Los Angeles PFS - Friday Slide Deck by International Myeloma Foundation

2024 Los Angeles Patient and Family Seminar

August 16-17, 2024

THANK YOU TO OUR SPONSORS!

What do the dots mean?

More than 1 year since diagnosis

Stem cell transplant recipient

Less than 1 year diagnosed

Care Partner for someone with Myeloma

Parking Reimbursement

Hotel Guests: Please put parking on your room, and the IMF will cover this directly with the hotel.

Meeting Attendees: Please See Meghan at the registration desk for a voucher.

Local Support Groups: You

Are Not Alone!

 Myeloma Stompers

(Napa/Sonoma)

Meets virtually the 2nd Friday of each month at 10AM  San Francisco Bay Area Myeloma Support Group

 San Gabriel Valley Myeloma Support Group

Meets in-person the 1st Monday of each month at 6:30PM

 Upland, CA Myeloma Support Group

Meets hybrid the 1st Friday of each month at 10AM

Meets virtually the 3rd Saturday of each month at 10AM

 Westlake Myeloma Support Group

Meets virtually the 2nd Saturday of each month at 11AM

 San Fernando Valley Myeloma Support Group

Meets in-person the 3rd Wednesday of each month at 7PM

 Sacramento Area Myeloma Support Group

Meets virtually the 1st Saturday of each month at 10AM

Local Support Groups: You

Are Not Alone!

 San Diego Multiple Myeloma Support Group

Meets hybrid the 2nd Monday of each month at 6:30PM

 Inland Empire, CA

Myeloma Support Group

Meets hybrid the 3rd Saturday of each month at 10:30AM

 Orange County

Myeloma Support Group

Meets in-person every other month & virtually the alternate months on the 1st Thursday of each month

 Los Angeles Multiple Myeloma Support Group

Meets virtually on the 3rd Saturday of each month at 10:30AM

 Santa Cruz Multiple Myeloma Support Group

Meets virtually the 1st Monday of each month at 4:30PM

 Rancho Mirage, CA

Myeloma Support Group

Meets virtually on the 1st Thursday of each month at 3PM

IMF – Special Interest Virtual Groups

Special interest groups are designed as a supplemental support for specific populations of patients, in addition to their local Support Groups

 Las Voces de Mieloma

 Designed for Spanish speaking patients only

 New!

Care Partners Only

 Designed to address the needs of care partners only

 Smolder Bolder

 Created for people living with Smoldering Multiple Myeloma

 Living Solo & Strong with Myeloma

 Designed for patients without a care partner

 High Risk Multiple Myeloma

 Designed to address the needs of the high-risk MM population

 MM Families

 For patients/care partners with young children

EVALUATION

Please be sure to complete your program evaluation today.

Questions 1 – 5 can be completed before the program begins.

Questions 7 & 8 can be answered after each presentation.

If you are attending Friday program only, we ask that you turn the survey in at the end of the day.

If you are coming back for the Saturday sessions, please hold onto your survey, bring it back tomorrow and turn it in at the end of the program.

We greatly appreciate your time and feedback!

Hot Topics in Myeloma

Joseph Mikhael, MD, MEd, FRCPC, FACP

Chief

Medical Officer, International Myeloma Foundation

Professor, Translational Genomics Research Institute City of Hope Cancer Center

Shared Decision Making

Teresa Miceli, RN, BSN, OCN

International Myeloma Foundation InfoLine Advisor & Nurse Leadership Board; Mayo Clinic-Rochester

Shared

Decision Making: Be An Active Member Of Your Health Care Team

Teresa Miceli, RN BSN OCN

International Myeloma Foundation - InfoLine Advisor, NLB Member, Support Group Leader (MMSS, Smolder Bolder)

Mayo Clinic – Myeloma Nurse Navigator

Individual Beliefs & Preference s

Transplant

Eligible Patients

Individual Care Partner

Family

Initial Therapy

A Person With `

Treating Myeloma

Transplant (ASCT) Maintenance

Transplant

Ineligible Patients

Everyone

Social Network & Obligations

Treatment of Relapsed disease

Consolidation / Maintenance Continued therapy

Myeloma Symptom s & Treatment

Options

Supportive Care

Employm ent & Finances

beyond the clinical-trial setting: understanding the balance between efficacy, safety and tolerability,

SDM: Patient-Centered Care

“The aim of shared decisionmaking is to ensure that: - Patients understand their options and the pros and cons of those options.

- Patient's goals and treatment preferences are used to guide

https://www.ahrq.gov/cahps/quality-improvement/improvement-guide/6-strategies-for-improvin g/communication/strategy6i-shared-decisionmaking.html#6i1

Steps in Shared Decision-Making

 Identify that a decision is needed: The HCP informs the patient that a decision is to be made and that the patient's opinion is important (Choice talk).

