BlOG: Amplifying Patient Voices in Market Access: Real-World Cases

By: Henry Lee, Consultant
Published: June 2024

In the previous position piece (Amplifying Patient Voices in Market Access: Insights from DKI Health’s Patient and Caregiver Advisory Council), DKI Health received input from our Advisors regarding patients’ ongoing struggles to access their medicines, such as prior authorization, step therapy, and co-pay accumulators.

As patient voices in Market Access continue to rise, DKI Health identified several real-world cases in which patients demonstrate their influence in payers' coverage decisions in the US.

Case 1: Advocacy groups and professional societies prompt CVS Caremark to insert Eliquis back into its formulary.

Beth Waldron is a patient with venous thromboembolism (VTE) and an advocate at the National Blood Clot Alliance who has been stable on Eliquis for 8 years. In November 2021, Beth received a letter from CVS Caremark stating that Eliquis would be removed from its formulary starting January 2022. The alternative options given to her were Xarelto and Warfarin.¹

Though Waldron was temporarily switched to Xarelto, her HCP was able to help her get an exemption from CVS Caremark to remain on Eliquis.^1,2

Beth’s fear of losing her drug access again motivated her to advocate for other patients who were at risk of losing their anti-coagulant. She recognized that not every patient has the knowledge to advocate for themselves, so she started a social media campaign to fight against CVS Caremark’s policies on non-medical switching.^1,3

In part due to Beth’s advocacy work, within a month, 14 non-profit organizations (including the National Blood Clot Alliance) collaborated and urged CVS Caremark to reverse its decision on Eliquis, citing the undesired consequences of non-medical switching and the increased risk of stroke or death.^4,5

CVS Caremark’s decision regarding Eliquis also garnered attention from professional societies. The American College of Cardiology and the American Society of Hematology met with CVS Caremark to express their concerns with limited patient-physician medication choices and consequential increased administrative burden for HCPs.⁶

After objections from the patient and professional organizations, CVS Caremark renegotiated a new drug price with the drugmaker and eventually put Eliquis back on its formulary, starting in July 2022.⁷

-Beth Waldron, VTE Patient and Advocate 

This is about decisions between doctors and patients being respected. This is testimony to the power of collective action.

Case 2: The Colorado Prescription Drug Affordability Board's review of Trikafta shows the power of patient voice in drug access and its value to drugmakers.

In August 2023, the Colorado Prescription Drug Affordability Board (PDAB) selected 5 drugs for affordability review, including Trikafta, a drug developed by Vertex Pharmaceutical for cystic fibrosis (CF). If a drug is concluded unaffordable, the State government has the power to set upper payment limits for all insurers in the State.⁹

Intuitively, patients and patient organizations would applaud the decision to lower the drug price, but not in this case: Cystic Fibrosis Foundation (CCF) and nearly all CF patients who participated in the PDAB survey and two public hearings defended the affordability of Trikafta for Vertex, citing the importance of this life-changing medicine and the fear of losing it due to the price cap.¹⁰

The surprising reaction from patients reflects the close relationship between Vertex, CFF, and CF patients, which can be traced back more than two decades. In 1998, Vertex and CFF launched an R&D collaboration.¹¹ Up until 2016, CFF had funded Vertex $150M for drug development, including Trikafta, in exchange for sales royalties, which CFF sold for more than $3.8B by 2020.^12,13 In 2022, Vertex provided CFF $5.77M in annual grants and licensing, more than any other pharmaceutical company.¹⁴ In addition to the financial partnership, Vertex also closely works with CFF to help CF patients navigate its co-pay assistance program.¹⁵

The long-established relationship contributed to Vertex's victory in the affordability review. In December 2023, the Colorado PDAB voted unanimously not to cap Trikafta’s price. On the other hand, Enbrel, a drug costing less than 1/4 of Trikafta (average paid per person in 2022: $234,439 vs. $46,772), had a price cap imposed in February 2024.^10,16,17

Patients’ influence extends far beyond Colorado. As of Oct 2023, 10 States have boards tasked with assessing the affordability of drugs, and more States are introducing legislation to create PDABs.¹⁸ The victory in Colorado positions Vertex at an advantage in securing the price of Trikafta across the US.

