Preparation is key in preventing delays for obtaining orphan drug designation

Orphan Drug products give patients with rare diseases hope. The Orphan Drug Act, originated in 1983, features incentives for companies to develop drugs to treat a rare disease or condition by giving tax credits on clinical trials, eligibility for seven years of market exclusivity and no user fees.  More and more companies are jumping on this bandwagon which no doubt means a demanding number of requests for the FDA to review.  FDA continues to work on their review processes as Gayatri Rao, M.D., J.D. explains in her recent article on the FDA voice.  Her message to companies is to prepare designation requests with much attention to detail and requirements.  This preparation will minimize the number of review cycles needed with the FDA and get the therapies to the patient faster.  Call on Pearl Pathways to work with you and provide the attention to detail you require in your orphan drug designation requests.

Pearl Pathways’ Pannucci reflects on the Innovation Showcase

I attended the Innovation Showcase yesterday at the Dallara headquarters in Speedway Indiana.  Aside from the event itself, we were all surprised to be a part of the breaking news when Governor Mike Pence showed up to deliver a speech to announce a new $1B investment in the Indiana entrepreneurial and innovation ecosystem.  Big news for the state that can only help to propel new technology and new startup company formation in Indiana.  There were 75 exhibiting companies at the event consisting of pre-commercialization startups and scale up companies in software, technology and life science sectors.  There were also scores of venture capital groups and investors attending the event to make connections and watch the pitch events.  Fantastic panel discussions and advice from venture capital leaders like, Tim Kopp, Bill Godfrey and Don Aquilano added substance and real world experience that many of the startups in the room were craving to hear.  Ben Pidgeon, Joana Milliken and Ting Gootee did an excellent job to bring this event all together.  All in all,  an incredible turnout for a mid-week event which really underscores the activity level and momentum that central Indiana, and the state as a whole, has been building in 2016.  The appetite for deals, new companies, top notch technology and venture capital has really been building as of late, spurring new investment, corporate relocations, jobs and economic announcements that seem to happening daily.

So what does this all mean and why is this happening in Indiana.  I think that answer is a multi-part answer, but goes something like this:

  1. We have fantastic research and talent coming out of our universities (particularly Purdue via the Purdue Foundry folks)
  2. We have a growing entrepreneurial mindset to start and grow new companies (thanks in part to companies  and success stories like ExactTarget, now Salesforce)
  3. We have state and regional programs creating an infrastructure platform and resources to support startups (recent $1B investment most recent example of that)
  4.  We have seasoned leaders to help lead and advise our startups
  5.  We have venture capital ready, willing and able to help finance startup companies (as evidenced by the turnout at the Innovations Showcase)

Seems like a perfect recipe to succeed.  Next steps: Rinse, lather and repeat.

Could FDA be made into an independent federal body?

Former FDA commissioners recently gathered at the Aspen Ideas Festival’s Spotlight Health discussing ways to minimize bureaucratic and political red tape.  FDA’s science based recommendations of safety and efficacy of food and drugs many times have political pressures and challenges to overcome.  Former regulation executives coming together to share and leveraging their many years of experience may ultimately produce a positive change.   Perhaps there could be a better path forward than the current system we have in place?  Check out Sy Mukherjee’s informative article on Fortune.

cGMP clinical grade stem cells’s Melissa Fassbender recently posted an article on a talk she had with James M. Anderson, M.D., Ph.D., Deputy Director, NIH Division of Program Coordination, Planning and Strategic Initiatives. This exchange reveals information on clinical grade stem line development that is hoped to help accelerate early-stage clinical research for diseases including Alzheimer’s, Parkinson’s, spinal cord injury, diabetes and muscular dystrophy.  Anderson talks about how clinical grade cells are developed under regulations and quality control measures that ensure quality and safety standards of the cells for potential clinical applications in humans.  Click here to read Fassbender’s article and contact us if you need help with your cell therapy regulatory filings or clinical trials.  Pearl has extensive experience with cell therapy products.

