Specialty Medicines: Frequently Asked Questions
Introduction
Your doctor has prescribed a medicine that's considered a specialty medicine. This fact sheet will help you learn about specialty medicines and answer some questions you may have.
This fact sheet includes some words and phrases that may be new to you. These terms are highlighted in red and defined in the glossary on page 6.
What is a specialty medicine?
Specialty medicines typically have one or more of these features 1-6 :
- ӹ They treat serious and complex conditions, such as rheumatoid arthritis and cancer.
- ӹ They are also used to treat rare diseases, such as hemophilia and sickle cell disease.
- ӹ Many are biologic products. Biologic products are derived from a variety of natural sources (human, animal, or microorganism). They may be produced by biotechnology and other leading-edge technologies.
- ӹ They often need special storage and handling.
- ӹ Patients who use specialty medicines may require more education and monitoring from a health care provider than is needed with traditional medicines.
- ӹ Most cost more than traditional medicines.
- ӹ They are not commonly dispensed at local retail pharmacies. These pharmacies cannot typically provide the additional patient education or special storage and handling needed with specialty drugs.
Health plans may classify specialty medicines differently. 7 Check your health plan's formulary (drug list) to see what your plan considers a specialty medicine.
How do I take my specialty medicine?
Specialty medicine can be taken in various ways 6 :
By mouth
As an injection (a shot)
By infusion
Inhaled (breathed in)
If your medicine must be given to you with the help of a health care provider, it may be done at 1,2 :
Your home
Your doctor's office
A hospital
An outpatient facility (for example, an infusion center)
Specialty Medicines: Frequently Asked Questions
What diseases do specialty medicines treat?
The number of specialty drugs grew from less than 30 in the early 1990s to 400 in 2017. 8 New specialty medicines continue to be approved. About two-thirds of novel prescription medicines approved in 2019 were specialty drugs. 9
Until recently, specialty drugs were only available to treat serious and complex conditions, such as cancer and rheumatoid arthritis. Today, a wide variety of diseases and conditions are treated with specialty drugs. For instance, people with common health conditions, such as asthma and atopic dermatitis, can be helped by treatment with specialty drugs. 10,11
In addition, new medicines have been developed to treat serious rare diseases, such as sickle cell disease and hemophilia. 3,12,13 In some cases, a specialty drug may be the only treatment available for a life-threatening disease. 10
| Examples of Diseases and Conditions Treated by Specialty Medicines 3,10,11,13 : | |
|---|---|
| ӹ Asthma | ӹ Infertility |
| ӹ Atopic dermatitis | ӹ Migraine |
| ӹ Blood disorders, such as anemia, neutropenia, and thrombocytopenia | ӹ Multiple sclerosis |
| ӹ Cancer | ӹ Organ transplant |
| ӹ Crohn's disease | ӹ Osteoarthritis |
| ӹ Cystic fibrosis | ӹ Osteoporosis |
| ӹ Gaucher disease | ӹ Psoriasis |
| ӹ Growth hormone deficiency | ӹ Rheumatoid arthritis |
| ӹ Hemophilia | ӹ Sickle cell disease |
| ӹ Hepatitis | ӹ Ulcerative colitis |
| ӹ HIV/AIDS |
Specialty Medicines: Frequently Asked Questions Specialty Medicines: Frequently Asked Questions
How can specialty medicines help you?
Many specialty medicines are used to help manage chronic conditions over time. 10 For instance, drugs used to treat rheumatoid arthritis aim to reduce inflammation and pain and prevent joint damage. 14
Some specialty medicines can cure certain diseases. For example, new antiviral medicines treat hepatitis C, a type of liver disease. After eight to 12 weeks of treatment, about 90 percent of patients can be cured. 15
New types of specialty medicines, called gene therapies, hold promise to treat or halt the progression of some diseases. Some gene therapies fix or add working copies of the faulty gene inside the cells of a patient's body. 16 Other gene therapies enhance the immune system's ability to recognize and attack cells that are a threat. 17 Currently, only a small number of gene therapies have been approved by the U.S. Food and Drug Administration (FDA). Many more are being tested, and experts predict many different gene therapies may be available in coming years. 16
Why might I have to get testing before my doctor prescribes specialty medicine?
