The Mission of the IMWG Summit

The mission of the IMWG Summit is to identify, support, and implement the most promising research to prevent the onset of active disease, to improve treatment, and to find a cure.

IMWG Summit Leadership

The Chairpersons of the IMWG Summit are Drs. Brian G.M. Durie and S Vincent Rajkumar.

The Co-leaders are Drs. Jesús San Miguel, Philippe Moreau, and Nikhil Munshi.

There are 254 members in the IMWG. Approximately, one-half of these members participate annually in the IMWG Summit. Timely topics are presented and discussed with guest speakers as appropriate. Additionally, work groups review ongoing and new research projects and guidelines.

The Venues for the Annual Summit

The venues for the Annual Summit have been the following:

2010 Barcelona 

2011 London 

2012 Amsterdam

2013 Stockholm 

2014 Milan 

2015 Vienna 

2016 Copenhagen 

2017 Madrid 

2018 Stockholm

2019 Amsterdam 

The 2020 Summit will be in Frankfurt, Germany.

2019 IMWG Annual Summit

The International Myeloma Working Group (IMWG) celebrated the tenth anniversary of its annual Summit from June 10 to 12, 2019. The Summit convened in Amsterdam, The Netherlands, just prior to the annual meeting of the European Hematology Association (EHA), gathering more than 100 of the world’s top myeloma experts to discuss cutting-edge therapy, debate current conflicts in myeloma, learn about scientific advances in other areas of medicine, and set an agenda for research and publications in the coming year.

The 2019 IMWG Summit was particularly noteworthy not only because it was the tenth, but because seven years ago, when the Summit was last held in Amsterdam, the Black Swan Research Initiative® (BSRI®) was created and its first research projects planned. The IMWG and the BSRI have come so far in the last decade: 38 publications and guidelines, new diagnostic criteria, 1,500+ citations of IMWG publications in the medical literature, a new imaging consensus guideline published this year in The Lancet Oncology, and a third phase of boundary-pushing BSRI research projects and clinical trials, several of which were updated at the 4-hour BSRI meeting held on Monday afternoon.

Introduction to A.I. in medicine

The IMWG Summit begins each year on a Monday evening with a welcome reception and dinner featuring a young expert in a new field of science that is likely to have an impact on future myeloma research. This year’s keynote presentation, “Machine Learning, Deep Learning, and A.I. – Recent Advances and Implications for the Study of Disease,” was given by Dr. Casey Greene of Princeton University. Deep learning, also known as “deep neural learning” or “deep neural network,” is an artificial intelligence (A.I.) function that imitates the workings of the human brain in processing data and creating patterns used in decision making.

Dr. Greene discussed the applications of deep learning in radiology, demonstrating how computers are trained to interpret X-rays by grouping pixels together in predefined ways to identify areas of disease. To demonstrate that human judgement is sometimes superior to A.I., Dr. Greene showed a slide in which photos of light-furred chihuahuas alternate with pictures of blueberry muffins.

The computer, seeing three dark spots that looked like eyes and a nose, could not differentiate the dogs from the muffins. As it happens, the IMWG’s Dr. Jens Hillengass is working with a team at the University of Heidelberg to develop software that will read myeloma patients’ CT scans – we hope with great accuracy! 

Dr. Greene and others in the field of deep neural learning see a future role for A.I. in interpreting “big data” gathered from meta-analyses of clinical trials and DNA sequencing projects.

Smoldering multiple myeloma

Whom should we treat with SMM?

The plenary session of the IMWG Summit started on Tuesday morning, offering up big unanswered questions that sparked lively debate: How do we stratify risk in smoldering multiple myeloma (SMM)? Who should be treated, at what point, and how? The conventional wisdom has been that all SMM patients should be monitored closely, but not treated outside clinical trials until they have myeloma-defining events (MDE, test results that point to active disease within two years) or actual organ damage (CRAB criteria). Even after moving patients with MDE from the SMM to the active myeloma category, there are still many SMM patients who will progress to active disease within two years. In the last three years, 51 clinical trials for SMM have emerged with treatment approaches ranging from prevention (with single-agent, usually continuous therapy) to cure (with more aggressive triplet or quadruplet therapy with/without autologous stem cell transplant [ASCT]). Eligibility criteria for these trials have ranged from anyone with a diagnosis of SMM to only those with a high risk of progression within two years.

