A Guest Blog by Julia Jenkins:

Director of Government Relations, EveryLife Foundation


The Grassroots Movement Behind ULTRA-FAST &

How you can join the Movement!

We are very excited to share the news that rare disease Accelerated Approval language made it into the House draft PDUFA bill.  The language that incorporates the goals of ULTRA is the only mention of rare diseases in the entire 205-page bill.

This is a huge accomplishment for the rare disease community and is a major step toward spurring the development of lifesaving treatments.  While there is still work to be done, and it is too soon to pop open the champagne, I will take a few moments to tell the story of how a few parent activists ignited the grassroots patient community to influence the legislative process.

But first, please help us build on this great success by taking two quick actions to support the FAST Act that includes the rare disease language.  The more support we have for the FAST Act, the better positioned we will be to keep our language in PDUFA:

1) Please add your organization’s name to the FAST support sign-on letter by emailing Emily Roberts at BIO at eroberts@bio.org by Wednesday March 21st
2) Click here to email your Member of Congress and ask him/her to become a Co-Sponsor of FAST

Below is the story of why your advocacy matters:

Last fall, a parent advocate met Representative Stearns (R-FL) at a political fundraiser in Florida.  The parent shared his personal story that compelled Representatives Stearns into action.  Working with Stearns’ office, we drafted a small but practical piece of legislative language that fixed the “catch-22” of the current regulatory pathway that was preventing rare diseases access to the accelerated approval process.  To ensure the bill would pass, our goal was to activate the grassroots community to get enough support from Congress that the language would be included into Prescription Drug User Fee Act reauthorization (PDUFA).

Representative Stearns was joined by Representative Towns (D-NY) to introduce bipartisan legislation HR 3737, the Unlocking Lifesaving Treatments for Rare disease Act (ULTRA).  A team of parent advocates assembled and worked to secure more than 140 patient organizations to sign on to support ULTRA and encouraged more than 2700 advocates to write their Members of Congress asking them to support ULTRA.

We had a huge break when BIO negotiated a deal with PhRMA and the FDA on broader Accelerated Approval reform language that incorporated some of the goals of ULTRA.  The language was included in Senator Hagan’s (D-NC) TREAT Act – S. 2113, the Transforming the Regulatory Environment to Accelerate Access to Treatments.  Over the next few weeks, we worked with Representative Stearns and Towns on language for a new bill that would incorporate both TREAT’s Accelerated Approval language and the language in ULTRA (that directed the FDA to use other scientific data to qualify a surrogate endpoint for use in a clinical trial when the required historical clinical information on the surrogate does not exist or could never practically be obtained).

The result was H.R. 4132, the Faster Access to Specialized Treatments (FAST) Act which was introduced last week just in time to have it incorporated into the house version of PDUFA that was released on Monday.  One cannot avoid the pun that everything came together in the end – ULTRA FAST.  Today we are encouraged that we are joined by both BIO and NORD in advocating for support of FAST and that our few paragraphs that fixed the catch 22 for very rare diseases was included into the draft PDUFA language.

We could not have achieved this goal without the incredible leadership of Representatives Stearns and Towns and their staff. However, our success was due to the hard work of the grassroots rare disease community and individual parent advocates.   Together we were able to:

*Send more than 2700 emails to Congress asking them to support ULTRA
*Bring 70 patient advocates to Washington DC for a Lobby Day to support UTLRA
*Have an additional 200 advocates participate in the Lobby Day from their home by making phone calls to Congress
*Place ads in Capitol Hill trade publications (in print and online) showing the rare disease organizations’ support for ULTRA

Most importantly, having the support of the grassroots community encouraged all the stakeholders to work together to ensure the needs of rare disease patients were incorporated into the broader goals of improving the FDA’s Accelerated Approval pathway.

We could not have been so successful if it were not for the work of BIO, PhRMA and NORD for advocating for the needs of rare disease patients as well as the nearly 3000 advocates who took action to support improving the accelerated approval process for rare diseases.  There are a few key advocates who deserve a very special thanks for going above and beyond, taking time off work, donating money, food, and spending every other spare moment they had on Facebook, emailing, and calling to build a grassroots movement.  There are also a few organizations that deserve mention for not only supporting our efforts but making them their top priority.  Thank you to Roy Zeighami, Jill Woods, Tracy VanHoutan and Patricia Beggiato, the National MPS Society, Sarcoma Foundation of America, and the RARE Project.

That’s all the time we have for congratulations and ‘thank you’s; we must get back to work.  First we must ensure that identical Accelerated Approval language is introduced in the Senate’s version of PDUFA (currently, the Senate’s TREAT Act is missing the considerations in the guidance language that specifically says: “to incorporate novel approaches to the review of surrogate endpoints based on pathophysiologic and pharmacologic evidence in such guidance, especially in instances where the low prevalence of a disease renders the existence or collection of other types of data unlikely or impractical.”)

If identical Accelerated Approval language is not in both the House & the Senate bills then we are subject to Conference Committee where our language will be negotiated by Congressional staff, and really anything could happen.  We also need to ensure that no meaningful changes are made during Mark Up of the two bills when they are in the House’s Energy & Commerce Committee & the Senate’s HELP Committee.  That is why your advocacy efforts to support the FAST Act are so essential.  We must act today, but also as a community, we need to be ready and able to respond within hours’ notice to contact Congress if our rare disease language comes across any additional obstacles.

If you’ve made it this far into the story, I’m going to ask you again to take action:

1)      Please add your organization’s name to the FAST support sign-on letter by emailing Emily Roberts at BIO at eroberts@bio.org by Wednesday March 21st

2)      Click here to email your Member of Congress and ask him/her to become a Co-Sponsor of FAST

3)      Please click the links to “like” our Foundation Facebook page & “follow” us on Twitter to get the latest updates and action alerts on PDUFA

Congress must pass PDUFA by the end of September this year or the FDA will no longer be able to review and approve treatments.  We can celebrate then for a short time, and then go back to work again to ensure the FDA develops the guidance on time and provides a predictable pathway for very rare diseases to use biomarkers and surrogate endpoints in clinical trials.  The EveryLife Foundation, through its Rare Disease Workshop Series, will be hosting its fourth Workshop in November together with Academia, NIH, FDA, Patient Groups, and Industry to work on proposals that the FDA could use in drafting their guidance.

While the scientists and academics debate on the science to be included in the guidance, the patient organizations must continue to build our grassroots advocacy community.  We’ve accomplished a great deal, but more legislation is needed to spur the development of lifesaving treatments.  We were successful with 3000 activists, imagine what we could accomplish if we had 30,000 activists.  I hope that you will continue to join us in our endeavor.  We must continue to move ULTRA FAST to ensure that the patients we are fighting for receive lifesaving treatments in their lifetime.

I will leave you with a familiar quote that really rang true in this story: “A small group of thoughtful people could change the world. Indeed, it’s the only thing that ever has.” Margaret Mead

Read more about what’s in PDUFA: Inside Health Policy: Draft Energy & Commerce Bill Reveals FDA Reform Priorities for User Fee Package



RAREProject and The Global Genes Project
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