 Understand the options:

The HCP explains the evidence-based options and their pros and cons. The patient expresses their preferences, and the HCP supports the patient in decision-making (Option talk).

 Come to a decision:

The HCP and patient discuss the patient's wish to take part in the decision making and incorporate the patient's values and preferences into the decision (Decision talk).

Follow-up: Review and evaluate the decision, adjust as needed

Advantages to Partaking in SDM

Patients, regardless of age, want to be a part of treatment decision-making

 Requires staying informed

 Reduces uncertainty and alleviates concerns

 Decisions reflect personal and family values

 Promotes patient and care partner engagement and sense of empowerment

 Positive impact on QOL and continuation on therapy

“The 'efficacy' of treatment means different things to different patients, and treatment decision-making in the context of personalized medicine must be guided by an individual's composite definition of what constitutes the best treatment choice.” Terpos, et al. Terpos, et al.

https://www.ahrq.gov/cahps/quality-improvement/improvement-guid e/6-strategies-for-improving/communication/strategy6i-shared-decisi onmaking.html#6i1

Influencing Factors to Treatment Decision-Making

 Disease-derived

 Time: Stage, risk stratification, Urgent intervention needed vs time to consider options

 Treatment: Availability/access, effectiveness, toxicity, current research

 Patient-derived

Choon-Quinones, Mimi, Hose D, Kaló Z, Zelei T, Harousseau JL, Durie B, Keown P, Barnett M, Jakab I. Patient and Caregiver Experience Decision Factors in Treatment Decision Making: Results of a Systematic Literature Review of Multiple Myeloma Decision Aids. Value Health. 2023 Jan;26(1):39-49. doi: 10.1016/j.jval.2022.04.003. Epub 2022 May 22. PMID: 35613958.

 Provider-derived

 Time limitations

 Support for patient involvement

 Provider bias and preference

 Understanding complex treatment options

 Physical and emotional wellness

 Comfort in speaking up “Doctor knows best”

 Financial, Cultural and Religious factors

 Care partner & social network, transportation

https://www.ahrq.gov/sites/de fault/files/wysiwyg/cahps/qua lity-improvement/improveme nt-guide/6-strategies-for-impr oving/communication/cahps-s trategy-section-6-i.pdf

Strategies for Patient Empowerment

 Consider your priorities

 Consider your goals/values/preferences

 Include your care partner/network in the discussion

 Be a part of the conversation, create a dialog

 Ask questions & Express your goals/values/preferences

 Ask for time to consider options, if needed

 Arrive at a treatment decision together

 Arrange follow up to review and adjust the plan, if needed

Strategies for Patient Empowerment

Know the members of your care teamUnderstand their different roles

 Myeloma specialist and General Heme/Onc

 Primary care: for health screening, general check ups, vaccinations

 Sub-specialists: specialty needs

 Keep a contact list of your providers

Primary Care Provider (PCP)

Subspecialists

Allied Health Staff

Strategies: Prepare For Medical Visits

Prepare

 Medications: Bring a current list of prescribed and over-thecounter

 Questions: Prioritize questions & concerns including financial issues

 Paperwork needing medical signature (ex FMLA, prior authorizations)

Inform

 Updates: Medical or life changes since your last visit

 Symptoms: How have they changed (improved, worsened, stable)? Keep a symptom diary. Bring it along

 Communicate effectively so your health care team can help

Follow Up

 “Next Steps”: Future appointments, medication changes, plan of care. Ask for the information in writing or on your patient portal

Include a care partner, especially for pivotal appointments

Strategies: Prepare For Tele-Med Visits

 Is telemedicine an option?

 What is the process and what technology is needed?

 Are labs needed in advance? Do you need an order?

Preparation is similar for “in-person” appointment PLUS:

 Location: quiet, well-lit location with strong Wi-Fi is best

 Yourself: Do you need to show a body part - wear accessible clothing

 Vital signs (blood pressure, temp, heart rate, weight) selfserve blood pressure cuff is available at many pharmacies and for purchase

Include a care partner, especially for pivotal appointments Check with your healthcare team –

Create a Care Network

 Care partners assist in many ways

 Attending medical appointments, being present to learn and discuss possible treatment options and alert the medical team of side effects to treatment

 Some treatment options available only if care partner support exists

 Care partners can be one person or a rotation of many people

 Building a partnership is based in good communication

 Finding the balance:

- helping the patient with needed activities while maintaining a sense of independence

- allowing the care partner to have time for good self-care

Myeloma causes the highest burden of symptoms, most commonly effecting people of older age with other medical issues. Care partner support is valuable in SDM Terpos, et al. 2021; Soong, et al., 2023

Care Partner Tip Card https://www.myeloma.org/resource-library/tipcard-care-partners

Credit: https://www.mmtoldtrue.com/community/care-partner-corner

Key Take-Aways - Think About It ….