When to intervene in SMM

Dr. Shaji Kumar presented data on a proposed new risk stratification model to predict who among SMM patients is likely to progress to active disease within 2 years. He outlined a system based on data from thousands of SMM patients diagnosed since 2004 (data was contributed by IMWG members from around the globe) who progressed to active myeloma after an analysis of:

• The percentage of bone marrow plasma cells,

• Amount of monoclonal protein,

• Ratio of involved to uninvolved free light chains, and

• Presence of certain cytogenetic abnormalities.

Risk was assigned on a point scoring system. Those with the highest point totals were placed in the high-risk and intermediate-risk groups because those patients had a 50% or greater risk of progression to active disease within two years. These patients, suggested Dr. Kumar, should be treated in clinical trials. With the data generated in those trials, it will be possible to determine if the diagnostic criteria should be changed once again to allow these patients to be treated outside the trial setting.

Treating SMM: Who, when, and how?

Dr. María-Victoria Mateos, who has conducted two clinical trials for patients with SMM in Spain, addressed the “who, when, and how” questions. Her first SMM trial, called “QuiRedex,” randomized participants to receive either Revlimid® (lenalidomide) + dexamethasone (Rd) or observation only. The data showed a significant benefit in progression-free survival (PFS) and overall survival (OS) in patients who had been treated with Rd. Allaying fears that treatment with Rd would make the myeloma cells resistant to subsequent treatment if the patients’ disease progressed, Dr. Mateos stated that the relapsing SMM patients were treated as normal when their disease progressed, and their survival was not different than those patients who had not received treatment for SMM. OS benefit was sustained even among patients with high-risk SMM; in fact, those at highest risk of progression benefited most from early intervention. This is an important point, one reinforced by the data from the ECOG E3A06 trial in which SMM patients were randomized to Revlimid or observation. Dr. Sagar Lonial presented the E3A06 clinical trial outcomes at the 2019 annual meeting of the American Society of Clinical Oncology (ASCO): patients who benefited from treatment with Revlimid were those at the highest risk of progression, while patients at low and low-intermediate risk did not benefit at all.

While some experts believe all patients with high-risk SMM should be given ongoing preventive treatment, others believe the goal of early therapy should be to eradicate disease clones definitively and attempt cure with aggressive but limited therapy, not treat continuously in an attempt to modify the immune response. Perhaps, Dr. Mateos suggested, a genomic model could be developed to help identify which patients would benefit from a preventive strategy and which should have curative therapy. We must wait for further trial results to see which approach leads to better long-term survival. Expert opinion is now leaning toward the belief that SMM as a disease category will disappear in the near future as research further elaborates disease biology; doctors will be able to determine who has MGUS and who has active disease that should be treated.

The next generation of frontline therapies

Dr. Rajkumar reviewed the state of therapy for newly diagnosed myeloma. With Velcade® (bortezomib) + Revlimid + dexamethasone (VRd) as the standard of care for newly diagnosed patients, questions that remain to be answered by definitive clinical trial data are:

• The relative benefits of other frontline triplet therapies 

such as the newly FDA-approved Darzalex® (daratumumab) + Rd (DRd).

• The possible benefits of such quadruplet frontline therapies at Darzalex + Velcade + melphalan + prednisone (DVMP) and Darzalex + Velcade + thalidomide + dexamethasone (D-VTd), both approved, as well as Darzalex + VRd (D-VRd), which is still in clinical trials.

• Factors that would predict who is a better candidate for quad therapy and who can benefit from a triplet.