Before some specialty medicines are prescribed, certain tests may be required to help determine the therapy most likely to result in a favorable response. For instance, biomarker testing can help patients with cancer and their doctors determine the right treatment. Some cancer treatments will only work on patients whose cancer cells contain certain proteins, genes, or other molecules. 18
Where can I get my specialty medicine?
Many health plans require members to get specialty medicines filled by a specific specialty pharmacy. 19 In some cases, your specialty pharmacy may ship your medicine directly to you or to your doctor. In other cases, you may be able to pick up your specialty medicine at a local retail or hospital pharmacy. 19,20,21
In addition to filling your prescription, specialty pharmacies provide services to support you before and during treatment, including 1 :
- ӹ Helping you obtain insurance coverage for your medicine
- ӹ Training in how to use your medicine
- ӹ Providing ongoing monitoring and education
- ӹ Managing medicine dosing and side effects
- ӹ Coordinating your care with your doctor, your health plan and, in some cases, with the drugmaker 22
Specialty Medicines: Frequently Asked Questions Specialty Medicines: Frequently Asked Questions
Why do specialty medicines cost more?
There are many reasons why specialty medicines may cost more than traditional medicines. These could include 23,24 :
- ӹ They are often complicated to manufacture and administer.
- ӹ Handling and distributing these medicines can be complex and costly.
Your specialty medicine may be covered under your health plan's pharmacy or medical benefit 6,25 :
- ӹ If the medicine needs to be administered to you by a health care provider, it will likely be covered under your medical benefit.
- ӹ If you take or give the medicine yourself, it will likely be covered under your pharmacy benefit.
How much you pay out-of-pocket for specialty medicines will depend on your health plan's coverage. Many plans place drugs into different "tiers" on their formularies (drug lists). Each plan can divide its tiers in different ways. (See table below.)
The copayment or coinsurance that you pay will differ, depending on the tier. Generally, a drug in a lower tier will cost you less than a drug in a higher tier. 26,27
Most health plans put costly drugs, such as specialty drugs, in the highest—and most costly—tiers of their formularies. 27 Some specialty medicines may have generic or biosimilar versions available:
- ӹ A generic drug has the same active ingredient as a brand-name drug. 28
- ӹ A biosimilar is a biologic product that is highly similar to an FDA-approved biologic product. 29
Generic and biosimilar versions may cost you less out-of-pocket than brand-name drugs. 29,30,31
Health plans usually have other payment requirements as well. For instance, your drug costs may be higher until you have met your plan's deductible and may go down when you reach your out-of-pocket limit. 32
Example of Formulary Tiers 27
| Tier | What's Covered | What You Pay |
|---|---|---|
| 1 | Most generic prescription drugs | Lowest copayment |
| 2 | Preferred brand-name prescription drugs | Medium copayment |
| 3 | Nonpreferred brand-name prescription drugs | High copayment |
| 4 | Specialty drugs | Highest copayment or coinsurance Generic specialty drugs, biosimilars, and preferred specialty drugs may have lower copayments or coinsurance 30,31 |
Specialty Medicines: Frequently Asked Questions
What if I can't afford my specialty medicine?
If you need help paying for your medicine, you may be eligible for patient assistance programs. Some programs may ask that you meet medical or financial requirements. Most specialty pharmacies can connect you to these
NeedyMeds
needymeds.org/pap
Website with free information on programs that may help people who can't afford medicines and health care costs.
E-mail: info@needymeds.org
Phone: 1-800-503-6897
Medicine Assistance Tool
medicineassistancetool.org
Patients can search for potential financial assistance programs.
Phone: 571-350-8643
RxAssist
rxassist.org/patients
Resource center with an online database where you can search for patient assistance programs by drug name.
