An article in the January issue of Therapeutic Innovation and Regulatory Science (formerly the Drug Information Journal) offers continuing insights into how the pharmaceutical and biotechnology industries and their regulators need to be thinking about drug development for the future … and these insights are particularly appropriate when it comes to the rare disease space.

Grignolo’s article (“Collaboration and convergence: bringing new medicines to global markets in the 21st Century”), unfortunately, is only accessible on line to members of the Drug Information Association (DIA). However, the points he makes in his article include the following:

• Manufacturers of all biopharmaceuticals (including those for rare disorders) are going to “face intense pressure to fulfill unmet medical needs by developing safe and effective medicines at affordable prices.”
• Industry must learn to apply “intelligent innovation” to the development of medicines that are “truly innovative and capable of significantly changing the practice of medicine.”
• “Misguided drug development plans, increasingly sophisticated regulatory requirements, and sponsor, regulator, payer misalignment are among the top barriers to global market access.”
• Convergence among regulatory agencies around the world toward common processes and common regulatory requirements is clearly evident.
• The importance of patient access to effective, safe, accessible, and affordable medicines, which implies “drug development with reimbursement in mind.”

Based on a presentation given by Grignolo at the 4th Drug Information Association China annual meeting in Shanghai last year, the article presents a challenge to the biopharmaceutical industry that correlates closely with three of the four key elements of NORD’s vision and mission:

• A nation where people with rare diseases can secure access to diagnostics and therapies that extend and improve their lives.
• A social, political, and financial culture of innovation that supports both the basic and translational research necessary to create diagnostic tests and therapies for all rare disorders.
• A regulatory environment that encourages development and timely approval of safe and effective diagnostics and treatments for patients with rare diseases.

The next decade or so is going to be critical to the development and delivery of innovative treatments for tens of thousands — perhaps even millions — of patients with rare disorders. Only yesterday, PhRMA issued a report entitled Innovation in the Biopharmaceutical Pipeline: A Multidimensional View that included specific comment on a variety of issues of importance to orphan drugs and the management of rare disorders.  However, the understanding that development and approval of such new therapies is not enough on its own, but that we must all work hard to make sure that these products are accessible to the patients who need them, is a critical factor that will require collaboration between many different parties.