Over the next two days:

 Evaluate where you are at in the process (What decisions need to be made?)

 Absorb the information being presented (What are the options?)

 Consider how the information impacts you and your family (What are your preferences?)

 Create questions that will lead to better understanding (What more do I need to know before making a decision?)

 Become an active member of your health care team

Share Decision Making

Resource List

Bylund CL, Eggly S, LeBlanc TW, Kurtin S, Gandee M, Medhekar R, Fu A, Khurana M, Delaney K, Divita A, McNamara M, Baile WF. Survey of patients and physicians on shared decision-making in treatment selection in relapsed/refractory multiple myeloma. Transl Behav Med. 2023 Apr 15;13(4):255-267. doi: 10.1093/tbm/ibac099. PMID: 36688466.

Chari A, Romanus D, DasMahapatra P, Hoole M, Lowe M, Curran C, Campbell S, Bell JA. Patient-Reported Factors in Treatment Satisfaction in Patients with Relapsed/Refractory Multiple Myeloma (RRMM). Oncologist. 2019 Nov;24(11):1479-1487. doi: 10.1634/theoncologist.2018-0724. Epub 2019 Aug 1. PMID: 31371520; PMCID: PMC6853123.

Choon-Quinones, Mimi, Hose D, Kaló Z, Zelei T, Harousseau JL, Durie B, Keown P, Barnett M, Jakab I. Patient and Caregiver Experience Decision Factors in Treatment Decision Making: Results of a Systematic Literature Review of Multiple Myeloma Decision Aids. Value Health. 2023 Jan;26(1):3949. doi: 10.1016/j.jval.2022.04.003. Epub 2022 May 22. PMID: 35613958.

Rifkin RM, Bell JA, DasMahapatra P, Hoole M, Lowe M, Curran C, Campbell S, Hou P, Romanus D. Treatment Satisfaction and Burden of Illness in Patients with Newly Diagnosed Multiple Myeloma. Pharmacoecon Open. 2020 Sep;4(3):473-483. doi: 10.1007/s41669-019-00184-9. PMID: 31605300; PMCID: PMC7426337.

3718 Cytokine Release Syndrome: The Patient, Caregiver and Healthcare Professional Experience. Janelle Soong, Giuseppe De Carlo, Naziah Lasi-Tejani, Sumanjit K. Sethi, Natacha Bolaños, Martine Elias, Yelak Biru, Solène Clavreul, G. Scott Chandler, Klaus Finzler, Yann Nouet, Antonio Giuseppe Del Santo. Blood (2023) 142 (Supplement 1): 3718

Terpos E, Mikhael J, Hajek R, Chari A, Zweegman S, Lee HC, Mateos MV, Larocca A, Ramasamy K, Kaiser M, Cook G, Weisel KC, Costello CL, Elliott J, Palumbo A, Usmani SZ. Management of patients with multiple myeloma beyond the clinical-trial setting: understanding the balance between efficacy, safety and tolerability, and quality of life. Blood Cancer J. 2021 Feb 18;11(2):40. doi: 10.1038/s41408-021-00432-4. PMID: 33602913; PMCID: PMC7891472.

https://www.ahrq.gov/health-literacy/professional-training/shared-decision/index.html

https://www.ahrq.gov/cahps/quality-improvement/improvement-guide/6-strategies-for-improving/communication/strategy6i-shared-decisionmaking.ht ml#6i1

Advanced Care Planning

Wendy Thomas, RN, MSN, CHPN

Palliative Care Nurse Specialist, Kansas University Medical Center

KC Area Support Group Leader

Advance Care Planning

Wendy Thomas, RN, MSN, CHPN

Wendy Thomas, RN MSN CHPN

• Outpatient Palliative Care

Nurse Navigator

• Kansas City Area Myeloma

Support Group Leader

About me:

• Nurse 27 years

• 14 years in blood and marrow transplant

• 8 years in palliative care

• 10 years as a myeloma support group leader

• Worked for the University of Kansas Health System for 17 years

• Based at the Bloch Cancer Care Pavilion, Westwood Kansas

Advance Care Planning

What is Advance Care Planning?

• Discussing and preparing for future medical care decisions

• Important at any stage of life

• Crucial for anyone with a serious illness

• Goes into effect ONLY when you are unable to speak for yourself

Do you have an Advance Directive?