E-mail: info@rxassist.org
programs, which are offered by drugmakers and other organizations. 19,33
You might also want to find assistance on your own. Below are some organizations that can help you get started.
If you don't qualify for assistance, ask your doctor if there is a similar medicine covered by your health plan that costs less. 34
Specialty Medicines: Frequently Asked Questions
Glossary:
Antiviral: Prescription medicines that fight viruses in your body. 35
Biologic products (also known as biologics): Biologic products are derived from a variety of natural sources (human, animal, or microorganism). They may be produced by biotechnology and other leading-edge technologies. Biologics can be made of sugars, proteins, or nucleic acids, or of complex combinations of these substances. Biologics may also be living entities, such as tissues and cells. Types of biologic drugs include vaccines, blood and blood components, allergens, gene therapy, tissues, and proteins. 4,5
Biomarker testing (also known as genomic testing or tumor testing): Examining proteins, genes, or other markers in cancer cells to help determine the best treatment. 18
Biosimilar: A biologic product that is highly similar and has no clinically meaningful difference to an existing approved biologic drug. 29
Coinsurance: The percentage of the cost of a covered drug or health care service that you must pay. For instance, if a covered medicine costs $100 and you owe 20% coinsurance, then you would need to pay $20 and your health plan would pay $80. 28
Copayment: A fixed dollar amount you must pay for a covered medicine or health care service. 28
Deductible: The amount you must spend on covered medicines or health care services before your health plan begins to pay. For instance, if you have a $2,000 deductible, you would have to pay the first $2,000 of covered services yourself. After you pay your deductible, you usually pay only a copayment or coinsurance for covered medicines or services. Your health plan pays the rest. 28
Formulary: A list of drugs covered by your health plan. 28 Most health plans place drugs into different "tiers" on their formularies with each tier costing a different amount. Generally, a drug in a lower tier will cost you less than a drug in a higher tier. 27
Generic drug: Medicines that have the same active ingredient and are as safe and effective as an existing brand-name drug. These medicines usually cost less than the brand-name drug. 28
Gene therapy: According to the American Society of Gene + Cell Therapy, "Gene therapy is the introduction, removal or change in genetic material—specifically DNA or RNA—into the cells of a patient to treat a specific disease." 16
Infusion: When medication or fluids are administered through a needle, usually into a vein (intravenous infusion). 36
Out-of-pocket: The amount that you pay for health services and medicines that are not reimbursed by health insurance. These costs may include copayments, coinsurance, and deductibles. 28
Out-of-pocket limit: The most you have to pay for covered health care or medicines in a year. It may include deductibles, coinsurance, and copayments. Once the limit is reached, your health plan pays 100% of the costs. 28
Specialty Medicines: Frequently Asked Questions
Notes
References
- ASHP, ASHP Specialty Pharmacy Resource Guide. Published December 2015. Accessed June 2021. https://www.ashp. org/-/media/assets/pharmacy-practice/resource-centers/specialty-pharmacy/specialty-pharmacy-resource-guide.ashx 1.
- National Association of Specialty Pharmacy. NASP Definitions of Specialty Pharmacy and Specialty Medications. Published February 24, 2016. Accessed June 2021. https://naspnet.org/wp-content/uploads/2017/02/ NASP-Defintions-final-2.16.pdf 2.
- CVS Specialty. CVS Specialty Pharmacy Distribution Drug List. Published July 2021. Accessed July 2021. https://www.cvsspecialty.com/content/dam/enterprise/specialty/pdfs/SpecialtyDrugs.pdf 3.
- National Cancer Institute. NCI Dictionary of Cancer Terms. Accessed June 2021. https://www.cancer.gov/ publications/dictionaries/cancer-terms 4.
- U.S. Food and Drug Administration. What Are "Biologics" Questions and Answers. Published February 6, 2018. Accessed June 2021. https://www.fda.gov/about-fda/center-biologics-evaluation-and-research-cber/ what-are-biologics-questions-and-answers 5.