-Catherine Harshbarger, Colorado PDAB

“The [public meeting for patients and caregivers]… was particularly raw; a lot of feelings were expressed by patients impacted by Trikafta. I can’t begin to tell you how touching those stories were, how impactful.”

Drug Affordability Review Process

Case 3: A student from Penn State University with severe ulcerative colitis fights denials against UnitedHealthcare, wins coverage, and exposes payer practices. ^20,21

Christopher McNaughton, a former college student-athlete, was diagnosed with severe ulcerative colitis (UC) in 2014. Failing to get meaningful relief from the drugs his doctors initially prescribed, he started to receive treatment from Dr. Edward Loftus Jr. at the Mayo Clinic, the best gastroenterology hospital in the US, after several referrals.

In 2018, McNaughton finally achieved remission, and his life improved drastically after Dr. Loftus prescribed him an unusual off-label use of a high-dose combination of two biological drugs: Remicade and Entyvio. At that time, his insurance had no issue to cover the combination treatment.

However, everything changed in July 2020 after McNaughton enrolled at Penn State University (PSU) and switched to the student plan provided by UnitedHealthcare (UHC). His coverage status went from “approved” to “pending” by September 2020 and from “pending” to “denial” in January 2021. Suddenly, the total outstanding bill for his treatment was $807,086.

McNaughton was fortunate enough to have his parents, who are PSU faculty members, help him appeal to PSU administrators and regain drug coverage. In March 2021, UHC agreed to pay for McNaughton’s bill until August 2021. Nevertheless, in June 2021, UHC informed him that, starting the next academic year, his drug would only be covered in significantly reduced doses, and that this decision was agreed upon by his doctor, Dr. Loftus, who had tried those drugs at a lower dose but failed, and titrated them up to help McNaughton achieve remission. 

McNaughton immediately spoke to Dr. Loftus and discovered that UHC misrepresented his doctor’s approval for the coverage decision. In August 2021, McNaughton filed a lawsuit against UHC, bringing other systematic issues regarding his claim review process to light.

In the claim review process, UHC conducted an internal review with its own doctor, who has not practiced medicine since the early 1990s. In his deposition, the UHC doctor said he just copied and pasted the nurse’s recommendation and typed “agree” on his review of McNaughton’s case. Additionally, UHC sought external opinions. It was discovered during the case that UHC selectively only collected opinions in favor of coverage denial and buried opinions that disagreed. In September 2021, UHC agreed to cover McNaughton’s treatment. 

In July 2023, McNaughton started a new chapter at PSU law school. He is now an advocate with the goal of becoming a health insurance attorney to represent patients who have had medical treatments denied by insurance companies.

-Christopher McNaughton, Law Student at Penn State University

I’m very passionate about patients having the rights to obtain the medical treatments they need, as this is an area where I have a lot of personal experience. I want to make a difference and help to change how people are treated by insurance companies.

Case 4: Co-pay accumulator policies get expanding oversight at both Federal and State levels, thanks to patient advocacy groups.

A “co-pay accumulator” is a tactic that payers leverage to block copay assistance from counting toward the patient’s deductible or out-of-pocket (OOP) maximum, and it leaves patients exposed to financial stress. Thanks to efforts from patient advocacy groups, the Federal and State governments realized the issues caused by co-pay accumulators and started to take legal action to restrict the use of co-pay accumulators.

In October 2023, the Federal court struck down the Health and Human Services (HHS) rule allowing co-pay accumulators on drugs that lack generic equivalents.²³ The case was brought by three patient groups, including the HIV+ Hepatitis Policy Institute, the Diabetes Patient Advocacy Coalition, and the Diabetes Leadership Council, and three patients dependent on co-pay assistance.²³ In addition, 29 patient groups jointly submitted an amicus brief to support the case.²³ Although HHS filed a notice of appeal, the case came to an end quickly when the U.S. Department of Justice dismissed HHS’s appeal in January.²⁴ Patient groups celebrated.

