Breakthrough Business Models Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary (2009) / Chapter Skim
Currently Skimming:
7 Strategies for Facilitating Clinical Trials
7 Strategies for Facilitating Clinical Trials
Pages 82-97
The Chapter Skim interface presents what we've algorithmically identified as the most significant single chunk of text within every page in the chapter.
Select key terms on the right to highlight them within pages of the chapter.
From page 82... ...
Patients should not feel isolated or stigmatized by the diagnosis of such a disease. All therapies must be proven safe and effective in clinical trials with human subjects before they can be approved for broad use, regardless of the size of the target population.
|
From page 83... ...
resources. Regulatory Tools The Orphan Drug Act has been successfully implemented, resulting in the approval of 326 products to treat rare and neglected diseases over the past 25 years. There are additional tools pertaining to the regulation of nonorphan drugs, such as the Prescription Drug User Fee Act (PDUFA)
|
From page 84... ...
A sister organization to MMRF, founded in 2004 by Kathy Giusti, MMRC has as its mission accelerating the development of novel and combination treatments for multiple myeloma by facilitating clinical trials and correlative studies. MMRC integrates the research efforts of 15 member institutions that represent the lead ing myeloma centers in the United States and the world.
|
From page 85... ...
MDA works through private–private partnerships with other nonprofit organi zations, such as the TREAT-NMD neuromuscular network in Europe, the Interna tional Coordinating Committee for Spinal Muscular Atrophy Clinical Trials, and the Duchenne Research Collaborative International. http://www.mda.org
|
From page 86... ...
• FDA guidance available: Guidance for Industry Fast Track Drug Development Programs -- Designation, Development, and Application Review (http://www. fda.gov/Cder/Guidance/5645fnl.pdf)
|
From page 87... ...
Communication with FDA Regardless of whether a product is designated as an orphan drug or has fast-track designation, early, frequent, and quality communication between FDA and the drug developer is crucial. There are a variety of opportunities for communication with FDA; however, small companies and individual
|
From page 88... ...
There may then be only one pivotal trial, as opposed to a pivotal trial followed by a confirmatory trial, as is typically the case for nonorphan drugs. On occasion, a sponsor will initiate an IND, not communicate with the agency for 5–7 years, and then submit an NDA with a
|
From page 89... ...
NDA/BLA Issues Some of the issues FDA encounters frequently with NDAs and Biological Licensing Applications (BLAs) for orphan drugs arise from misunderstandings about what is required for an orphan drug.
|
From page 90... ...
The published literature, such as case reports, is often inadequate for rare diseases. The most severe cases tend to be published and may not be representative of the broader affected population and/or attenuated presentations.
|
From page 91... ...
For example, review divisions may have patient consultants participate in meetings, or patients may serve on advisory committees as special employees of the government. As discussed earlier, patient advocacy foundations have contributed significantly to the funding of clinical trials.
|
From page 92... ...
Early on, MMRF determined that to accelerate drug development, it needed to operate like a drug development company. The leadership team consists of the CEO and founder, who is a myeloma patient with 10 years of experience in drug commercialization at two major pharmaceutical companies; a chief scientific officer with 16 years of industry experience in drug and target discovery; and a chief medical officer, who is a trained hematologist/oncologist with more than 20 years of industry experience in clinical research and drug development.
|
From page 93... ...
During concept and protocol development, for example, MMRC brings sponsors and centers together in weekly teleconferences, and it is developing a standard protocol template. With regard to site selection, MMRC knows the number of its own trials that are open at each site, as well as the total number of myeloma trials and the number of myeloma patients seen by each center.
|
From page 94... ...
The resulting information can help companies understand who the target population is and who is most likely to benefit from the drug, and bring drugs that truly work in a given population to the market more quickly. Muscular Dystrophy Association's approach to Maximizing Assets in Clinical Trials The Muscular Dystrophy Association (MDA)
|
From page 95... ...
An industry drug sponsor would then be able to run a quick search of the database to determine how many eligible patients exist in multiple countries across the world. Another collaboration in which MDA is involved is the International Coordinating Committee for Spinal Muscular Atrophy Clinical Trials.
|
From page 96... ...
Given that clinical research networks already exist for such diseases as ALS, SMA, and DMD, MDA decided that it would work with these networks, augmenting their efforts by funding an FTE clinical coordinator at 10 ALS and DMD centers. MDA plans to expand this funding to include five clinics focused on SMA next year, with the ultimate goal of funding 50 clinics in the network. Finally, MDA is actively working to develop, validate, and standardize end points.
|
From page 97... ...
Young pointed out that MMRC recently announced a collaboration with the Leukemia and Lymphoma Society, an organization with which MMRC competes directly for funding. The collaboration is focused on stem cell research, identifying and targeting the myeloma stem cell.
|
Key Terms
This material may be derived from roughly machine-read images, and so is provided only to facilitate research.
More
information on Chapter Skim is available.