• Who would you want to speak for you if you were unable to speak for yourself?

• Do your family/loved ones know what your wishes would be in a healthcare emergency?

• Do you know what your wishes would be?

• Are you confident they could carry out your wishes?

Advance Care Planning

DPOA & Healthcare Directive

CA requires notary or two adult witnesses to signature

What are your wishes?

Code Status

• What is a Code Status?

– Cardiopulmonary Resuscitation or CPR

• Why do they keep asking?

– Code status expires at discharge

– Out of hospital DNR

– Living will

• How aggressive do you want care to be?

– ICU

– Mechanical Ventilation

– Medically administered nutrition

Deciding about CPR CPR

• No pulse, not breathing

• One of the few treatments that patients must choose to NOT have performed

• A physician order is to NOT perform CPR

• Older people and people with cancer may have & quality of life

• You can CHOOSE to allow a NATURAL death if you prefer

Level of Medical Interventions?

Pulse present &/or still breathing

• Full treatment – most aggressive ICU and intubation with mechanical ventilation

• Midlevel treatment – less aggressive Antibiotics, fluids, medication to support blood pressure, transfusions

• Best supportive care – least aggressive Treat with dignity and respect, comfort-focused medical treatment

• DNR doesn’t equal non-aggressive care

Out of hospital DNR

• Patients should complete with your healthcare provider

• Requires healthcare provider signature

• Original form stays with patient

• Copy should be provided to all of your healthcare providers/health systems

• Some states have transportable DNR laws

POLST

• Physician Orders for LifeSustaining Treatment

• Provides more control over end-of-life care to seriously-ill patients

What to do with Forms & Documents?

• Make certain your family/loved ones know the location

• Give a copy to your healthcare providers/health systems

• Easy to find in case of emergency

• These documents DO NOT belong in your safe deposit box

• Fridge and beside table good locaitons

Other Practical issues

Planning ahead

• Eases the burden for family/loved ones

• Protects your assets

• Allows you to manage your personal effects

New Complications

• The electronic era brings new challenges

• Cellphones, computers, online accounts, social media and photos

Finances

• Bank Accounts

• Bill Pay

• Property Cell Phone

• Access

• Contacts

• Photos Passwords

• Account Log-in

• Social Media

• EVERYTHING!

The Most Important Part

Talking with your loved ones about your healthcare wishes brings comfort

National Healthcare Decision Day: April 16th

• Go Wish Cards

• ACP Bubble Map

• Coalition for Compassionate Care of CA

• Social Worker

Myeloma.org

Robin Tuohy Vice President, Patient Support

International Myeloma Foundation

BREAK

Return at 2:45

THANK YOU TO OUR SPONSORS!

Myeloma 101 & Understanding Your Labs

Joseph Mikhael, MD, MEd, FRCPC, FACP

Chief Medical Officer, International Myeloma Foundation

Teresa Miceli, RN, BSN, OCN

International Myeloma Foundation Nurse Leadership Board Member

Q&A with Teresa and Dr. Joe: Understanding Myeloma Basics

Joseph Mikhael, MD, MEd, FRCPC, FACP

 Professor, Applied Cancer Research and Drug Discovery, Translational Genomics Research Institute (TGen), City of Hope Cancer Center

 Chief Medical Officer, International Myeloma Foundation

 Consultant Hematologist and Director, Myeloma Research, Phase 1 Program, HonorHealth Research Institute

 Adjunct Professor, College of Health Solutions, Arizona State University

Teresa S. Miceli RN BSN OCN

Mayo Clinic, Rochester, MN

• Mayo Associate

• Assistant Professor of Nursing

• Myeloma Research RN Navigator

International Myeloma Foundation

• InfoLine Advisor

• Nurse Leadership Board

• Support Group Leader

NCI Myeloma Steering Committee

How common is Myeloma?

Rate of New Cases per 100,000 Persons by Race/Ethnicity & Sex How common is Myeloma?

Percent of New Cases by Age

https://seer.cancer.gov/statfacts/html/mulmy.html; dated

What

Causes Myeloma? How/Why Did I Get This?

