes, and more. Focus on RWD and RWE comes as an increasing number of patients are using wearables and other digital tools to track and manage their health.

As health information technology (HIT) use expands, many patients are willing to share their data to contribute to research initiatives.11 This trend and the proven efficacy of digital health interventions has prompted specialty pharmacies to incorporate HIT as a key part of their medication therapy management (MTM) programs.12 The data collected by way of HIT-optimized MTM has expanded the amount and quality of RWD specialty pharmacies can access and analyze to advance the understanding of new drugs.

“The RWD that specialty pharmacies collect can significantly contribute to the advancement of research and understanding of new therapies. By analyzing and translating RWD into RWE specific to individual patients [with MM] and the greater MM community, such RWE can be used to improve and optimize care of those with MM,” said Royce Burruss, RPh, MBA, FASCP, corporate director, clinical services, for BioMatrix Specialty Pharmacy, and author of peer-reviewed pharmacy operations research.

As new therapies in the MM pipeline are approved, specialty pharmacies will use RWD to provide research and analysis informing future clinical, regulatory, and payer-driven initiatives.

A Promising Future

The American Society of Hematology hosted its annual meeting in December 2020. In a postmeeting poll, hematologists indicated new data around MM as being the most significant and practice changing.13 Physicians, patients, pharmacists, and other professionals involved in the treatment and management of MM are looking forward to more positive developments on the horizon.

Stoneking, Oleru, and Burruss agreed that the future for patients with MM is promising.

“There is such a high level of excitement around new ways to tackle MM,” Stoneking noted. “I have never seen more trials in process. The goal is curative therapy, and it is absolutely amazing to see the progress toward that end.”

“There is a true sense of collaboration among a diverse group of health care stakeholders, with everyone focused on success for the patient. The sheer number of treatment options in development is incredibly exciting for everyone involved with MM,” Oleru added.

“An increasing number of pharmacotherapy options and care delivery adjuncts, such as mobile HIT applications, will decrease disease burden and improve quality of life. Progress in precision medicine is also very exciting, as it provides solutions for [patients with MM] with unmet needs, including those at risk for early disease progression or who are refractory to current therapies,” said Burruss.

As new MM drugs gain regulatory approval, specialty pharmacies will be at the forefront facilitating medication access, coordinating clinical support, and advancing ongoing research to promote the best possible outcomes for patients with MM.


  1. Key statistics about multiple myeloma. American Cancer Society. Updated January 12, 2021. Accessed May 10, 2021.
  2. Understanding survival statistics. Multiple Myeloma Research Foundation. Accessed May 10, 2021.
  3. Shaw DL, Dhruva SS, Ross JS. Coverage of novel therapeutic agents by Medicare prescription drug plans following FDA approval. J Manag Care Spec Pharm. 2018;24(12):1230-1238. doi: 10.18553/jmcp.2018.24.12.1230
  4. Cover My Meds. 2021 Medication Access Report. Accessed May 10, 2021.
  5. Rajkumar SV, Harousseau JL. Next-generation multiple myeloma treatment: a pharmacoeconomic perspective. Blood. 2016;128(24):2757-2764. doi:10.1182/blood-2016-09-692947
  6. Khorana AA, Tullio K, Elson P, et al. Increase in time to initiating cancer therapy and association with worsened survival in curative settings: a U.S. analysis of common solid tumors. J Clin Oncol. 2017;35(suppl 15):6557. doi:10.1200/JCO.2017.35.15_suppl.6557
  7. Kumar V, Alhaj-Moustafa M, Bojanini L, et al. Timeliness of initial therapy in multiple myeloma: trends and factors affecting patient care. JCO Oncol Pract. 2020;16(4):e341-e349. doi:10.1200/JOP.19.00309
  8. Treatment guides. Multiple Myeloma Research Foundation. Accessed May 10, 2021.
  9. Virani A, Schlei Z, Gleason C, et al. Impact of an oncology clinical pharmacist specialist in an outpatient multiple myeloma clinic. Clin Lymphoma Myeloma Leuk. 2020;20(9):e543-e546. doi:10.1016/j.clml.2020.04.012
  10. Real-world evidence. US Food and Drug Administration. November 30, 2020. Accessed May 10, 2021.
  11. Kim J, Kim H, Bell E, et al. Patient Perspectives About Decisions to Share Medical Data and Biospecimens for Research. JAMA Netw Open. 2019;2(8):e199550. doi:10.1001/jamanetworkopen.2019.9550
  12. Stranz M, Lindhorst J. Opportunities and trends in digital health. Specialty Pharmacy Times®. June 18, 2020. Accessed May 10, 2021.
  13. Ternyila D. 2020 ASH recap: hematologists review most impactful data across hematologic malignancies. Targeted Oncology. December 16, 2020. Accessed May 10, 2021.

Related Videos
cancer pain management | Image Credits: © Burlingham -
multiple myeloma clinical trial daratumumab/ Image Credits: © Dragana Gordic -
multiple myeloma clinical trial/Image Credits: © Studio Romantic -
3d rendered illustration of lung cancer 3D illustration - Image credit:  appledesign |
pharmacy oncology, Image Credit: © Konstantin Yuganov -
male pharmacist using digital tablet during inventory in pharmacy | Image Credit: sofiko14 -
Pharmacist holding medicine box in pharmacy drugstore. | Image Credit: I Viewfinder -
© 2024 MJH Life Sciences

All rights reserved.