At the State level, as of June 2024, 19 States have banned co-pay accumulators in State-sponsored programs.²⁵ However, there is more work to be done. Patient advocacy groups, such as the Cystic Fibrosis Foundation and the National Bleeding Disorder Foundation, continue their policy advocacy to ban co-pay accumulators across the country by raising public awareness and educating policymakers at the regional level.^26,27

The slow growth of the prevalence of co-pay accumulators from 2019 to 2023 can be explained by the increasing regulatory scrutiny,²⁸ where patient advocacy groups have marked another victory in market access.

States Prohibiting Co-pay Accumulators

Arizona, Arkansas, Colorado, Connecticut, Delaware, Georgia, Illinois, Kentucky, Louisiana, Maine, New Mexico, New York, North Carolina, Oklahoma, Tennessee, Texas, Virginia, Washington, and West Virginia, as well as Puerto Rico

Conclusion

It is clear that patients’ influence on market access is growing. However, the broader patient and care partner community must still be educated, empowered, and equipped with resources to fight for access to the medications they need. Companies that want patients to stand by their side in market access should continuously invest in patient engagement, listen to patients' access challenges, and provide patients with the necessary support.    

References:

1. Waldron B. Nonmedical switching of anticoagulants: The patient impact when formulary exclusions limit drug choice. Research and Practice in Thrombosis and Haemostasis. 2022;6(2):e12675. doi:https://doi.org/10.1002/rth2.12675

2. PBM Exclusions Increasing, Finds New Analysis. Formulary Watch. Published June 7, 2022. https://www.formularywatch.com/view/pbm-exclusions-increasing-finds-new-analysis

3. How a Formulary Change and a Few Tweets Led to a HIPAA Complaint. Managed Healthcare Executive. Published April 20, 2022. https://www.managedhealthcareexecutive.com/view/how-a-formulary-change-and-a-few-tweets-led-to-a-hipaa-complaint

4. Urgent Patient Advocacy Update from the National Blood Clot Alliance. Blood Clots. Published December 9, 2021. https://www.stoptheclot.org/news/urgent-patient-advocacy-update-from-the-national-blood-clot-alliance/

5. Brennan T. Partnership to Advance Cardiovascular Health. Published December 2, 2021. https://static1.squarespace.com/static/56e6efdb5559866c54beb696/t/61b3b6a6916aab0fc1bee793/1639167659313/CVS+Switch+Letter+Final.pdf

6. ACC, ASH Meet With CVS Caremark on New DOAC Formulary Change. American College of Cardiology. https://www.acc.org/Latest-in-Cardiology/Articles/2022/01/13/14/15/ACC-ASH-Meet-With-CVS-Caremark-on-New-DOAC-Formulary-Change

7. CVS Caremark Puts Eliquis Back On Its Formularies. Managed Healthcare Executive. Published June 24, 2022. https://www.managedhealthcareexecutive.com/view/cvs-caremark-puts-eliquis-back-on-its-formularies

8. Waldron B. (2022) Experience with non-medical switching at Cardiovascular Health Policy Summit. https://www.linkedin.com/in/beth-waldron-1616797/recent-activity/all/

9. Reid S. STATEMENT: Colorado Becomes Third State With Prescription Drug Affordability Board. Patients For Affordable Drugs Now. Published June 16, 2021. https://patientsforaffordabledrugsnow.org/2021/06/16/colorado-third-state-pdab/

10. Final Public Trikafta Affordability Review Report 2023

11. Vertex and Cystic Fibrosis Foundation Therapeutics to Collaborate on Discovery and Development of New Medicines to Treat the Underlying Cause of Cystic Fibrosis | Vertex Pharmaceuticals. Vertex Pharmaceuticals. Published 2024. https://investors.vrtx.com/news-releases/news-release-details/vertex-and-cystic-fibrosis-foundation-therapeutics-collaborate

12. Vertex snares $75M windfall in revised CFF deal; Ex-Celgene R&D prez Daniel starts advisory group. Endpoints News. https://endpts.com/vertex-snares-75m-windfall-in-revised-cff-deal-ex-celgene-rd-prez-daniel-starts-advisory-group/