Biochemical or Symptomatic Progression/Relapse

Environmental Factors:

• Exposure to some chemicals

• Radiation exposure

Examples:

 Agent Orange

 Burn pits

 Pesticides, Herbicides

 Firefighter/First Responder exposures

Individual Factors:

• Age

• Family History of related disorders

• Personal History of MGUS or SMM

• Obesity

In most cases, the honest truth

WE DON’T KNOW

Bone Marrow Cells – Good & Bad

Hematopoietic stem cell

White Blood cell

Graphic Credit: Teresa Miceli

Platelets

Red Blood Cells

Plasma cell

Photo Credit

Clonal Plasma cells

(Mono)clonal Plasma Cells

Heavy Chain: G, A, M, D, E

Heavy Chain = M-Spike

cells

Spectrum of Monoclonal Protein Disorders

Condition MGUS1-4 (Monoclonal Gammopathy of Undetermined Significance)

1-5,8 (Smoldering Multiple Myeloma)

• AL-Amyloid

• POEMS

• Light or Heavy Chain Deposition Disease

• MGRS = Renal

• MGNS = Neuro

Presence of Myeloma Defining Events

Likelihood of progression

1. Kyle RA, et al. N Engl J Med. 2007;356:2582-90.

2. IMWG. Br J Haematol. 2003;121:749-57.

3. Jagannath S, et al. Clin Lymphoma Myeloma Leuk. 2010;10(1):28-43.

* In clinical trial

5. Mateos M-V, et al. Blood. 2009;114:Abstract 614.

6. Durie BG, Salmon SE. Cancer. 1975;36:842-854.

7. Durie BG, et al. Leukemia. 2006;20(9):1467-1473.

4. Kyle RA, et al. Curr Hematol Malig Rep. 2010;5(2):62-69.

8. Rajkumar SV, et al. Lancet Oncology 2014; 15:e538-e548.

Myeloma and Myeloma Defining Events

Testing For Myeloma: Blood & Urine

Test Name

CBC + differential

Complete metabolic panel

Beta-2 Microglobulin (B2M)

Lactate Dehydrogenase (LDH)

Serum Immunofixation and Protein electrophoresis (SPEP+IFE)

Immunoglobulins (G, A, M, D, E)

Free light chain assay with kappa/lambda ratio

Urine immunofixation & protein electrophoresis (UPEP+IFE)

What it means

Hemoglobin, WBC, Platelets

Creatinine, Calcium, Albumin, Liver function

Part of staging and risk stratification

Measures the level of normal and clonal protein Identifies the type of clonal protein

This Photo by Unknown Author is licensed

Testing For Myeloma: Imaging

Imaging:

– Skeletal survey: Series of X-rays; less sensitive than other techniques

– Whole body low dose (CTWB-LD CT )

– Positron Emission Tomography (PET/CT)

– Magnetic Resonance Imaging (MRI)

Healthy bone versus myeloma bone disease

Testing For Myeloma: Bone Marrow

Bone marrow genetics

• Cytogenetics

• Fluorescence in situ hybridization (FISH)

• Next generation sequencing (NGS)

This Photo by Unknown Author is licensed under CC BY-SA

Staging and Risk Stratification

International Staging System (ISS) (only β2M and albumin)

Result

β2M < 3.5 mg/L; serum albumin ≥ 3.5 g/dL 2 β2M < 3.5 mg/L; serum albumin < 3.5 g/dL; or β2M 3.5 to 5.5 mg/L, irrespective of serum albumin

Risk FISH Results*

Myeloma Treatment Schema

Transplan t Eligible Patients

Transplan t Ineligible Patients Consolidation / Maintenance Continued therapy Everyone Supportive Care

Clinical Experience Research Results Your Preference TREATMENT DECISION

Philippe Moreau. ASH 2015.

Drug Class Overview

(thalidomide)

(lenalidomide)

Drug Class Overview

Peptide Drug Conjugate*

BCMA Targeted Antibody Drug

Conjugate (ADC)*

Bispecific Antibodies

(Melphalan Flufenamide)

Blenrep (belantamab mafodotinblmf) Bela, Belamaf, or B

Abecma (idecabtagene vicleucel)

Carvykti (ciltacabtagene vicleucel)

Tecvayli (teclistimab)

Talvey (Talquetamab)

Elrexfio (Elranatamab)

Cevostamab, Iberdomide, Mezigdomide, Venetoclax

Linvoseltamab, LCAR-B38M, ABBV-383 ……………………………

* These agents are currently off the market but available through special programs

Measuring Disease Response: IMWG Response Criteria

Negative by next generation flow (NGF) (minimum sensitivity 1 in 10-5 nucleated cells or higher)*

mCR AND normal Free Light Chain ratio, Bone Marrow negative by flow, 2 measures

CR AND negative PCR

Complete Response: Negative immunofixation (IFE); no more than 5% plasma cells in BM; 2 measures

Very Good Partial Response: 90% reduction in myeloma protein

Partial Response: at least 50% reduction in myeloma protein

Minimal Response

Progressive Disease: At least 25% increase in identified myeloma protein from lowest level Stable Disease: Not meeting above criteria

MRD = Minimal Residual Disease

sCR = Stringent Complete Response; BM = Bone Marrow

Kumar, S., Paiva, B., Anderson, K. C., Durie, B., Landgren, O., Moreau, P., ... & Dimopoulos, M. (2016). International Myeloma Working Group consensus criteria for response and minimal residual disease assessment in multiple myeloma. The lancet oncology, 17(8), e328-e346.