Pearl’s Caldwell completes Goldman Sachs 10,000 Small Businesses & welcomed to alumni group

GS10KSBPearl Pathways is excited to announce that its President and CEO, Diana Caldwell recently completed an elite entrepreneurship training program in the Babson College Cohort of Goldman Sachs’ 10,000 Small Business program.  She is among 137 entrepreneurs who graduated in May 2016 representing 34 states, Washington D.C, and Puerto Rico.  Goldman Sachs 10,000 Small Businesses is a national program created to help entrepreneurs create jobs and economic opportunity by providing greater access to education, capital, and business support services. The spring graduates completed the blended online and face-to-face program that delivers intensive practical entrepreneurship training from Babson’s business experts and peers.

To read the full press release, click here.

Pearl Pathways – We’re hiring: Sales and Marketing Associate

Pearl Pathways is a small entrepreneurial minded life sciences services company poised for success, and positioned for growth.  This is a great opportunity to join our company as a Sales and Marketing Associate and help us grow our business to the next level.  Click on this link for details and information on how to apply.

Pharmaceutical and medical device companies need to navigate through many hurdles as they work towards commercializing new drugs, devices and diagnostics that improve and save human life. Pearl Pathways supports life science companies in the development and commercialization of their products.

Reminder of eCTD Deadlines Approaching

A reminder that Electronic Common Technical Document (eCTD) deadlines are approaching. All submissions to the FDA including NDA, ANDA, BLA and DMFs are required to be submitted in eCTD format beginning on May 5, 2017.  IND submissions are required to be submitted in eCTD format beginning on May 5, 2018.  FDA informs that submissions that do not meet the specifications in the eCTD guidance will not be filed or received.  Paper will no longer be accepted after the dates noted.

Pearl Pathways has an experienced team to help with your regulatory submission authoring, publishing, and eCTD filings. Contact us for a value added partner to assure no interruption in your regulatory filings.

Let Pearl Pathways help with your biocompatibility concerns

Pearl Pathways invites you to look over a final guidance just released by the FDA Use of International Standard ISO 10993-1, “Biological evaluation of medical devices – Part 1: Evaluation and testing within a risk management process.”  This encourages medical device manufacturers to first start with a risk assessment to determine if new biocompatibility testing is required for premarket submissions.  It also explains new test-specific recommendations to follow.

The FDA is also holding a free webinar on July 21, 2016 discussing this final guidance. Click here for information on how you can dial-in to hear the presentation and also ask questions. Registration is not necessary.

Pearl Pathways’ experienced team is ready to assist your product development team with your biocompatibility evaluations. Contact us to be your partner in accelerating your product development regulatory pathways.

News on Compassionate Use

The FDA has been working to improve the process used by physicians to request Expanded Access — Compassionate Use to investigational drugs and biologics for their patients who need these therapies to stay alive.  In a statement released in early June from FDA Commissioner Robert Califf, M. D., the FDA released 3 guidance documents with information on Compassionate Use drugs and biologics.

The first guidance to note covers Individual Patient Expanded Access Applications – Form FDA 3926.  New Form 3926 is specifically for physicians to use to request access to investigational drugs for their patients who need these therapies to stay alive.  FDA also has instructions for completing the form.

There are 2 additional guidance documents to note that provide additional clarity on Compassionate Use. One of the guidance documents explains Compassionate Use using a Question and Answer format – click here to read.  The other guidance covers how a patient may be charged for these investigational drugs – click here to read.

Pearl Pathways’ and Pearl IRB’s experienced teams are ready to help you with this process. Expediting regulatory pathways is what we are about — contact us.

Update on OTC Drug User Fee program activity

FDA’s recent public meeting on June 10th discussing a user fee program for over-the-counter (OTC) drugs indicates new activity in this program. Expert opinion was heard at the meeting with the acknowledgement that this is a very enormous undertaking with a small staff of 18 employees to manage.  The idea is that implementing a user fee program may result in great benefits and advances in over-the-counter drugs to help us manage symptoms and conditions safely without the oversight of a doctor.

Zachary Brennan, RAPS, posted an article that has additional informative links that follow the current FDA activity on this topic.  Brennan indicates that FDA is still seeking input/perspectives from stakeholders as they work toward a more defined approach.  Help them take a first “bite” into this huge project by commenting.