Monoclonal antibodies in frontline therapy

Dr. Thierry Facon reviewed the impressive data from current clinical trials with Darzalex for newly diagnosed myeloma. In the CASSIOPEIA clinical trial of D-VTd conducted in Europe, the 18-month PFS was 93%, which surpasses the PFS rate of VTd followed by ASCT. In the ongoing GRIFFIN clinical trial of D-VRd, more than 90% of patients have achieved CR, demonstrating the power of a monoclonal antibody plus an immunomodulatory drug (IMiD®) and a proteasome inhibitor. Dr. Facon was the principal investigator of the MAIA clinical trial testing D-Rd, a study in which 44% of the participants were older than 75. This triplet worked well for these elderly patients. Median PFS has not yet been reached in either the MAIA clinical trial or in the ALCYONE clinical trial of DVMP, and is expected to reach approximately 50 months!

VRd and current approaches

Dr. Paul Richardson raised some interesting questions about the use of VRd with and without ASCT. He is conducting the DETERMINATION clinical trial, in which patients are randomized to VRd with and without ASCT. All patients receive maintenance therapy with Revlimid. At four years, patients are doing so well that there have not been enough events of progression or death to conduct an interim analysis. It is still not clear if ASCT should be used for all patients or for only a subset.

There is impressive emerging data on the use of frontline therapy with Kyprolis® (carfilzomib) + Rd (KRd). In Dr. Francesca Gay’s FORTE clinical trial of KRd continuously for 12 cycles vs KRd + ASCT + KRd consolidation, the regimens were equally effective in inducing high-quality responses, with about 50% of high-risk patients in either arm achieving minimal residual disease (MRD) negativity at 10-5. However, while there was no difference in time to disease progression among patients with low-risk myeloma, ASCT did reduce the risk of early relapse in patients with high-risk myeloma. The benefit of ASCT is certain to be a question for some but not all patients. Early data with quadruplet combinations (D-VRd, isatuximab-VRd, and D-KRd) are promising, and we have a new wave of immune therapies and antibody-drug conjugates (ADCs) to move things forward.

Discussion following Dr. Richardson’s presentation led to a lively debate on the definition of “cure.” At what point is a myeloma patient considered cured? Is it a number of years? Is it possible to define cure? Dr. Durie’s famous definition of cure is “getting older and dying from something else.”

CAR T-Cell therapy and BiTEs: Role for the future

Dr. Yi Lin presented an overview of CAR T-cell therapy trials to date. With one CAR T product, bb2121, on its way to FDA review later this year or in early 2020, the age of CAR T-cell treatments in myeloma has all but arrived. Novel CAR T-cell constructs are being evaluated, with 50 US clinical trials active and many more ongoing around the globe, particularly in China. Among them are clinical trials using other tumor antigen targets than BCMA, varied compositions of CD4 and CD8 T cells, and “off-the-shelf” allogeneic CAR T cells (from donors, not patients). Questions that remain to be answered are:

• Should CAR T-cell therapy be used earlier in the disease setting when the patient’s immune system is not so impaired?

• Should CAR T-cell therapy trials be randomized with more standard Darzalex-containing regimens?

• Should CAR T-cell therapy be used in the post-transplant setting, when tumor burden is low?

• Should CAR T-cell therapy be combined with IMiDs to improve the duration of response?

One major challenge that has emerged in early clinical trials is the lack of consensus for grading and management of the acute toxicity (cytokine release syndrome, or CRS) that comes with CAR T-cell therapy. Another is the limited predictive utility of MRD testing in CAR T-cell therapy clinical trials: bone marrow clearance is very rapid, and often precedes the attainment of complete response (CR), but relapses are fairly rapid (the longest PFS is just shy of one year) despite the attainment of MRD-negativity.

Emerging next-generation antibodies

Dr. Tom Martin’s overview of other types of promising and novel immune therapies included the antibody-drug conjugate (ADC), the leader among which is GlaxoSmithKline’s GSK 2857916 (GSK 916 for short), which is already in three phase II trials. The overall response rate (ORR) in the phase I trial of single-agent GSK 916 in very heavily pretreated patients was 60%, with PFS of 12 months. Dr. Martin is also keeping his eye on ADCs now in phase I trials, such as Seattle Genetics’ SGN-CD48A and SEA-BCMA, Sutro Biopharma’s STRO-001, and Fortis Therapeutics’ FOR46.

