Thoughts from the NLA Presidents: A Five Year Perspective

I was asked to write a perspective on a look back of the past five years on the NLA and clinical lipidology. It is impossible to state all the advances made in clinical lipidology and the contributions our organization has made over the past five years in a short and focused perspective. Therefore, I decided to constrain my perspective to those advancements made in one of the highest risk populations, familial hypercholesterolemia (FH)-not surprising for those who know me well. Our organization produced the first US practice-based recommendations for FH in June of 2011.1  Okay, this is not in the last five years, but I would be remiss if I did not mention them. This document was vital in providing much needed guidance on screening, diagnosis, and treatment of US adults and children with FH. We also identified gaps in our understanding of FH, which provided a platform for further research.

In late 2012 and early 2013,2,3 the first pharmacologic agents were approved for use in patients with homozygous FH. Lomitapide and mipomersen offered these patients the first effective drugs to lower atherogenic lipoproteins in the 25 to 60% range for the first time, offering these high-risk patients an option to or in combination with low-density lipoprotein (LDL) apheresis.

Late in 2013, the American College of Cardiology/American Heart Association published their much-anticipated guidelines,4 which were clouded by controversies that I won’t completely rehash here. These guidelines identified four groups of patients most likely to benefit from statin therapy. One of these groups were those individuals most likely to have FH (baseline LDL cholesterol ≥ 190 mg/dL). Lipid goals were largely abandoned and non-statin therapies were not strongly endorsed unless shown to reduce cardiovascular (CV) events in randomized controlled trials-the type of trials that would be unethical to conduct in patients with FH.

In 2014, we produced our first Recommendations for Patient-Centered Management of Dyslipidemia also known as Part 1.5 Our recommendations were consistent with previous guidance given for FH patients1 and reaffirmed lipid goal-based therapy. I feel this was a trajectory point back to lipid goal-based therapy.6,7 The NLA in collaboration with the FH Foundation submitted a proposal to the National Center for Health Statistics (NCHS), the Federal agency responsible for revising the ICD codes, to create new, specific ICD-10 codes for FH. These codes were approved in 2016, and we hope will have a profound impact on the diagnosis, treatment, and research of FH going forward.

Probably the most important advancement made in the treatment of patients with FH since 1987 when the first statin was approved by the FDA,8 was the approval of alirocumab9 and evolocumab10 in 2015. These fully human monoclonal antibody (mAbs) medications when administered with maximally tolerated statin therapy reduced LDL cholesterol to levels a majority of lipidologists and FH patients only dreamed about.  The benefit of these mAb-based therapies on reduction in CV events is now becoming apparent,11 and the next step our organization and others involved in the care of FH patients is to improve access.

Too many insurance companies and pharmacy benefit managers, protective of their bottom line, find too many creative ways to restrict coverage despite the effectiveness of these new drugs. Let’s make it a priority to ensure true access to these innovative medicines.

References

1. Goldberg AC, Hopkins PN, Toth PP, Ballantyne CM, Rader DJ, Robinson JG, Daniels SR, Gidding SS, de Ferranti SD, Ito MK, McGowan MP, Moriarty PM, Cromwell WC, Ross JL, Ziajka PE; National Lipid Association Expert Panel on Familial Hypercholesterolemia. Familial hypercholesterolemia: screening, diagnosis and management of pediatric and adult patients: clinical guidance from the National Lipid Association Expert Panel on Familial Hypercholesterolemia. J Clin Lipidol. 2011 Jun;5(3 Suppl):S1-8.

2. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm333285.htm (accessed on March 18, 2017)

3. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm337195.htm (accessed on March 18, 2017)

4. Stone NJ Robinson JG Lichtenstein AH Bairey Merz CN Blum CB Eckel RH Goldberg AC Gordon D Levy D Lloyd-Jones DM McBride P Schwartz JS Shero ST Smith SCJr Watson K Wilson PWF 2013 ACC/AHA guideline on the treatment of blood cholesterol to reduce atherosclerotic cardiovascular risk in adults: a report of the American College of Cardiology/American Heart Association Task Force on Practice Guidelines. J Am Coll Cardiol  2014;63(25, Part B):2889–2934.

5. Jacobson TA, Ito MK, Maki KC, Orringer CE, Bays HE, Jones PH, McKenney JM, Grundy SM, Gill EA, Wild RA, Wilson DP, Brown WV. National Lipid Association recommendations for patient-centered management of dyslipidemia: part 1 - executive summary. J Clin Lipidol. 2014 Sep-Oct;8(5):473-88.

6. Lloyd-Jones DM, Morris PB, Ballantyne CM, Birtcher KK, Daly DD Jr, DePalma SM, Minissian MB, Orringer CE, Smith SC Jr. 2016 ACC Expert Consensus Decision Pathway on the Role of Non-Statin Therapies for LDL-Cholesterol Lowering in the Management of Atherosclerotic Cardiovascular Disease Risk: A Report of the American College of Cardiology Task Force on Clinical Expert Consensus Documents. J Am Coll Cardiol. 2016 Jul 5;68(1):92-125. doi: 10.1016/j. jacc.2016.03.519.

7. Jellinger PS, Handelsman Y, Rosenblit PD, Bloomgarden ZT, Fonseca VA, Garber AJ, Grunberger G, Guerin CK, Bell DS, Mechanick JI, Pessah-Pollack R, Wyne K, Smith D, Brinton EA, Fazio S, Davidson M. American Association of Clinical Endocrinologists and American College of Endocrinology Guidelines for Management of Dyslipidemia and prevention of Atherosclerosis EXECUTIVE SUMMARY. Endocr Pract. 2017 Feb 3. doi: 10.4158/EP171764.GL. [Epub ahead of print]

8. Endo A. Proc Jpn Acad Ser B Phys Biol Sci. 2010 May 11; 86(5): 484–493.

9. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm455883.htm (accessed on March 18, 2017)

10. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm460082.htm (accessed on March 18, 2017)

11. Sabatine MS, Giugliano RP, Keech AC, Honarpour N, Wiviott SD, Murphy SA, Kuder JF, Wang H, Liu T, Wasserman SM, Sever PS, Pedersen TR; FOURIER Steering Committee and Investigators. Evolocumab and Clinical Outcomes in Patients with Cardiovascular Disease. N Engl J Med. 2017 Mar 17. doi: 10.1056/NEJMoa1615664.