When Do I Need A New Treatment?

Biochemical or Symptomatic Progression/Relapse

• Not every relapse requires immediate therapy

• Each case is different

Symptomatic or extramedullary disease

Asymptomatic biochemical relapse on 2 consecutive assessments

Consider Treatment

Patient-/Disease-Specific Monitor Carefully

Asymptomatic high-risk disease or rapid doubling time or extensive marrow involvement Consider Observation Monitor Carefully

Initiate Treatment

Targets on the Myeloma Cell Surface and Therapeutic Antibodies

Bi-Specific Antibodies

Talquetamab

Antibody Drug

Elotuzumab

Bi-Specific Antibodies

Antibody Drug

Daratumumab and Darzalex Faspro Isatuximab

Immune Therapies

Ide-cel CAR-T

Cilta-cel CAR-T

Teclistamab

Other CAR-Ts

Other Bi-Specific Antibodies

The Evolution of Myeloma Therapy

Rev/Dex

CyBorD

VTD

VRD

KRD

D-VMP

DRD

Tandem ASCT (?)

Nothing

Thalidomide?

Bortezomib

Ixazomib

Lenalidomide

Combinations

D-VRD

Isa-VRD

D-KRD

Isa-VRD “More” induction?

Daratumumab?

Carfilzomib?

Lenalidomide + PI

ASCT, autologous stem cell transplant; CAR, chimeric antigen receptor; Cy, cyclophosphamide; d- daratumumab; D/dex, dexamethasone; isa, isatuximab; K, carfilzomib; M, melphalan; PDL1, programmed death ligand-1; PI, proteasome inhibitor; Rev, lenalidomide; V, bortezomib.

Speaker’s own opinions.

Bortezomib

Lenalidomide

Carfilzomib

Pomalidomide

Selinexor

Panobinostat

Daratumumab

Ixazomib

Elotuzumab

Isatuximab

Belantamab mafodotin*

Melphalan flufenamide*

Idecabtagene autoleucel

Ciltacabtagene autoleucel

Teclistamab, Talquetamab

Elranatamab

CAR T Cell Therapy

Bispecific/Tri-specific Antibodies

Cell Modifying Agents

Venetoclax*

PD/PDL-1 Inhibition?

Small Molecules

* These agents are currently off the market but available through special

What about Disease Control and Cure in Myeloma?

Biochemical or Symptomatic Progression/Relapse

 Control is the immediate priority with active disease  Cure remains the overall goal

Defining “Cure” has many considerations:

Minimal Residual Disease Negative (MRD-)

Time Off Therapy

Functional Cure

Requiring Treatment Stable or Unmeasurable Disease, Receiving Treatment

Unmeasurable Disease, Receiving No Treatment Active Disease

Advocacy

Danielle Doheny Director of Public Policy and Advocacy, International Myeloma Foundation

LA Patient and Family Seminar

Advocacy at the IMF

Informing and influencing policymaking on the critical healthcare issues that directly impact myeloma patients.

Principles Priorities Grassroots

Advocacy at the IMF

We believe in the value of working to improve healthcare policies to ensure there are no barriers to care as patients navigate their myeloma journey.

The IMF is the voice of the myeloma community in Washington, DC - informing and influencing critical policy decisions.

Our advocacy priorities are determined by the following three principles:

1. Ensure Equitable Access to Care

2. Eliminate Financial Barriers

3. Secure Research Funding

2024 U.S. Advocacy Priorities Snapshot

1. ENSURE ACCESS TO CARE

INSURANC

E REFORM: DRUG ACCESS

INSURANC

2. ELIMINATE FINANCIAL BARRIERS

3. SECURE RESEARCH FUNDING Step Therapy Protocols Safe Step Act

H.R. 2630 / S. 652

E REFORM: DRUG ACCESS PBM Reform PBM Reform Act

H.R. 5378 / S. 1339

INSURANC E REFORM: COINSURANCE

INSURANC E REFORM: COPAYS Copay Accumulators HELP Copays Act H.R. 830 / S. 1375 Oral Parity Cancer Drug Parity Act H.R. 6301 / S. 2039

FEDERAL FUNDING

ANNUAL APPROPS

Annual Appropriations

NIH: National Cancer Institute, National Institute on Minority Health, ARPA-H

CDC: Comprehensive Cancer Control Initiative

DoD: Congressionally Directed Medical Research Program (CDMRP) for Myeloma.