Among the bispecific T-cell engager (BiTE®) therapies, AMG 420 is the first in myeloma, and results in the phase I single-agent clinical trial were impressive, with a 70% ORR; some of the responses are ongoing at more than one year. AMG 420 is given continuously by intravenous (IV) pump for 4 weeks, followed by a 2-week break. Also in clinical trials is AMG 701, a version of 420 with a longer half-life, enabling it to be given weekly by IV infusion. Dr. Martin posed the question of whether these highly effective drugs should be moved up earlier in the disease course because they don’t eradicate the immune response and don’t harm the bone marrow. There are several other BiTEs in clinical trials, most targeting CD3 on the T cell and BCMA on the myeloma cell.

Future immunotherapies

Dr. Philippe Moreau next took the podium to look at the data on novel immune therapies critically and raise some profound questions. While the new therapies are exciting and hold great promise, they have not demonstrated long PFS, which has been uniformly at about 12 months for bb2121 (CAR T-cell therapy), GSK 916 (ADC), and AMG 420 (BiTE). Dr. Moreau asked, “How are we going to use these agents in the future?” If they are given late in the disease course, “no one will be cured.” CAR T cells do not persist, and patients relapse. They do not target the bone marrow microenvironment where myeloma cells grow, and they can cause infectious complications. Dr. Moreau believes that they should be used earlier in the disease, suggesting that perhaps they could be given to high-risk patients who are refractory to their first treatment, or they may be combined with IMiDs for better efficacy. He would like to see trials of ADCs or BiTEs versus triplet combination therapies. These topics, he promised, would be discussed at the immunotherapies working group session later in the afternoon.

High-risk disease: Classification and treatment

High-risk disease remains a major challenge in myeloma. Dr. Pieter Sonneveld introduced this session with an overview of where we are with the classification of high-risk cytogenetic abnormalities in myeloma. He considers the fluorescence in situ hybridization (FISH) test to be a poor indicator of risk because it identifies only a small number of the cytogenetic abnormalities (genetic mutations in the chromosomes) that occur in myeloma cells. He further lamented that there are different risk stratification approaches, with none quite agreeing what constitutes high risk.

Treatment of high-risk disease also poses a problem. Prognosis is generally poor for high-risk patients, although there is ample evidence that ASCT helps high-risk patients, and that they benefit from Darzalex. If they reach MRD negativity, their OS is comparable to patients with standard cytogenetics. Nevertheless, elderly high-risk patients do not do as well as their younger counterparts. Clearly, we need to better define high risk and to develop other treatment approaches. Currently, high-risk patients are treated in clinical trials along with standard-risk patients; Dr. Sonneveld asked, “Should we be treating high-risk patients differently?” He expressed the hope that there will be clinical trials exclusively for patients with high-risk disease, and emphasized the two outstanding problems that must be resolved: How do we define high-risk myeloma? How do we best treat high-risk disease?

Classification of high-risk disease

Dr. Wee Joo Chng addressed the first of these questions. He noted that one definition of high risk is retrospective: high-risk myeloma can be defined as OS < 3 years. What is needed, of course, is a prospective definition so that high-risk patients can be appropriately identified and treated.

Dr. Chng pointed out that the IMWG’s Revised International Staging System (R-ISS) includes only 3 cytogenetic abnormalities (CA), and that one of them – t(4;14) – is no longer relevant because this risk is negated by treatment with a proteasome inhibitor. Moreover, the high-risk CA deletion 17p (loss of the short arm of chromosome 17, on which sits a major tumor suppressor gene) is not equally bad for all patients. Is there a certain percentage of myeloma cells that must express this loss? Data has shown that prolonged exposure to proteasome inhibitors increases the survival of del 17p patients. Does R-ISS stage influence risk? We know that patients with 1q amplification, which has recently gained focus as a high-risk CA, seem to be at high risk specifically in the setting of stage III disease. We do know at this point that outcomes are worse as the number of poor-risk genetic features increases. Novel treatments can improve outcomes, but at most can only partly overcome poor prognosis in high-risk patients.

