I often hear people say things like “we need more government research in X disease”. It’s times like this I want to bury my face in my hands or bang it on a wall a couple of times, because statements like this display a certain naivete which – while cute – isn’t particularly helpful in getting the job done. It’s just not how things work. Of course the government can have a significant role to play in advancing disease-specific research, but the greatest leaps forward are usually through a public-private partnership. So, I thought… why not provide a relatively short summary of just what goes into researching a cure… especially with the movie Extraordinary Measures coming out in only a few weeks? After all, this film deals with a desperate dad trying to save his children’s lives, and is not an uncommon story within the biotechnology community at all.

Step one – Fund research: This is likely the step that most people are thinking of when they say “we need more government research”. The National Institutes of Health’s (NIH) 50,000 competitive grants support more than 325,000 researchers at over 3,000 universities medical schools and research institutions across the United States. Without progressive increases in NIH funding, the number of researchers will inevitably decrease as the funding dries up. But that is only the first step.

Step two – Protect patents: Each biologic drug (for example) takes approximately $1.2 billion and 12-15 years in development before it reaches the approval stage. Without strong patent protection, there is little incentive for private research institutions to spur innovation and breakthrough treatments.

Step three – Invest in start-ups: Of all the FDA-approved biotech therapies on the market through 2005, about one-third were from companies receiving SBIR funding (aka: small businesses or start-ups). Counterproductive tax policies and limiting SBIR funding to research start-ups like these can literally rip the rug out from under promising therapies.

Step four – Strengthen the FDA: The FDA regulates products worth nearly $1.5 trillion annually, but total FDA appropriations don’t even cover operating costs. By fully funding the FDA, the agency will be able to manage (in a timely fashion) the growing volume of breakthrough therapies that have to be fully vetted before going to market… otherwise, it will act as an unnecessary bottleneck in getting desperately needed treatments to people.

Step five – Fair pricing: More than 400 biotech drugs are in clinical trials for 200+ diseases such as Alzheimer’s, heart disease, diabetes, AIDS and many others. Government-imposed pricing mandates can eviscerate the necessary early-stage investment, leaving desperate patients with nowhere to turn.

Step six – Assure safety: Patients must be helped to understand that there are risks as well as benefits to any given treatment. No treatment is 100% effective, and unrealistic expectations of a treatment’s safety will lead to lawsuits and limit access to needed medications. However, enhancing safety through high-tech post-market surveillance can enhance safety dramatically. It’s really all about having a common sense approach to medicine and accepting a certain level of personal responsibility when undergoing treatment.

All of these are crucial components to finding effective treatments, so I look forward to seeing how this will be portrayed in Extraordinary Measures. In short, finding cures is much more complicated than many people realize, and the people involved in it are taking heroic risks with their careers and sanity. A simplistic demand for “more government research” into a disease doesn’t accomplish much. Providing a competitive environment for researchers to take the necessary risks does.

What do you think? Is there anything missing from this list?