- Bunger A, Cline M, Holcomb K. Commercial Specialty Medication Research: 2019 Benchmark Projections. Milliman. Published December 2019. Accessed June 2021. https://www.milliman.com/-/media/milliman/ pdfs/articles/commercial_specialty_medication_-research_2019_benchmark_projections.ashx 6.
- AMCP. Specialty Pharmaceuticals. Published July 18, 2019. Accessed June 2021. https://www.amcp.org/ about/managed-care-pharmacy-101/concepts-managed-care-pharmacy/specialty-pharmaceuticals 7.
- CoverMyMeds. 2020 Medication Access Report. Accessed July 2021. https://www.covermymeds.com/ main/medication-access-report/ 8.
- Bangia I, Neumann U. Key Trends in U.S. Specialty Pharmacy. Certara. Published 2020. Accessed July 2021. https://www.certara.com/app/uploads/2021/01/Key-Trends-in-U.S.pdf 9.
- MacPhearson M, Clark K, Ghosh D, Rudoy J. Specialty Drugs: A Prescription for Managing Rising Cost and Care Needs. Published February 5, 2020. Accessed June 2021. https://health.oliverwyman.com/2020/02/ specialty-drugs--a-prescription-for-managing-rising-cost-and-car.html 10.
- Elsevier. Emerging Trends in the Specialty Drug Industry. Accessed June 2021. https://www.elsevier.com/clinicalsolutions/insights/resources/insights-articles/drug-information/emerging-trends-in-the-specialty-drug-industry 11.
- Vanscoy G, Koemer P, Faris, R. Rare Pharmacy Emerges from Specialty, Part 1. Pharmacy Times. Published July 30, 2020. Accessed July 2021. https://www.pharmacytimes.com/view/rare-pharmacy-emerges-from-specialty-part-1 12.
- IQVIA Institute. Orphan Drugs in the United States. Published December 2020. Accessed July 2021. https://www.iqvia.com/insights/the-iqvia-institute/reports/orphan-drugs-in-the-united-states-rare-diseaseinnovation-and-cost-trends-through-2019# 13.
- Arthritis Foundation. Rheumatoid Arthritis: Causes, Symptoms, Treatments and More. Accessed July 2021. https://www.arthritis.org/diseases/rheumatoid-arthritis 14.
- Schillie S, Wester C, Osbourne M, Wesolowski L, Ryerson AB. CDC Recommendations for Hepatitis C Screening Among Adults—United States, 2020. MMWR. Vol. 69, No. 2. Published April 10, 2020. Accessed July 2021. https://www.cdc.gov/mmwr/volumes/69/rr/rr6902a1.htm 15.
- American Society of Gene + Cell Therapy. Gene Therapy Basics. Updated July 26, 2021. Accessed September 21, 2021. https://patienteducation.asgct.org/gene-therapy-101/gene-therapy-basics 16.
- American Society of Gene + Cell Therapy. Blood Cancers and CAR T-Cell Therapy. Updated May 5, 2021. Accessed September 21, 2021. https://patienteducation.asgct.org/disease-treatments/car-t-basics 17.
continued
Specialty Medicines: Frequently Asked Questions
References continued
- National Cancer Institute. Biomarker Testing for Cancer Treatment. Published March 24, 2021. Accessed June 2021. https://www.cancer.gov/about-cancer/treatment/types/biomarker-testing-cancer-treatment#how-isbiomarker-testing-done 18.
- Patient Advocate Foundation. What's So Special About Specialty Pharmacies? Accessed June 2021. https://www.patientadvocate.org/wp-content/uploads/Specialty-Pharmacy.pdf 19.
- CVS Specialty. Medication Pick Up and Delivery. Accessed June 2021. https://www.cvsspecialty.com/ manage-prescriptions/medication-pickup-delivery.html#acc_uuid70aad480-d068-426c-bf3f-30e9c575731f 20.
