National Organization for Rare Diseases (NORD) and the Drug Information Association held their 2nd Annual Conference – the US Conference on Rare Diseases and Orphan Products: Shaping the Future Now. The Meso Foundation was in attendance representing mesothelioma and the meso community.
Sessions focused on advocacy for inclusion of patients and community members in the research process, patient access to health care, and development of relationships with stakeholders. Special attention was paid to the unique challenges that rare disease patient advocacy organizations face. Several NORD member patient advocacy organizations presented case studies of their advocacy successes. Many members of NORD face some of the same issues that face the Meso Foundation; including little or no treatment options, few clinical trials, and limited research funding.
One issue discussed at length was addressing the rising cost of health care. Mark B. McClellan, former director of the Centers for Medicaid and Medicare (CMS), presented on access to care and cost containment. He made the point that expanding coverage and lowering prices won’t support needed innovation, but we need financing reforms that achieve better quality and lower costs. We are facing even more pressure in the coming years. He suggested public insurance providers, Medicaid and Medicare, will try to contain costs by squeezing payment rates, looking to medical innovation, and use of technology to lower costs. He encouraged the rare disease community to remain vocal advocates, so that rare diseases are not ignored and drugs for orphan diseases continue to be developed. Last year there were more than 400 drugs in development for rare diseases, more than one third of those were for rare cancers, including mesothelioma. The Orphan Drug Act has made strides to provide pharmaceutical companies to invest in orphan drugs for rare diseases. One suggestion to improve development of orphan drugs in the United States would be to increase drug exclusivity to the same level as Europe from 7 to 10 years. The Meso Foundation has supported this advocacy effort.
Another important issue for the rare disease community, as well as the meso community, was the idea of risk tolerance. Both the National Institutes for Health (NIH) and the Food and Drug Administration (FDA) had representatives at the meeting that heard the community’s concerns about assessment of risk tolerance. The Meso Foundation had the opportunity to express the view of a patient facing a fatal illness who may be willing to undertake a greater risk than those with more treatment options. The NIH and FDA representatives assured the community that they do consider the severity of a condition as well as the treatment options when approving clinical trials and approving drug safety and efficacy.
The pending threat of the sequester on January 2, 2013 was discussed as well. Chris Jennings, former White House Senior Health Advisor, presented his thoughts on the issue. He compared the sequester to business or family budgets, stating that “no business or family would make across the board cuts to their budgets – they would preserve funding for the important things and make targeted cuts.” He also touched on an important theme of this election year, which is the need to see a bipartisan solution to balancing the budget and reducing the deficit. The Meso Foundation has been a vocal advocate for Congress to act this year to avert a sequester.
Last night, Dr. Tobias Peikert of the Mayo Clinic joined us on a conference call, as part of the Meso Foundation’s Meet the Experts series, to discuss an exciting new clinical trial. The trial, which uses the measles virus to attack the mesothelioma cells, is currently enrolling pleural mesothelioma patients regardless of whether they’ve undergone prior therapy. This is a Phase 1 trial, meaning that the goal of the trial is to determine the level of toxicity of the agent and to establish the optimal dose.
According to Dr. Peikert, the measles virus is particularly interesting in the treatment of cancers. Historically, the notion of using this virus came to scientists mostly by accident. They noticed that when people with cancer became ill with measles, their cancer nearly or all disappeared. As a result, the idea has already been used for some time with treatment of lymphoma and other hematologic cancers.
This trial has now been open for six months and is administered in Rochester, Minnesota.
The Mesothelioma Applied Research Foundation conducts Meet the Experts conference calls every couple of months. To receive notifications of these calls, please sign up for news from the Meso Foundation.
The full podcast of the interview with Dr. Peikert is available on Meso Foundation’s website.
With this past weekend’s “Miles for Meso” Bike Run fundraiser, the events surrounding Meso Awareness Day have officially concluded for 2012! (Though awareness-building is far from over – continue checking our website for additional events coming up in November). This year, we are excited to report that in August, September and October, Meso Foundation supporters organized 15 events benefiting research, education, support and advocacy through the Meso Foundation.
In addition, the Meso Foundation hosted an awareness-building event on Rockefeller Plaza, obtained publicity from the Baltimore Orioles and organized a conference in New York in conjunction with Meso Awareness Day.