Dr. Chng reported that data showing early death in 18% of patients cannot be explained by any of the current risk factors. Sometimes risks cannot be identified by testing the bone marrow, and sometimes risk can vary by the site from which the bone marrow was extracted. Perhaps, he posited, liquid (blood-based) biopsy is a better way to assess risk than bone marrow biopsy. Beyond testing for genetic mutations, other signs of high risk in myeloma are early relapse from ASCT (≤ 1 year) and frailty.

Questions that remain to be answered are:

• Who is high risk and what are the criteria? Is the definition different for fit vs. non-fit patients?

• Do we need new criteria and methods to define high risk at diagnosis and during treatment?

• Should we treat high-risk patients differently and, if so, what are the options?

• Is there a need for continuous treatment?

Treatment of high-risk disease

Dr. Saad Usmani tackled the challenging question of treatment of high-risk disease by posing the reverse question, “What do we know about long-term survivors?” They have low disease burden and minimal organ damage, favorable disease biology, a CR or better during the first year of therapy, and they receive optimal frontline therapy that includes induction with a proteasome inhibitor and IMiD + ASCT + maintenance therapy. They do not have high disease burden, renal failure, active bone disease or extramedullary disease, nor do they develop second primary cancers or have multiple other medical conditions. We understand that when myeloma cells escape the bone marrow microenvironment, disease biology is very different; Dr. Usmani says that these patients at highest risk are likely to have plasma cell leukemia and involvement of the central nervous system, and are therefore excluded from clinical trials.

Definitions of high risk also remain a problem. Patients with loss of 17p in both alleles of a mutated gene have far worse prognosis than those with 17p- in only one allele, but no differentiation has been made between these subsets of patients in analyzing existing clinical trial data. We know that drugs may help overcome certain high-risk cytogenetic abnormalities, such as all proteasome inhibitors for t(4;14) and Kyprolis in particular for some patients with del 17p. Conversely, however, proteasome inhibitors seem to make matters worse for those with t(14;16). Pomalyst® (pomalidomide) may overcome poor PFS and OS for del 17p – but again, for which subset of 17p-deleted patients? High-dose melphalan with ASCT may help when coupled with RVd induction and consolidation, and tandem transplant may be of benefit in places where proteasome inhibitors and IMiD-based induction therapy is not available. Darzalex helps high-risk patients, but not “all the way.” Dr. Usmani ended with a plea for better clinical trials designed for those with high-risk myeloma.

Unraveling the complexities of treatment options for relapsed myeloma

The morning’s final plenary session, introduced with Dr. S. Vincent Rajkumar’s annual overview, covered the thorny topic of the many options for treatment of relapsed disease. There is no consensus on the sequence of therapies, although six phase III trials have demonstrated unequivocally that triplet therapy is superior to doublet therapy. Thus far, CASTOR (Darzalex + Vd) and POLLUX (Darzalex + Rd) clinical trials have the best results, although Empliciti® (elotuzumab) + Pomalyst + dexamethasone (EPd) is close behind and a possible option after D-Vd or D-Rd, as is D-Pd. In the United States, Kyprolis + Pd is also available based on a phase II clinical trial.

Dr. Rajkumar offers what he calls the TRAP criteria for determining treatment at first, second, or subsequent relapse:

• Timing of therapy,

• Response to prior therapy,

• Aggressiveness of the relapse,

• Performance status of the patient.

He recommends transplant in eligible patients who have not had a prior one, and consideration of clinical trials in all cases of relapse. Remaining questions are determination of which regimen is best in which situation, what can be done about access to and cost of drugs in the US and around the globe, and how best to incorporate therapy based on underlying disease biology.

