- Lab-Grown Mini Organs Could Speed Up Drug Discovery (forbes.com)
The thought of lab-grown organs conjures up Frankenstein-like imagery. The reality however, is somewhat less visually dramatic, with the term ‘organoids’ used to describe tiny 3D structures of human tissue, a millimeter or so in diameter...these tiny lumps of cells are creating a lot of excitement in the world of medical research...Cells in dishes and animal models have been used for preclinical testing of drugs for decades. Success in these experiments is a key hurdle for any new medicine to overcome before being given the green light for all-important human clinical trials...Organoids are most commonly made either from a small sample of tissue needled out of a person or from stem cells cultured in a cocktail of nutrients intent on pushing them towards becoming a particular tissue type. So far, organoids have been made resembling several tissues including lung, liver, brain, kidney and intestine...as a relatively new innovation they are being used to investigate dozens of conditions from infectious diseases to cancer.... A study published last year in Science Translational Medicine by scientists at the University Medical Centre, Utrecht generated organoids formed from the rectal tissue of 71 people with cystic fibrosis and exposed them to experimental drugs. By observing changes in the organoids, the scientists accurately predicted which patients would respond to the therapies in just one week at a cost of around $1200 per patient. The results were so convincing that a positive organoid test is now considered sufficient evidence for insurance companies to fund the new therapies in the Netherlands...
- Experts critical of America’s right-to-try drug laws (thelancet.com)
Laws that allow terminally ill patients access to experimental drugs are gaining momentum across the USA, but experts say they could do more harm than good…the right-to-try laws...are designed to circumvent the FDA…proponents of the laws view the agency's bureaucracy as a roadblock, not a pathway, for desperate patients seeking experimental drugs. "When patients are almost willing to try anything to live…we shouldn't see government stand in the way"…Critics...say the laws are misguided, that drug companies, not the FDA, restrict the availability of experimental drugs because of cost, limited supplies, or concerns about the effect on clinical trials…skeptics believe the laws could do more harm than good, possibly raising false hopes about drugs whose safety and effectiveness have not yet been proven…
- Drugmakers can’t charge beyond making costs for compassionate use: FDA (reuters.com)
Food and Drug Administration said companies could only charge patients for the cost of manufacturing experimental treatments used under compassionate grounds, and it cannot force government or private health insurers to pay for these drugs..."Compassionate use" of experimental drugs allows physicians to prescribe unapproved treatments for patients who have no other satisfactory alternatives in the market...The FDA's move seems to be intended to soften the repercussions of its possible rejection of Sarepta Therapeutics Inc's muscle-wasting drug (Duchenne muscular dystrophy)...FDA last week deferred its decision on whether to approve Sarepta's drug, eteplirsen, after an advisory panel determined that the treatment was not effective...The FDA is trying to create a compromise, saying drug companies can charge for a drug even if its not approved...Sarepta's drug has been in the spotlight over the past few months with patient groups and parents arguing passionately in favor of the treatment to pressure the regulator to approve the drug.
- Europe launches new fast approval scheme for promising drugs (reuters.com)PRIME: priority medicines (ema.europa.eu)
European regulators launched a new scheme...to speed the approval of promising new drugs that address unmet medical needs by offering enhanced support to medicine developers as they work on clinical trials...The European Medicines Agency's initiative called PRIME, which stands for PRIority MEdicines, is the latest example of regulators on both sides of the Atlantic working to evaluate truly innovative drugs more swiftly than in the past...The European agency said it expected around 100 applications a year for its new PRIME scheme, which aims to foster better planning of medicine development to help companies generate the data needed for approval more rapidly...Experimental drugs accepted into the program will get continuous support from an EMA expert, who will be appointed early in the process and provide guidance on overall development plans and regulatory strategy.