Here are some statistics from the awareness events. Our supporters and volunteers:
- Raised in excess of $120,000 for the Meso Foundation and its mission
- Have been covered by at least 11 news outlets
- Brought to their events over 2,000 attendees
- Distributed 2,500 brochures
- Received coverage on national television
Here are some photos from events. Please take a look and celebrate this success with us!
by Lee M. Krug, MD
At the International Mesothelioma Interest Group Meeting in Boston, a group of investigators convened at a Clinical Trials Design Workshop to discuss the challenges of conducting trials in this disease. The rarity of mesothelioma poses various issues with regards to proper study design, accrual, and interest by pharmaceutical companies. One theme that emerged from that workshop was the need to establish a clinical trials consortium. This concept has been quite successful with other rare cancers such as pediatric tumors and sarcoma. I propose that the Meso Foundation would provide the optimal mechanism to initiate such a consortium for mesothelioma trials.
The Meso Foundation Trials Consortium (MFTC) would offer the following advantages:
- It would provide scientific input to ensure that the trials are scientifically sound. The investigators involved in the Consortium best understand the biologic underpinnings of the disease. They would have important insight into the optimal design of the trials. Trials could be prioritized based on scientific merit and promise. This would ensure that valuable resources would not be wasted on trials unlikely to be successful.
- It would establish an international network of participating institutions with the highest volume of mesothelioma patients. This would have drastic effects on speeding accrual to complete trials in a timely fashion. The vorinostat phase III trial took over 5 years to complete enrollment, despite enlisting 125 institutions in 23 countries. The majority of those sites enrolled only 1 or 2 patients over that time. Typically, pharmaceutical companies hire Contract Research Organizations (CROs) who identify sites to conduct trials and collect the data. CROs do not know which institutions will have the appropriate patients for the trial.
- It would offer a referral mechanism to boost accrual. Patients look to the Meso Foundation as the primary source for medical advice, so they could be steered toward institutions that are participating in the designated studies.
Of course, the Meso Foundation does not have the resources to conduct clinical trials itself. The funding will initially need to come from the pharmaceutical industry. The process would work as such: Pharmaceutical companies interested in conducting large trials in mesothelioma would formulate an agreement to work with the Meso Foundation. Their protocols would be reviewed by a subcommittee of the Science Advisory Board who would provide feedback on the design and technical aspects. The Meso Foundation would solicit key institutions with strong research programs and high patient volumes to open the trial at their sites. Patients would be preferentially referred to these studies for participation. Once the success of this mechanism has been established, other companies will surely look to the Foundation for assistance. Ultimately, investigator-initiated trials could even be considered.
This year, more pharmaceutical companies than ever are planning clinical trials for patients with mesothelioma. This heightens the urgency to develop this consortium. As a research community, we cannot wait any longer. Let’s work together to make this happen.
Lee M. Krug, MD, is an Associate Attending Physician in the Division of Thoracic Oncology, Department of Medicine at Memorial Sloan-Kettering Cancer Center in New York, NY. Dr. Krug is also the Director of the Mesothelioma Program at Memorial Sloan-Kettering Cancer Center and Chair of the Science Advisory Board of the Mesothelioma Applied Research Foundation.
Last week, the National Institutes for Health (NIH) issued a notice stating non-competing research grant awards will be funded below the level indicated in the most recent Notice of Award, or “generally up to 90% of the previously committed level,” until the FY 2013 appropriations are enacted. This means that all researchers, including those involved in mesothelioma research, will need to adjust their operations to adapt to a 10% funding cut.
The continuing resolution provides stopgap funding to keep the federal government running for six months. It provides funding at the Fiscal Year 2012 level plus 0.6 percent until expiration on March 27, 2013.
The NIH said that until FY 2013 appropriations are enacted, it will issue non-competing research grant awards below the level indicated on the most recent Notice of Award — generally up to 90 percent of the previously committed level. NIH said that the decreases are consistent with previous practices during the continuing resolutions of FY 2006 – FY 2012.
It added that award levels could be adjusted upward after FY 2013 appropriations are enacted, though it “expects institutions to monitor their expenditures carefully during this period.”
Find the notice at: http://grants.nih.gov/grants/guide/notice-files/NOT-OD-13-002.html