13. A Message From Our CEO | Cystic Fibrosis Foundation. www.cff.org. Published November 2, 2020. https://www.cff.org/node/871

14. Industry Contributions | Cystic Fibrosis Foundation. www.cff.org. https://www.cff.org/about-us/industry-contributions

15. Vertex Copay Assistance: Tips from a CF Pharmacist | Cystic Fibrosis Foundation. www.cff.org. https://www.cff.org/community-posts/2022-11/vertex-copay-assistance-tips-cf-pharmacist

16. Final Public Enbrel Affordability Review Report 2023

17. Colorado moves to cap price of arthritis drug Enbrel in first-in-the-nation action by state affordability board. The Denver Post. Published February 23, 2024. https://www.denverpost.com/2024/02/23/enbrel-price-cap-colorado-prescription-drug-affordability-board/

18. What You Need to Know: Prescription Drug Affordability Boards and People with CF | Cystic Fibrosis Foundation. www.cff.org. https://www.cff.org/about-us/what-you-need-know-prescription-drug-affordability-boards-and-people-cf

19. Colorado.gov: Colorado Prescription Drug Affordability Review Board & Advisory Council. https://doi.colorado.gov/insurance-products/health-insurance/prescription-drug-affordability-review-board. 2024

20. Miller DAR. UnitedHealthcare tried to deny coverage to a chronically ill patient. He fought back, exposing the insurer’s inner workings. ProPublica. Published February 2, 2023. https://www.propublica.org/article/unitedhealth-healthcare-insurance-denial-ulcerative-colitis

21. LinkedIn. Christopher McNaughton. http://linkedin.com/in/chris-mcnaughton-005991285/Court strikes down HHS rule that allowed insurers to not count copay assistance. HIV+Hepatitis Policy Institute. https://hivhep.org/press-releases/court-strikes-down-hhs-rule-that-allowed-insurers-to-not-count-copay-assistance/

22. Court strikes down HHS rule that allowed insurers to not count copay assistance. HIV+Hepatitis Policy Institute. https://hivhep.org/press-releases/court-strikes-down-hhs-rule-that-allowed-insurers-to-not-count-copay-assistance/

23. UnitedHealthcare Tried to Deny Coverage to a Chronically Ill Patient. He Fought Back, Exposing the Insurer’s Inner Workings. Christopher McNaughton sits on the campus of Penn State University. He has been battling United Healthcare for coverage of his treatment for ulcerative colitis. Credit: Nate Smallwood, special to ProPublica. Published February 2, 2023. https://www.propublica.org/article/unitedhealth-healthcare-insurance-denial-ulcerative-colitis

24. Maas A. Copay Accumulator Lawsuit Comes to an End; Will Ruling Be Enforced? MMITNetwork. Published February 15, 2024. Accessed June 1, 2024. https://www.mmitnetwork.com/aishealth/spotlight-on-market-access/copay-accumulator-lawsuit-comes-to-an-end-will-ruling-be-enforced-2/

25. 2024 Copay Accumulator & Maximizer Programs | Crohn’s & Colitis Foundation. www.crohnscolitisfoundation.org. https://www.crohnscolitisfoundation.org/patientsandcaregivers/managing-the-cost-of-ibd/copay-accumulator-maximizer-programs#:~:text=As%20of%20summer%202023%2C%2019

26. Government Relations Update - March 2023 | NBDF. National Bleeding Disorders Foundation. Published April 4, 2023. https://www.bleeding.org/news/government-relations-update-march-2023

27. CF Foundation Supports California’s Efforts to Ban Co-Pay Accumulators and Maximizers | Cystic Fibrosis Foundation. www.cff.org. https://www.cff.org/statements/2024-03/california--ban-accumulators-maximizers

28. 2023 Update: Six Years of Deductible Accumulators and Copay Maximizers. www.iqvia.com. https://www.iqvia.com/locations/united-states/blogs/2024/03/2023-update-six-years-of-deductible-accumulators-and-copay-maximizers