Drs. Philippe Moreau and Paul Richardson reviewed currently approved drugs and clinical trial options in their respective parts  of the world (the European Union and the US), and the blessings and difficulties of having multiple treatment options. 

Dr. Richardson posed the questions, “What do we do when daratumumab fails?” and “What do we do when lenalidomide maintenance fails?” He suggested that Pomalyst + Velcade + dexamethasone (PVd) is a good option in the latter setting, because 90% of patients exposed to Revlimid responded to PVd, and a surprising 84% of those who were refractory to Revlimid responded. Both he and Dr. Moreau have expressed their strong belief that patient preference is of critical importance in determining relapse therapy. Another critical matter for both doctors is the lack of data on which regimen is optimal for treating patients who have relapsed on Revlimid maintenance therapy. They have proposed three research projects:

1. A definition of Revlimid-refractory (is a patient on 10 mg of Revlimid who progresses on maintenance truly refractory?),

2. Management of patients who are refractory to Revlimid,

3. Management of patients after failing Darzalex and CAR T-cell therapy.

Working group reports

The Tuesday plenary session was followed by two-hour working group meetings, each focusing on an area of research. Wednesday morning was reserved for each working group’s summary report to the full assembly of their discussion and action plans.

Group 1: Mass spectrometry

Dr. David Murray, who runs the Mayo Clinic Special Protein Lab, gave a brief overview of this new method to detect monoclonal proteins with great precision and presented his group’s discussion points:

1. Replacement of current testing methods SPEP (serum protein electrophoresis) and IFE (immunofixation electrophoresis) with mass spectrometry because of its increased sensitivity, more accurate quantitation, improved workflow in the lab, and ability to detect light chain glycosylation and AL amyloidosis.

2. Mass spectrometry has the ability to report

a. the mass of the myeloma light chain,

b. the presence and mass of treatment monoclonal 

antibodies such as daratumumab and elotuzumab,

c. isotype-specific kappa/lambda ratios that can replace 

the HevyLite® test,

d. loss of other proteins in the blood (albumin, alpha1, 

alpha2, and beta proteins).

3. Mass spectrometry will be used for MRD and early relapse detection.

4. Need to standardize mass spectrometry to Next-Generation Flow and Next-Generation Sequencing prior to regulatory approval.

Dr. Murray gave a progress report on work being done to apply for approval of mass spec by the FDA and the European regulatory agency and the trial data that will be submitted to  these agencies.

Group 2: Bone and imaging

Dr. Evangelos Terpos presented the summary of discussion, which included proposed studies and new guidelines on imaging. The first proposed study is a prospective one to compare sequential diffusion-weighted-imaging MRI (DWI-MRI) with PET-CT in the determination of MRD status in patients in complete response. The goal is to determine if DWI-MRI can detect abnormalities earlier than PET-CT and, importantly, to see if DWI-MRI, which does not use ionizing radiation, can replace PET-CT, which does use radiation in the CT portion of the test.

A proposed retrospective study, one that looks at data from completed clinical trials, will look at data from patients who have had both PET-CT and DWI-MRI at diagnosis and at CR. Another proposed prospective study would collect data from patients who have both PET-CT and DWI-MRI before CAR T-cell administration, and again after 1 and 3 months, because there have been several false positive results by PET-CT in this setting. A study must be conducted to determine if DWI-MRI would be more accurate than PET-CT.

The bone group would also like to write and publish new guidelines for the management of myeloma-related bone disease to include:

• Recommendations on the spacing of bisphosphonate therapy (every month? every three months? etc.)

• Recommendations for the use of denosumab in myeloma, including:

♦ Recommendations on the duration of therapy and especially on how to stop therapy,

♦ Risk of vertebral fractures if denosumab is stopped 

without bisphosphonate overlap (“rebound 

phenomenon” with denosumab),

♦ When to give denosumab and when to use a bisphosphonate.