- University of Iowa Hospitals & Clinics. Frequently Asked Questions About the Specialty Pharmacy. Accessed June 2021. https://uihc.org/frequently-asked-questions-about-specialty-pharmacy 21.
- Lindhorst J. How specialty pharmacy will thrive in the value era. Pharmacy Times. Published October 8, 2020. Accessed June 2021. https://www.pharmacytimes.com/view/how-specialty-pharmacy-will-thrive-in-the-value-era 22.
- Congressional Budget Office. Research and Development in the Pharmaceutical Industry. Published April 2021. Accessed June 2021. https://www.cbo.gov/publication/57126 23.
- Elsevier. Managing the Costs of Specialty Drugs. Published 2017. Accessed June 2021. https://www.elsevier.com/ clinical-solutions/insights/resources/insights-articles/drug-information/whitepapers/ managing-the-costs-of-specialty-drugs 24.
- Rice G. Navigating the specialty medication journey between pharmacy and medical benefits. Presented at 38th Annual Eastern Medicaid Pharmacy Administrators Association Meeting. Presented October 19-22, 2014. Accessed June 2021. http://www.empaa.org/downloads/EMPAA2014/presentation/RiceGary_Navigating_the_ Specialty_Medication_Journey.pdf 25.
- Kaiser Family Foundation. Employer Health Benefits: 2020 Annual Survey. Published October 8, 2020. Accessed June 2021. https://www.kff.org/health-costs/report/2020-employer-health-benefits-survey/ 26.
- Centers for Medicare and Medicaid Services. Your Guide to Medicare Prescription Drug Coverage. Revised September 2019. Accessed June 2021. https://www.medicare.gov/Pubs/pdf/ 11109-Your-Guide-to-Medicare-Prescrip-Drug-Cov.pdf 27.
- Healthcare.gov. Glossary. Accessed June 2021. https://www.healthcare.gov/glossary/ 28.
- U.S. Food and Drug Administration. Biosimilar and Interchangeable Products. Published October 23, 2017. Accessed June 2021. https://www.fda.gov/drugs/biosimilars/biosimilar-and-interchangeable-products 29.
- Blue Cross Blue Shield Blue Care Network of Michigan. How to Read a Drug List. Accessed June 2021. https://www.bcbsm.com/index/health-insurance-help/faqs/plan-types/pharmacy/read-drug-list.html 30.
- Chambers JD, et al. Coverage for biosimilars vs reference products among U.S. commercial health plans. JAMA. Vol. 323, No. 19. Published May 19, 2020. Accessed June 2021. https://jamanetwork.com/journals/ jama/fullarticle/2766151 31.
- Berman S, Giardino P. Understanding Cost Sharing, Deductibles, Copayments & Coinsurance. American Academy of Pediatrics. Published September 28, 2018. Accessed June 2021. https://www.healthychildren.org/English/family-life/ health-management/health-insurance/Pages/Understanding-Cost-Sharing-Deductibles-Copayments-Coinsurance.aspx 32.
- sPCMA. The Management of Specialty Drugs. Published February 8, 2016. Accessed June 2021. https://www.spcma. org/spcma-launches-website-and-releases-white-paper-the-management-of-specialty-drugs/ 33.
- Zand JM. Patient education: coping with high drug prices (beyond the basics). UptoDate. Updated March 10, 2020. Accessed June 2021. https://www.uptodate.com/contents/coping-with-high-drug-prices-beyond-the-basics 34.
- Centers for Disease Control and Infection. What You Should Know About Flu Antiviral Drugs. Updated June 1, 2020. Accessed June 2021. https://www.cdc.gov/flu/treatment/whatyoushould.htm 35.
- Doyle GR, McCutcheon JA. Clinical Procedures for Safer Patient Care. Chapter 7.5. Intravenous Medications by Direct IV Route. Published 2015. Accessed July 2021. https://opentextbc.ca/clinicalskills/ 36.
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