- Long-term investment in research
- Comparative effectiveness as a tool for personalized medicine
- Develop guidelines that foster public-private partnerships
- Provide incentives for the private sector to get involved in research
- Have an expansive view of the healthcare systems (including HIT, clinical trials, treatment development, etc.)
- The importance of studying the impact of healthy lifestyles and their impact on health outcomes
- Risk-taking research
- HIPPA as it relates to the stimulus package
- Privacy
- Patient access to compassionate use
What else would you like to add to the discussion?
A few of of the participants wanted us to includ evidenced-based research and quality of life vs. measurable medical benefit as important principles of consideration
ReplyDeleteExpand the possibility of global research network or collaboration ... gaining advantage of greater patient population for human trial, and local/regional regulation/non-regulation in medical research.
ReplyDeleteI would like to suggest looking at some of the loopholes in the current Orphan Drug Act, as it currently is for drugs being developed and marketed by drug companies, and they have a say in whether they want to seek Orphan Drug status.
ReplyDeleteThere are some drugs that are currently on the market now for various uses, that in recent years have been found to be excellent and life-saving for some newly discovered conditions, and some that their genetic cause has ben discovered in this century.
Anakinra is one of these drugs, and this IL-1 receptor antagonist drug is currently one medication that the drug companies have resisted seeking Orphan Drug status for, or even FDA approval for on-label use for the genetic conditions that have proven to benefit directly from this drug. Despite repeated requests by patients and doctors, the drug companies have resisted protection for this drug for the use in some rare, but devastating conditions, such as Cryopyrin-Associated Periodic Syndrome (CAPS) and a newly discovered Deficiency of Interleukin 1 Receptor Antagonist (DIRA) condition.
Both of these rare conditions have excellent research (New England Journal of Medicine and many other journals) to prove that these drugs directly resolve the symptoms of these conditions, yet anakinra is at risk as it has been sold in the last year from Amgen to Biovitrum, and the new company (Biovitrum) owners must only continue under contract to make anakinra for 3 years, then they can choose to continue it. This drug targets the genetic cause for the condition, is a miracle drug for these conditions and must stay available for patients and also be more available to patients worldwide.
In the case of DIRA, patients are born without natural IL-1 Receptor antagonist ( anakinra is a biologically created replacement based on human IL-1 RA) all die within months of birth, to before school age. With CAPS, there are some new drugs that have shown promise in treating the overproduction of IL-1 in the body in CAPS patients, but anakinra is the drug of choice to use on very young patients, or very severe patients. Many need to stay on this medication until more research is done on the other new drugs that affect IL-1, and young children, even infants are being put onto anakinra for CAPS and DIRA to save their lives, and prevent severe complications from these syndromes.
With the discovery of genetic causes for various diseases happening more and more, there will likely be more conditions that are found with genetic causes that may benefit from drugs already being produced for other uses. The laws need to be changed to develop protection for those conditions, and the drugs that are found to be of benefit to them.
Currently, there is no protection unless the drug company chooses to file for Orphan Drug status for a drug, and FDA approval for new uses for currently produced and FDA-approved drugs. Mostly, Orphan Drug status is sought during the time that the drugs are first being developed, but now with the rise of new genetic findings, there will be more conditions that will be discovered to have genetic causes that will show direct benefit from current or other drugs that are not under the Orphan Drug protection that act on the condition.
There must be some way to ensure more protection for patients, as there is so much being discovered today, and developed by scientists daily. The effort to implement these laws and changes to the system will save lives, and benefit more conditions over time. It is a necessary thing to do to the benefit of many, and the right thing to do.
Thanks, and please contact me for more information. There is an informative pdf about CAPS on our website at this link:
http://www.nomidalliance.net/downloads/finalCAPSbrochure_web.pdf
Sincerely,
Karen Durrant, RN
President of the NOMID Alliance
www.nomidalliance.net