Group 3: CAR T cell, BiTEs, and other immunotherapies

This group’s discussion and action plans were presented by Dr. Yi Lin, and centered on T-cell-based and BCMA-based therapy, including the need for:

• A uniform system for grading toxicities and how to manage them, including cytokine release syndrome, macrophage activation syndrome, CAR T-cell-related encephalopathy, and cytopenias (low blood counts),

• A data bank to help define and evaluate disease that is resistant to BCMA-targeted therapy,

• A consensus on which tests to use to assess response and when best to perform them,

• Immune profiling to determine CAR persistence,

• Determination of quality of life (QOL) outcomes with the various treatments (CAR T cells, BiTEs, and ADCs).

Group 4: SMM

Dr. Shaji Kumar presented a summary of the many issues facing treatment of patients with smoldering myeloma. Among the needs assessed by the SMM working group were:

• The need to study risk factors other than cytogenetics, especially immune parameters that may allow identification of higher-risk patients,

• The need to determine whether to treat for prevention or eradication,

• If those identified as being at 80% or greater risk of progression at 2 years are treated as if they have myeloma, the need to determine how to treat patients at 50% risk of progression at 2 years:

♦ Treatment to prevent progression?

♦ Treat like myeloma?

♦ No treatment, observation only?

♦ In clinical trials for prevention?

• The need to determine microenvironment signatures that predict long-term disease stability (“MGUS signature”),

• The need to develop surrogate markers that predict imminent CRAB features before damage occurs.

Clinical trial challenges were also discussed:

• How do we define progressive disease in a trial for SMM? Would biochemical progression be sufficient?

• If biochemical progression is used, are patients still smoldering when they progress?

• Can they go on another trial for SMM? Is “prevention” a  line of therapy?

• The need to develop uniform treatment strategies for patients who progress on SMM treatment trials and are “relapsed smoldering.”

Areas of agreement among group members were:

• SMM patients at the lowest risk of progression (around 4% risk at 2 years) are like MGUS patients, and can be followed like MGUS after the first 3-6 months of close monitoring. They are not candidates for intervention with more toxic approaches.

• Patients who do participate in intervention trials should have their stem cells collected for future transplant. We must work out reimbursement issues with insurance over payment for collection and storage of stem cells in this setting.

Group 5: New drugs in myeloma, next steps to improve drug access

Dr. Sonja Zweegman presented the discussion from this group, which was comprised of IMWG members outside the US, where access to new therapies is limited and often delayed. 

Dr. Zweegman began with the good news that a record 15 new oncology therapeutics were launched worldwide in 2018, and that 31% of newly approved therapies in the past five years were for hematologic cancers.

The other side of the coin is that there is a delay in reimbursement approval, with some countries having to wait three years or longer after the first EU countries have the drug. Because regulatory agencies in France only accept OS as a clinical trial endpoint, daratumumab, which extends OS by years, is not yet approved. Low-income countries where drug costs are prohibitive may never receive approval at all. Many countries require health technical assessments (HTA). The IMWG could assist the effort to expand drug coverage by:

• Advocating for joint clinical assessments for HTA rather than leaving it to each individual country,

• Providing data for PFS as a surrogate endpoint for OS,

• Performing analyses of best treatment approaches, best sequencing of therapies, and optimal duration of therapy,

• Performing QOL analyses that could be used in health technical assessments.

The IMWG Summit adjourned after an exhilarating two days, in time for Drs. Durie, Moreau, and Joseph Mikhael to participate in the live-streamed Conference Series, in which they weighed in on the data from the recent ASCO, EHA, and IMWG meetings. With much accomplished and much that lies ahead, IMWG members can look forward to another highly productive year and a summary of progress made in six months, when they meet again at the 2019 annual meeting of the American Society of Hematology (ASH) in Orlando, Florida.

Summit Sponsors:

Abbvie, Amgen, Binding Site, Bluebird Bio, Bristol-Myers Squibb, Celgene, EDO, Glaxo Smith Kline, Janssen Oncology, Karyopharm Therapeutics, Kite Pharma, Oncopeptides, Roche, Sanofi Genzyme, Takeda


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