MEDICARE REFORM:

PHYSICIAN ACCESS

Tele-Health/Medicine

Telehealth Modern. Act

H.R. 7623 / S. 2016

MEDICARE REFORM: ANNUAL COST LIMITS

Inflation Reduction Act implementation Cap & Smoothing (MPPP), Drug Pricing

CLINICAL TRIAL DIVERSITY

Focus on underserved, POC, rural settings and socioeconomically disadvantaged groups

Role of Grassroots Advocacy

Grassroots Advocacy is the critical component to influencing policy decisions

The IMF brings advocates to Capitol Hill to share their experience with lawmakers.

Together, we champion legislative priorities that directly impact the lives of millions of patients and elevate the voices of of the myeloma community.

The IMF Grassroots Advocacy Program is multi-faceted and growing

• Advocacy Training & Leadership Development

• Policy and Legislative Education

• Grassroots Campaign Planning

• Health Policy Forums & Roundtables

• Advocacy Resource Development

• Storytelling and Personal Narratives

Medicare Changes Detailed | A Phased Approach

No copays for vaccines under Part D

Insulin copays limited to $35/month

Expanded Eligibility for the Federal Extra Help Program (Low-Income Subsidy Program) to help pay premiums, deductibles, coinsurance, etc.

$3,250 annual cap (approx.) on out-of-pocket spending for prescription drugs under Part D (eliminating 5% coinsurance in catastrophic phase)

$2,000 Annual Cap in out-of-pocket spending for prescriptions under Part D

Option for a monthly payment program to “smooth out” total out-of-pocket spending throughout the year, with an overall monthly maximum

• Patients will need to enroll into the program (opt-in)

• The earlier in the year you join the program, the more you can benefit

• Your monthly bill may fluctuate somewhat

• No one will pay more than $2000 for the year

$ Thousands Per Year

Recent Advocacy Events

Congressional “Lobby Day” Focus on Cancer Research Funding

Lobby Congress during annual appropriations process and ask for increased research funding via:

1. NIH (National Cancer Institute, National Institute on Minority Health & Health Disparities, ARPA-H)

2. CDC: Comprehensive Cancer Control Initiative

3. DOD: Congressionally Directed Medical Research Program (CDMRP) (myeloma-related research)

Congressional Briefing Focused on Oral Parity

Educate Congressional staff about the issue of Oral Parity and request their support to co-sign/advance the Cancer Drug Parity Act.

• One of the most widely attended Congressional Health Briefings of 2024 (attendance was 3x the average)

• Met with staff from key Congressional Committees

• Possible “IMF Lobby Day” in Sept. to move legislation

Thank You

Clinical Trials

Joseph Mikhael MD, MEd, FRCPC

Chief Medical Officer, International Myeloma Foundation

Professor, Translational Genomics Research Institute, City of Hope Cancer Center

Yelak Biru

President and CEO , International Myeloma Foundation

28-year Myeloma Patient

Objectives

• Provide The Rationale For Clinical Trials

• Outline The Phases Of Clinical Trials

• Discuss The Risks And Benefits Of Clinical Trials

• Listen To Patients Who Have Been On A Clinical Trial

Clinical TrialsOverview

Remember some of the important principles of clinical trials:

• The drive of research has brought us to where we are

• No one is expected to be a “guinea pig” with no potential benefit to them

• Research is under very tight supervision and standards

• Open, clear communication between the physician and the patient is fundamental

Clinical

Trials Myths

MYTH: If I participate in a clinical trial, I might get a placebo, not active treatment

MYTH: If I participate in a clinical trial, I can’t change my mind

• Phase 1 and 2, everyone gets active treatment

• Phase 3 standard of care vs new regimen: often standard regimen with/without additional agent in MM trials

• Patients can withdraw their consent for clinical trial participation at any time

MYTH: Clinical trials are dangerous because they have new medicines and practices

• Some risk is involved with every treatment, but medicines are used in clinical trials with people only after they have gone through testing to indicate that the drug is likely to be safe and effective for human use

MYTH: Clinical trials are expensive and not covered by insurance

• Research costs are typically covered by the sponsoring company

• Standard patient care costs are typically covered by insurance

• Check with clinical trial team/insurers; costs such as transportation, hotel, etc may not be reimbursed and are paid by patient

Clinical Trials –Why Me??

• Every patient is unique and must be viewed that way

• Benefits of trials are numerous and include:

• Early access to “new” therapy

• Delay use of standard therapy

• Contribution to myeloma world – present and future

• Financial access to certain agents

• Must be balanced with potential risks

• “Toxicity” of side effects

• Possibility of lack of efficacy

Overview of New Drug Development

Identify a target for therapy in the laboratory

Confirm the anticancer activity in laboratory and animal studies

Clinical trials (human studies) to determine safety, dosing and effectiveness

The whole process costs millions of dollars and years of effort!

