The CureTheProcess Campaign strives to inspire science-driven public policy that will increase the predictability of the regulatory process for rare disease treatments. Our goal is to give even the rarest diseases access to the accelerated approval process and fulfill more completely the original intentions of the Orphan Drug Act.  The following goals need to be reached to put orphan treatments on the fast track:  

1.    Establish a new Office of Drug Evaluation for Genetic and Biochemical Diseases, consolidating expertise to review treatments to ensure they are safe and effective.  Biochemical and genetic disorders require specialized training and experience to best evaluate new therapies. The establishment of a new Office of Drug Evaluation for rare biochemical and genetic diseases would assure that the appropriate rare disease experts are recruited and integrated with existing expertise at FDA.

2.  Devise new clinical study design and analysis paradigms for rare diseases that properly account for clinical heterogeneity and disease complexity to properly capture treatment effects.  While traditional randomized, controlled studies have been used in rare diseases, this design is relatively insensitive to changes in heterogeneous patients and fails to allow the assessment of all types of patients with all types of disease outcomes.  A creative effort is needed to develop new paradigms in study design that capture individual benefit in a broad array of patients, utilizing all the clinical data to establish efficacy.  The medical science needs to drive the statistical analysis.

3.  Create a new standard for the surrogate and biomarker endpoints used for rare disorders, to allow treatments for these diseases to have full access to the accelerated approval pathway.  Due to the rarity of the disorders, the use of direct, relevant surrogate or biomarker endpoints as clinical study endpoints is essentially impossible for some rare disorders because none of these surrogates have been validated or evaluated in clinical studies and are therefore unavailable for development use.  However, the data show that biochemical markers relevant to biochemical genetic disorders are far better predictors of disease and treatment effect than many of the approvable surrogate markers currently used for drug approvals.

Outcomes:

• A streamlined development path will shorten timelines and reduce the financial risk associated with development of rare disease therapeutics. The result should be a surge in development activity for even the most rare disorders.
• More patients with rare biochemical and genetic disorders will get earlier access to specific, effective treatments.
• Certain treatments for rare biochemical or genetic disorders that are now unaddressed because of the difficulty in assessing the clinical outcome, will now be targets of drug development as appropriate surrogate markers are identified.
• Investment in early stage biotech companies focused on rare diseases will increase and have a positive impact in local communities and biotechnology jobs.
• A new Office with experts trained and knowledgeable in the disease area, will allow for an improved and more specialized FDA review.