Even Before Phase I

• Most agents are tested in lab models

• Various “myeloma cell lines”, also known as “in vitro”

• Next step is animal model

• We are more like mice than you think!!

• Earliest study in Phase I is called “First in Human”

• Often uses extremely low dose of drug to ensure safety

Phase 1 Clinical Trials

• All patients receive the experimental therapy

• Phase 1 trials find the optimal dose of a new drug or drug combination

• Patients get higher doses as the study continues

• Determine side effects of new drugs or combinations

• Explore how the drug is metabolized by the body

• Important for all stages of myeloma

Phase 2 Clinical Trials

• Determine if a new drug or combination is effective against the cancer

• May be added to a Phase 1 study once the ideal dose is found

• Patients usually receive the experimental therapy

• In some cases, the study may include two “arms” comparing either two different doses or a different treatment (another combination of drugs)

Phase 3 Clinical Trials

• Highest form of clinical evidence.

Typically, a large number of patients are required… usually required for full FDA approval

• Patients receive either an experimental therapy (one or more drugs) or the current standard treatment

o The patient is randomly assigned to a treatment—a process called “randomization”

o Neither the physician or the patient can determine which treatment is given

• May be placebo controlled, if no standard treatments are available

• Very closely monitored for effectiveness and side effects

Clinical Trials Phases

ANIMAL STUDIES: Examine safety and potential for efficacy

FIRST INTRODUCTION OF AN INVESTIGATIONAL DRUG INTO HUMANS

• Determine metabolism and PK/PD actions, MTD, and DLT

• Identify AEs

• Gain early evidence of efficacy, studied in many conditions; typically, 20 to 80 patients; everyone gets agent

EVALUATION OF EFFECTIVENESS IN A CERTAIN TUMOR TYPE

• Determine short-term AEs and risks; closely monitored

• Includes up to 100 patients, typically

GATHER ADDITIONAL EFFECTIVENESS AND SAFETY INFORMATION

COMPARED TO STANDARD OF CARE

• Placebo may be involved if no standard of care exists; hundreds to several thousand patients

• Often multiple institutions; single or double blind; sometimes open label

AGENTS IN NEW POPULATIONS OR NEW DOSE FORMS

Benefits of Participat ion

Possible benefits:

• Patients will receive, at a minimum, the best standard treatment

• If the new treatment or intervention is proven to work, patients may be among the first to benefit

• Patients have a chance to help others and improve cancer care

Risks of Participat ion

Possible risks:

• New treatments or interventions under study are not always better than, or even as good as, standard care

• Even if a new treatment has benefits, it may not work for every patient

• Health insurance and managed care providers do not always cover clinical trials

Why Do So Few Cancer Patients Participate

in Trials?

Patients may:

• Be unaware of clinical trials

• Lack access to trials

• Fear, distrust, or be suspicious of research

• Have practical or personal obstacles

• Face insurance or cost problems

• Be unwilling to go against their physicians’ wishes

• Not have physicians who offer them trials

• Have a disconnect with their healthcare team

Diversity in Clinical Trials

There has been a lack of diverse representation in clinical trials in myeloma.

• In the U.S., approximately 20% of all myeloma patients are of African descent, but only 5%–8% of patients in myeloma clinical trials are of African descent.

This is significant for the following reasons:

• All patients of all races and ethnicities should be able to benefit from clinical trials.

• Diverse patient representation in clinical trials is required to ensure that the outcomes are applicable to all patients.

Reasons for underrepresentation in clinical trials are complex and include:

• systemic racism, accessibility of clinical trials, sensitivity to diversity by medical professionals

• misconduct in medicine in the past, the lack of trust in the system, and more.

Importance of Participation by Diverse Populations in Clinical Trials

[P]eople from racial and ethnic minorities and other diverse groups are underrepresented in clinical research. This is a concern because people of different ages, races, and ethnicities may react differently to certain medical products.

– FDA

Leadership and commitment

Community engagement practices

Investigator hiring, training, and mentoring practices

Patient engagement practices

FDA = US Food and Drug Administration.

Regnante JM, et al. J Oncol Pract. 2019;15(4):e289-e299. FDA website. Clinical Trial Diversity. Accessed March 27, 2024. https://www.fda.gov/consumers/minority-health-and-health-equity/clinical-trial-diversity.

US Cancer Centers of Excellence: Strategies for Increased Inclusion of Racial and Ethnic Minorities in Clinical Trials